OUR Mission 2021 ALS Association Mission Toolkit

4/15/21 FOR INTERNAL USE ONLY

“The ALS Association has been the greatest organizationALS PROOF to be affiliated with because they’ve got chapters around the country to help patients and their families. They do that through support groups and providing in-home care where people may not be able to afford the cost. And that gives a break to the loved ones, the family members that are left to take care of us when we can’t take care of ourselves. ” —John Russo Co-Chair, ALS Focus Patient and Caregiver Advisory Committee Our Mission TABLE OF CONTENTS Our Case for Support ...... vi Our Impact ...... vii Our Vision and Mission ...... viii Our Research The ALS Association Research Program The ALS Association Research Program ...... 1-1 Research ...... 1-4 Research Infographics Scientific Focus Areas ...... 1-12 Current Projects ...... 1-14 Accelerating the Search for A Cure ...... 1-15 Research By the Numbers ...... 1-16 Funded Research ...... 1-17 Advances in ALS Research ...... 1-18 Mission Initiatives The ALS Association’s ALS Roundtable Program ...... 1-20 Thank You to Our 2020 Roundtable Sponsors ...... 1-22 ALS Voice of The Patient Report . . . .4/15/21 ...... FOR . . . . .INTERNAL ...... USE . . . . . ONLY 1-23 ALS Focus Survey Program ...... 1-25 Research Staff ...... 1-28

Our Care NetworkALS PROOF How We Work Summary ...... 2-1 How We Work ...... 2-2 Chapter Membership ...... 2-4

iv 2021 ALS Association Mission Toolkit How We Support Clinical Care Supporting Clinical Care ...... 2-11 Certified Centers ...... 2-14 Where We Support Care ...... 2-15 Impact & Resources Care Services Impact ...... 2-20 Filling the Gap ...... 2-21 Education ...... 2-22 Education ...... 2-23 Assistive Technology ...... 2-25 Children’s Programs and Resources ...... 2-27 The Jane Calmes ALS Scholarship Fund ...... 2-31 ALS Association Care Connection ...... 2-32 Care Services Staff ...... 2-33 Our Advocacy Introduction To ALS Advocacy Introduction ...... 3–1 History of ALS Advocacy ...... 4/15/21 ...... FOR . . . . .INTERNAL ...... USE...... ONLY. 3–2 Timeline of Events ...... 3–3 Advocacy In Action Advocacy Guiding Principles ...... 3–5 How We AdvocateALS . . PROOF...... 3–6 Priorities, Impacts & Coalitions Advocacy Impact ...... 3–10 How We Collaborate ...... 3–11 Advocacy Staff ...... 3–15 Appendix Abbreviated Glossary of Scientific Terms ...... APP-2

2021 ALS Association Mission Toolkit v Our Mission OUR CASE FOR SUPPORT he ALS Association is the only national nonprofit organization fighting the disease on every front — supporting cutting-edge research, enhancing access to clinical care, and Tpromoting better public policies . Our nationwide network of chapters comprise one team with a single mission: to discover treatments and a cure for ALS, and to serve, advocate for, and empower people with ALS .

Everything we do at the Association is centered on people with ALS — the care we support, the policies we advocate for, and the research we advance . The only way we will realize our vision of a world without ALS is to make sure that all of these parts are fully integrated and working together .

y The public policies we advocate for increase funding for ALS research and improve access to care and assistive technology for people with ALS . y The care services programs we support improve the quality of life for people living with ALS, and enables them to participate in clinical trials that advance research toward treatments and a cure . y The research we fund accelerates the development of treatments and technologies for people with ALS, and furthers support for more investment in ALS research . In the past year, the Association has made remarkable strides . Our global research program has supported projects and collaborations that led to breakthroughs in new therapies, diagnostic tools, and technologies to assist 4/15/21 FOR INTERNAL USE ONLY people living with ALS . Our nationwide network of more than 20,000 advocates helped protect and secure millions of dollars in funding for ALS research and enhanced access to treatments, including $40 million in the Department of Defense ALS Research Program and $10 million for ALS PROOF the National ALS Registry . Our care services team served more than 20,000 people living with ALS, including 9,296 people served through our Certified Treatment Centers of Excellence and Recognized Treatment Centers .

Keep reading to learn more about the tremendous work being done throughout the Association . We will continue our work to help people with ALS live their lives to the fullest while we search for new treatments and a cure .

vi 2021 ALS Association Mission Toolkit OUR IMPACT The ALS Association fights every day for people living with ALS, leading cutting-edge research to discover treatments and a cure for ALS and serving, advocating for, and empowering people living with ALS to live their lives to the fullest. RESEARCH Healey Center establishes first platform trial Bringing effective treatments closer to approval $3M Helped support the Healey Platform Trial to Including AMX0035 from Amylyx Pharmaceuticals speed up drug development by reducing the cost that was shown to extend life and slow disease of research by 30% and decreasing the trial time progression, and continued progress on Tofersen, a by 50% . genetic therapy for SOD1-ALS in late phase trials, along with progress on biomarker research . Starting a new effort to stop ALS as soon as possible Starting a new effort to stop ALS even before people get sick by funding the first ever development of Finding out what matters most to people with genetic testing and counseling guidelines for ALS to help with ALS and their families identify and manage their ALS and their caregivers genetic risk and guide treatment decisions on emerging Since launching, over 1,450 people have gene-based therapies . We are also partnering with CDC on participated in the ALS Focus . These surveys a workshop to problem-solve and translate research about bring the perspectives and needs of people with ALS and ALS causes into preventive approaches and treatments to caregivers to the forefront of program and policy decision reduce risk of ALS . making . To join, visit www.als.org/research/als-focus . ADVOCACY

Boosted funding for the ALS Research Program Ended the SSDI 5-month waiting period for people at the Department of Defense (DOD) from $20 living with ALS by spearheading successful efforts to $40 to $40 million in 2020 to support the creation of pass and enact into law the bipartisan ALS Disability MILLION more clinical trials that will lead to the discovery Insurance Access Act . of treatments and a cure . 4/15/21 FOR INTERNALEnsured $10USE million ONLY in continued funding for Protected access to noninvasive ventilators (NIV) $10M the National ALS Registry and Biorepository to for Medicare beneficiaries with ALS by fighting the NATIONAL identify genetic and environmental factors for ALS, Centers for Medicare and Medicaid Services (CMS) provide support to researchers to find treatments decision to add NIV to the competitive bidding program . ALS REGISTRY and a cure, and promote access to clinical trials . Supported over 630 meetings with members of Congress ALS630 PROOF+ and over 500 calls to Congress during the 2020 Virtual Advocacy Fly-In and National ALS Virtual Advocacy MEETINGS Conference . Empowered more than 40,000 ALS ACTION advocates through 20 action alerts — resulting in over 20 ALERTS 85,000 advocacy messages to Congress . CARE SERVICES

21,545 people living with 2,295 veterans received $4,885,387 in ALS served through the chapter assistance through our grants provided through our network in the past year nationwide chapter network Certified Center Program

42,696 people viewed, 25,831 attendees at 9,296 people served through downloaded, or ordered our support groups offered our Certified Treatment Centers educational materials, resource guides through our vast chapter of Excellence and Recognized and medical information packets network across the U .S . Treatment Centers

2021 ALS Association Mission Toolkit vii OUR Vision A WORLD WITHOUT ALS

OUR

4/15/21 MissionFOR INTERNAL USE ONLY TO DISCOVER TREATMENTS AND A CURE FOR ALS, AND ALS PROOF TO SERVE, ADVOCATE FOR, AND EMPOWER PEOPLE AFFECTED BY ALS TO LIVE THEIR LIVES TO THE FULLEST. 4/9/21 FOR INTERNAL USE ONLY

ALS PROOFOUR Research

“We’ve made incredible progress in terms of understanding the biology behind the disease, and there is great hope for people living with ALS” —Dr. Veronique Belzil, former Milton Safenowitz Research Fellow, Mayo Clinic Neuroscience Department, Jacksonville, FL 4/9/21 FOR INTERNAL USE ONLY

ALS PROOF The ALS Association Research Program THE ALS ASSOCIATION RESEARCH PROGRAM SUMMARY scientific staff . Final funding decisions are made by the Research Committee of the ince 1985, The ALS Association has Association’s Board of Trustees . been at the forefront of ALS research . SWe pursue an agenda of effective We also take into consideration ALS research and rigorous research to understand how that we do not fund, and how our funding ALS works and how it might be treated, can complement or accelerate these and to improve the lives of people living efforts . When projects are promising, but with ALS today . Though we are the world’s too large and expensive, we jointly fund largest philanthropic funder, the scientific a project with other ALS philanthropies . challenges posed by ALS are far bigger . We Sometimes those organizations approach fund what we can, coordinate with other us with funding ideas, and in other cases funders on the projects we cannot directly we identify projects and engage other support and invest in the people and funders for support . These projects are still infrastructure needed to make ALS research reviewed by external experts and approved efficient and effective . by our Research Committee . We have been fortunate to collaborate with just about FUNDING STRATEGY every ALS research funder, including ALS Our direct funding is focused on high impact Finding a Cure, Answer ALS, the Muscular 4/9/21 FOR INTERNAL USE ONLY projects anywhere in the world . Our project Dystrophy Association, Project ALS, Target selection approach is modeled after the ALS, and more . We also collaborate with National Institutes of Health . We receive private companies in the ALS space . We structured applications from scientists, no have funded small biotech companies, as well as companion studies, such as matter where theyALS are inPROOF the world, or if they work in academia or industry . Most biomarker studies to larger clinical trials often these applications are requested through our Lawrence and Isabel Barnett at specific times during the year, but we Drug Development Program and Biomarker accept requests for funding at any time . Program, respectively . Applications are scored and reviewed by Our goal is to always ensure impactful outside experts selected for their knowledge work gets funded, even if we do not fund it of the topic proposed . Scores and the ourselves . When we fund a project, we do so reviews are synthesized by Association with the intention that other larger funders,

2021 ALS Association Mission Toolkit 1-1 Our Research such as the federal government (like the almost all the ALS trials in the United States . NIH) or the private sector, will be able to We also are supporting the Healy Center carry the work forward . This strategy works . ALS Platform Trial, which promises to speed For example, an independent evaluation ALS clinical trials . All these efforts move found our Ice Bucket Challenge research forward under the formal stewardship of our spending has served as a critical multiplier research program staff, external reviewers, of funding from other sources . As a direct and Research Committee . result of $40 million in Ice Bucket research funding we awarded, ALS scientists reported MISSION INTEGRATION TO receiving an additional $122 million in ADVANCE RESEARCH follow-on grants from other funders . The Association plays a critical role in research through our other mission areas CAPACITY BUILDING as well . Our 70 plus Certified Treatment We work to increase the capacity of Centers of Excellence, overseen by our Care ALS research as an enterprise . We fund Services program, are critical settings for multiple postdoctoral research fellowships ALS human studies and clinical trials in their (apprenticeships for new PhDs) every year local communities . through the Milton Safenowitz Postdoctoral Fellowship Program . We are proud that over There are many challenges in a person’s 76% of our postdoctoral fellows go on to journey living with ALS, and there is a careers in ALS research, along with a large critical need for robust data to inform percentage of the remaining staying in the decisions of key opinion leaders to drive new programs and policies to improve ALS space in industry or nonprofit jobs4/9/21 . FOR INTERNAL USE ONLY their lives . We are using our ALS Focus We also fund the infrastructure to survey platform to help inject empirical make ALS research more efficient and patient and caregiver preference data into impactful . These infrastructure projects these decisions . We are making these data include repositories of specimens, genetic ALS PROOF available to the entire ALS community information and electronic data that make it without charge, and interoperable faster and cheaper for people to study ALS, with other ALS research tools through and a smarter investment for private capital . global unique identifiers for people We pay particular attention to the with ALS and their caregivers . Our ALS infrastructure and capacity for clinical Roundtable Program is geared toward trials . We are the primary supporter of identifying challenges and coming to the Northeast ALS Consortium (NEALS), a creative solutions with leaders in the ALS network of clinical trial sites that facilitate community – in government, industry,

1-2 2021 ALS Association Mission Toolkit and most importantly, people with ALS in ALS research simply would not have been and caregivers . This program has already possible without the infrastructure and identified the disconnect between FDA broad collaboration we have supported over treatment approval standards compared to time . We were able, for example, to fund insurance coverage and payment standards . multiple gene studies and databanks that pooled information and talent . More than Our Advocacy program supports the 250 scientists around the world analyzed National ALS Registry for ALS research, which over 100,000 samples to identify a new not only counts the number of people with genetic link to ALS, the KIF5A gene . ALS but collects data and funds scientists throughout the country to better understand MOVING FORWARD the causes of ALS . We fight successfully for increased federal research resources for Our research strategy is robust and ALS, and the federal government spends successful . There is an ever-growing more than $10 for every $1 we directly fund . portfolio of exciting drug targets and genes, We also recently fought to double the clinical studies, scientists, and companies Department of Defense commitment to new to the ALS space . As the ALS clinical ALS research to $40 million . Our Advocacy trial enterprise grows larger and faster, and Research programs also collaborate on we will have to pay closer attention to engagement with the FDA, which has led preclinical safety and toxicity projects, to many improvements in the conduct of to ensure there are enough promising candidates to test . And as these clinical clinical trials, including the new ALS Clinical studies bear fruit and practice starts to Trial Guidelines released in 2019 . 4/9/21 FOR change,INTERNAL we will USE have toONLY shift resources Our focus on the overall ALS research to study new treatments in real world enterprise, and efforts to steward the ALS settings, to ensure these new treatments research community to greater capacity, are being used effectively . Some of these efficiency, and collaboration, are working . In expanded areas of study might be disease 2014, we were ableALS to supportPROOF 42 principal modifying therapies in clinic . They may investigators, and by the end of 2018 that also be vital assistive technology and care support had grown five-fold, to 237 principal giving practices that need data to drive investigators . In addition, the number of both better service delivery and policy . And researchers collaborating on ALS-related we will be supporting research to detect scientific papers has nearly doubled since and treat ALS earlier and earlier in the 2014 . disease process, in the hopes of preventing Collaboration in the ALS space has already disability altogether . We are just getting borne fruit . Some of the recent discoveries started .

2021 ALS Association Mission Toolkit 1-3 Our Research RESEARCH Our Strategic Initiatives

he ALS Association has announced nine patient access to trials . The trial will take place important research strategic initiatives at the Sean M . Healey & AMG Center for ALS at Tsince the ALS Ice Bucket Challenge Massachusetts General Hospital in collabora- to advance the search for treatments and a tion with the Barrow Neurological Institute and cure for the disease . In this document, we the Northeast ALS Consortium (NEALS) . This have included details on the goals of each investment builds on the Association’s long initiative, along with information on The ALS history of supporting research at Mass General Association’s role and funding commitments . – over $9 million just in the last 10 years .

Strategic initiatives are large global Read the news: collaborations focused on the understanding y www.als.org/stories-news/als-association- of the disease, targeting new therapies, invests-3-million-first-ever-als-platform- expediting clinical trials, and making RNA trial-speed-clinical-trial and DNA sequencing data available to the entire ALS research community . These y www.massgeneral.org/neurology/als/ initiatives will generate data and resources news/2020-01-14-ALS-association-makes- available for researchers globally . Central commitment-healey-platform-trial to all the major new collaborations are the y Learn more about the ALS Platform Trial people living with ALS . here: www.massgeneral.org/neurology/ 4/9/21 FOR als/research/platform-trialINTERNAL USE ONLY Learn more about each strategic initiative here: www.als.org/research/research-we- PROJECT MINE fund/partnerships-initiatives/strategic- $1.4 million original commitment partnered initiatives with Greater New York and Georgia Chapters. ALS PROOF Total commitment since 2014 is over $2.1 ALS PLATFORM TRIAL million. The ALS Association invested $3M over three years in the ALS platform trial to accelerate An international, large-scale research drug development. initiative devoted to discovering genetic causes of ALS and to ultimately finding a This groundbreaking trial will test multiple cure . The goal is to identify GENES associated therapies for efficacy at the same time, pro- with ALS by performing whole genome viding time and cost savings and increasing sequencing on at least 15,000 ALS patients

1-4 2021 ALS Association Mission Toolkit plus 7,500 healthy controls worldwide, A consortium that is a collaboration between resulting in an open-source ALS genome numerous global laboratories capable of database, in conjunction with the collection generating and analyzing thousands of DNA of skin samples to make ALS patient- sequences from people with ALS . The goal is induced pluripotent stem cell (iPSC) lines . to discover new genetic contributors of ALS Our funding supports the U .S . arm of this to then translate into clinical solutions for initiative, led by Jonathan Glass, M .D . (Emory ALS . It houses all data in a central repository University) and John Landers, Ph .D . (University that is freely available to the research of Massachusetts Medical School) . Original community worldwide . Original funding funding announced in October 2014 . announced in October 2014 . Additional funding announced in February 2019 . NEW YORK GENOME CENTER— CENTER FOR GENOMICS GENOMIC TRANSLATION FOR OF NEURODEGENERATIVE ALS CARE (GTAC) DISEASES (NYGC CGND) $3.5 million commitment partnered with Greater New York Chapter In 2015, The ALS Association funded $2.5 million in our original commitment and partnered with the Greater New York Chapter. That commitment was matched with an additional $2.5 million contributed by the 4/9/21 FOR INTERNAL USE ONLY Tow Foundation. In 2019, the Association committed an additional $3.5 ALS PROOF million, including a $1 million commitment from the Association’s Greater New York Chapter. That brings our total contribution to $6 million.

2021 ALS Association Mission Toolkit 1-5 Our Research

A collaboration between Biogen and disease pathways linked to ALS and related Columbia University Medical Center (CUMC) disorders . In addition to sequencing samples to better identify new targets for treatment collected from study participants, CReATe development, in order to understand how is building a resource of biosamples that different genes contribute to various clinical have attached detailed clinical information, forms of ALS . This will translate into clinical providing a unique and critical resource for trials that are more focused . This project will biomarker development . The biorepository follow 1,500 people with ALS and collect will enable the discovery and validation of detailed clinical data, sequence their DNA biomarkers relevant to therapy development and store blood cell samples to generate for patients with ALS and related disorders . iPSCs . This study will allow correlation of In partnership with The ALS Association, ALS clinical symptoms to genetic causes and CReATe is funding pilot biomarker projects help stratify patients for future clinical trials . using this resource, as well as other Funding announced in August 2015 . biorepositories, including the Northeast ALS Consortium (NEALS) biorepository CReATe supported by The ALS Association . Funding Clinical Research in ALS and Related Disorders announced September 2015 . for Therapeutic Development (CReATe) Consortium: $450K commitment for biomarker study and biorepository and an $835K commitment to Drs. Paul Taylor, Jinghui Zhang, and 4/9/21 FOR INTERNAL USE ONLY Michael Benatar for DNA sequencing .

A Rare Diseases Clinical Research Consortium (RDCRC)ALS PROOF that forms part of the National Institutes of Health (NIH) Rare Diseases Clinical Research Network . The goal of CReATe is to identify new genes and novel

1-6 2021 ALS Association Mission Toolkit NEUROLINCS process; supports NeuroBANKTM (see below); and supporting Phase IIA pilot $2.5 million commitment partnered with the clinical trials with relevant biomarkers Greater Philadelphia Chapter aimed at developing novel high-potential A partnership with NIH’s National Institutes of ALS treatments . A TDP 43 PET Tracer Grand Neurological Disorders and Stroke (NINDS) . Challenge was launched as part of ALS ACT . This National Institutes of Health (NIH)-funded And was awarded to Dr . Timothy Miller in collaborative effort is between various research 2017 to develop a novel TDP-43 biomarker groups with expertise in iPSC technology, to track disease progression over time, disease modeling, OMICS methods, and improve diagnosis time, and understand computational biology . The goal is to use iPSC whether a drug is hitting its target . Original lines from ALS patients and healthy controls funding announced October 2014 . and OMICS methods to identify unique cell TM signatures that are specific to various subtypes y NeuroBANK : original funding of of motor neuron diseases, in order to better $200,000 under ALS ACT – further develop therapies and design clinical trials . expanded in August 2016 for an Funding announced July 2016 . additional $1,679,091, bringing our total commitment to $1,879,091. A patient- ALS ACCELERATED centric clinical research platform and THERAPEUTICS (ALS ACT) central repository that sets the framework to allow for standardization of ALS Original $10 million commitment matched patient information (including proteomic, with an additional $10 million contributed by genomic, and clinical data) that is linked ALS Finding a Cure® 4/9/21 FOR INTERNALacross simultaneously-running USE ONLY research A novel academic-foundation-industry studies, locations, and modalities . It is partnership with ALS Finding a Cure, initiated designed to host, curate, and disseminate with researchers from General Electric (GE) this information . Global Unique Identifier Healthcare and four academic NEALS sites (GUID) technology generates a patient- ALS PROOF to accelerate treatments for people living specific character string that securely with ALS . It is using the following strategies identifies a patient without revealing to develop new therapeutic approaches their true identity . NeuroBANKTM is part for ALS: supporting development of of NYGC projects, GTAC, Answer ALS, and neuroimaging tools as potential ALS other ALS research projects . biomarkers; supporting projects focused on decreasing the production of misfolded NEURO COLLABORATIVE proteins, and reversing neuroinflammation, A $4.8 million commitment in October two major contributors to the disease 2014 — funding through The ALS Association

2021 ALS Association Mission Toolkit 1-7 Our Research with contributions from the Orange County Finkbeiner laboratory at the Gladstone and Wisconsin Chapters. To date, we have Institutes, which is affiliated with University committed a total of $7.8 million. of California San Francisco, which will further develop robotic technology for screening An initiative founded as a collaboration drugs in motor neuron cell culture . The between three leading California laboratories Cleveland Laboratory is collaborating with aimed at discovering and developing new Martin Marsala, M .D ., at the University of potential ALS therapies that can be delivered California San Diego and Brian Kaspar, Ph .D ., to pharmaceutical companies for further at the Research Institute at Nationwide development . The three laboratories are the Children’s Hospital in Ohio . In 2014, The ALS Svendsen Laboratory at Cedars-Sinai in Los Association Golden West Chapter, along with Angeles, which will develop and maintain a Advisory Trustees Jim Barber and Linda Della, Motor Neuron Core Facility to create iPSC partnered with the National ALS Association lines from people with ALS that will be to build the Neuro Collaborative concept . For openly shared; the Cleveland Laboratory at more information, click here. Original funding the University of California San Diego, which announced October 2014. will spearhead the development of antisense therapy against the C9orf72 gene, the most ANSWER ALS common genetic cause of ALS; and the The ALS Association contributed to its development/business plan and is a partner with Team Gleason and others to advance this initiative. We plan to contribute funds as 4/9/21 FORthe programINTERNAL evolves. USE ONLY An initiative spearheaded by Steve Gleason to challenge ALS researchers to come up with a solid plan to find a cure for ALS . Its strategy includes two impact goal arms . One ALS PROOF is designed for immediate impact to help ALS patients live more productive lives by supporting affordable assistive technologies and services . The other arm is designed to increase our understanding of the biology of ALS through “omics,” approaches to identify new targets for therapeutic development . As part of this initiative, all DNA samples from participants will be sequenced by the

1-8 2021 ALS Association Mission Toolkit New York Genome Center (NYGC), which ticals . Partnership announced January 2016. will be funded through ALS Association Funding announced in November 2016. research programs . In addition, NeuroBANKTM will be an integral part of the program . GNS HEALTHCARE Projects funded as part of ALS ACT, the The ALS Association contributed $500,000 to Neuro Collaborative, and NeuroLINCS form GNS Healthcare to use artificial intelligence an important foundation for Answer ALS . (AI) to create a comprehensive disease model Partnership announced in September 2015. to advance research into ALS.

ALS ONE – MASSACHUSETTS GNS Healthcare will use its powerful machine ALS PARTNERSHIP learning platform, REFS, in conjunction with the rich Answer ALS patient datasets, which The ALS Association partnered with ALS are accessible to clinicians and scientists ONE and ALS Finding a Cure to fund $2 throughout the ALS research community . million each for specific clinical and research The project will be led by Dr . Iya Khalil, initiatives to maximize collaborations to find chief commercial officer and co-founder treatments and a cure for ALS. of GNS Healthcare . The ALS Association’s An initiative founded by Kevin Gosnell, a partnership with GNS Healthcare will person who lost his battle to ALS, to bring transform Answer ALS’s petabytes of patient together leading neurology experts and care data into mechanistic models, connecting specialists in Massachusetts to leverage their genetic, molecular, and biochemical variables institutions’ strengths to expedite progress to- to clinical outcomes that will allow in silico ward finding a treatment for ALS by 2020 while experiments to be performed at a rapid 4/9/21 FOR INTERNAL USE ONLY improving care now . Institutional partnerships rate on the computer . These rapid, high- include Massachusetts General Hospital, throughput computational experiments will the ALS Therapy Development Institute (ALS explore the numerous factors in the REFS TDI), the University of Massachusetts Medical Answer ALS data models that drive disease progression and drug response . Discoveries School, and CompassionateALS PROOF Care ALS . Under the ALS ONE umbrella, we funded research will then be evaluated and validated with projects of Dr . Steven Perrin from ALS TDI, Dr . wet lab experiments and, eventually, clinical Nazem Atassi from Mass General, and Dr . Rob- studies . Funding announced July 2018 . ert Brown from U . Mass Medical School . ALS REPRODUCIBLE ANTIBODY The Association’s funding to ALS TDI helped PLATFORM (ALS RAP) move its lead compound AT-1501 from pre- clinical studies into a phase I clinical trial The ALS Association contributed $200,000 through TDI’s affiliate Anelexis Pharmaceu- in partnership with the Motor Neurone

2021 ALS Association Mission Toolkit 1-9 Our Research

Disease Association and the ALS Society of Association’s investment will fund cloud Canada for a total contribution of $600,000 storage, cloud computing, and the genomic to support ALS-RAP. sequencing of 3,000 people living with ALS .

ALS-RAP was created as a public-private The creation of a central repository was partnership comprised of world experts in conceived by three researchers: antibody generation and validation, including y John Landers, Ph .D ., University of Structural Genomics Consortium (SGC) and its Massachusetts Medical School associated labs at the Montreal Neurological Institute (MNI) McGill University in Montreal y Jonathan Glass, M .D ., Department of (Canada), the University of Oxford (UK), Neurology & Pathology, Emory University and the Karolinska Institute (Sweden) . The y Bryan Traynor, M .D ., Ph .D ., National Institute funding will support the creation of an open- on Aging, National Institutes of Health access pipeline to validate antibody research y Read more here: www.als.org/stories- and provide the ALS research community news/als-association-contributes-33- with the highest quality reliable, renewable million-fund-groundbreaking-genome- antibodies for ALS genes . Standard operating sequencing-database procedures will be established to characterize ALS antibodies – both commercially available and newly created – to ensure these exceed SGC and the Neuro’s stringent quality criteria to establish a public list of “gold-standard” antibodies that will ultimately lay a solid foundation for successful ALS therapies 4/9/21 . FOR INTERNAL USE ONLY Funding announced in March 2018.

GENOME-SEQUENCING DATABASE ALS PROOF The Association contributed $3.3M to fund a centralized, cloud-based, genome-sequence database, including $100,000 from The ALS Association Georgia Chapter.

The first of its kind database will allow all researchers to share genomic data and conduct standardized analyses, leading to identification of new ALS drug targets . The

1-10 2021 ALS Association Mission Toolkit 4/9/21 FOR INTERNAL USE ONLY

ALS PROOF Research Infographics Our Research SCIENTIFIC FOCUS AREAS The ALS Association Commitment

The ALS Association supports a wide breadth of specific fields of study that are critical to advancing ALS research . We are always on the lookout for the next cutting-edge field to invest in . BIOMARKERS NATURAL HISTORY The ALS Association is committed to STUDIES biomarker discovery, as their potential These studies are important to understanding is immense . Identifying biomarkers is vital the natural disease course of ALS . The ALS to improving diagnosis, following disease Association is supporting several natural progression, tracking response to therapy, history studies of SOD1 and C9orf72 ALS, and make clinical trials more efficient . Our which are critical to helping inform patient support of the TDP-43 Biomarker Grand care and clinical testing of new treatment Challenge Program is just one example . approaches .

ASSISTIVE TECHNOLOGY CLINICAL STUDIES The ALS Association is working The ALS Association supports to develop accessible, portable devices clinical management grants to for people living with ALS, in order to help improve the lives of people living with ALS them maintain a high quality of life . The ALS 4/9/21 FORand INTERNALtheir caregivers, USE along ONLY with clinical trials Assistive Technology Challenge winners are to accelerate treatments through the drug dedicated to achieving this! development pipeline .

ENVIRONMENTAL COGNITIVE STUDIES FACTORSALS PROOF There is a great deal of evidence Multiple factors in one’s lifestyle and that cognitive impairment is connected to surroundings, such as smoking and military ALS, such as overlap with Frontotemporal service, are the only known ALS risk factors . dementia (FTD) . The ALS Association is The ALS Association champions multiple committed to improving understanding of efforts to better understand these risk factors why and how this connection takes place . and drive discovery of other factors that may contribute to ALS .

1-12 2021 ALS Association Mission Toolkit GENETICS STEM CELLS

The number of genes identified to Stem cell technology is progressing cause familial ALS has multiplied since the rapidly, and the ALS Association is discovery of SOD1 . Many efforts are underway spearheading work on several critical fronts to to identify more ALS genes and target them advance this key research tool . for therapy . The ALS Association continues to make significant investments in identifying DISEASE MODELS new genes and has supported all the major The ALS Association’s research ALS gene discoveries in history . portfolio supports a variety of model systems used for understanding DISEASE MECHANISMS disease pathways and testing promising ALS is a complicated disease involving compounds . multiple disease pathways . The ALS Association encourages research to PRECISION MEDICINE discover novel pathways . Understanding how The ALS Association has helped ALS disease works on many biological levels establish and currently supports several is necessary to identify potential therapeutic partnerships and precision medicine targets . programs to aid in the identification of new disease genes and targets for drug therapy . NANOTECHNOLOGY There is growing interest in using nanotechnology as a delivery tool for 4/9/21 FOR INTERNAL USE ONLY ALS therapeutics, and we are on the cutting edge, funding this exciting technology .

DRUG DEVELOPMENT The ALS AssociationALS PROOF is supporting development of several different treatment approaches, including small molecules, stem cells, and gene therapy . Our early support of antisense drugs in 2004 has paid off! Antisense therapies have already proven effective in spinal muscular atrophy (SMA), and are in trial targeting both SOD1 and C9orf72 .

2021 ALS Association Mission Toolkit 1-13 CURRENT PROJECTS The ALS Association is Accelerating Progress Towards Treatments (as of February 2021)

The ALS Association's Global Research Program

OR MN 2 3 MI NY NH RI WI 4 PA 19 1 2 3 8 MD MA CT 28 IL OH 12 2 2 CO 2 VA CA 1 MO 3 14 7 NC TN 1 2 AZ 4 GA 2 TX 4 LA

FIN 1 UK 1 FL CAN 5 5 GER BEL 7 IRL 2 1 3 FR SWI 1 ITL 2 4 ISR 1

4/9/21 FOR ActiveINTERNAL Projects USE by LocationONLY

CHL UNITED STATES 2 AUS 1 Arizona – 4 Missouri – 7 California – 14 North Carolina – 1 Colorado – 1 New Hampshire – 1 Connecticut – 2 New York – 19 Florida – 7 Ohio – 2 ALS PROOF Georgia – 2 Oregon – 2 Active Illinois – 2 Pennsylvania – 8 Louisiana – 1 Rhode Island – 2 Research Massachusetts – 28 Tennessee – 2 Maryland – 12 Texas – 4 162 Projects Michigan – 4 Virginia – 3 Minnesota – 3 Wisconsin – 3 In an effort to accelerate progress toward finding treatments and a cure WORLDWIDE for ALS, as of 02/01/2021, The ALS Australia – 1 France – 1 Israel – 1 Association is currently funding 162 Belgium – 1 Finland – 1 Italy – 4 active research projects total. There Canada – 5 Germany – 2 Switzerland – 2 are 134 within the United States. Chile – 2 Ireland – 3 United Kingdom – 5

1-14 2021 ALS Association Mission Toolkit ACCELERATING THE SEARCH FOR A CURE The ALS Association is funding scientific projects across the research pipeline . We support a wide breadth of scientific focus areas – each is critical to advancing ALS research .

Basic Research: Understand causes of the Biomarkers: Discover, validate, and develop disease and identify new therapeutic targets new ALS biomarkers . Biomarkers are needed through gene sequencing, omics analyses, and to improve measurement of ALS diagnosis, epidemiological studies . prognosis, and a person’s response to treatment . Therapeutic Development: Develop drugs and technologies against novel and genetic targets Patient Cohorts: Support infrastructure to run to test for preclinical efficacy and safety . clinical trials, support registry and recruitment 4/9/21 FOR INTERNAL USE ONLY for clinical trials, and enable open source Clinical Phases (I/II/III): Test drugs for safety sharing of data and biospecimens for research . and efficacy in people with ALS . (Please note: The ALS Association does not fund phase III Community Engagement: Integrate trials) . patient voice and perspective, and engage ALS PROOF stakeholders across the community including Managing ALS: Impact lives of people with ALS other nonprofits, industry, investors, today through funding research in developing government, and regulators . assistive technology, measuring patient/ caretaker burden, and improving quality of life . Training & Education: Support training through postdoctoral and clinical fellowships . Provide Research Tools: Generate, characterize, and training for principal investigators, trial distribute standardized tools and animal coordinators, and clinical sites for optimal models for preclinical use by the research clinical trials . community .

2021 ALS Association Mission Toolkit 1-15 Our Research RESEARCH BY THE NUMBERS The ALS Association’s collaborative and global approach to funding research continues to lead to significant advances by top ALS researchers all over the world .

dedicated to research ACTIVE RESEARCH since the ALS Ice Bucket Challenge to PROJECTS $113 ACROSS 13 advance treatments MILLION and a cure 162 COUNTRIES CLINICIAN potential new ALS antisense drugs and numerous SCIENTIST other drugs on the horizon DEVELOPMENT 4/9/21 FOR INTERNAL aimedUSE ONLYto slow or stop the 2 AWARDS IN 2020 3 progression of ALS new global (MATR3, CHCHD10, TUBA4A, TBK1, strategic NEW NEK1, C21orf2, ANXA11, TIA1, KIF5A, initiatives ALS PROOF ALS GLT8D1, and LGALSL) identified since launched or the 2014 ALS Ice Bucket Challenge each 9 supported 11 GENES representing new therapeutic targets

ALS drug trials and new postdoctoral 35+ observational fellows 33+ studies are chosen in actively enrolling*. 8 2020 *(as of 2/9/2021)

1-16 2021 ALS Association Mission Toolkit FUNDED RESEARCH The ALS Association Funded Research Across the Research Pipeline up to Phase 2 Clinical Trials Thanks to our generous donors, The ALS researchers focused on projects involving Association awards various research projects people living with ALS . throughout the year as part of its competitive y Lawrence and Isabel Barnett Drug Development portfolio, which include the following: Program fosters collaborations with companies/ academia to fund milestone-driven research y Multiyear Investigator-Initiated Grants to focused on preclinical studies to move established investigators . treatment approaches closer to the clinic . y One-year Starter Grants to investigators new y Pilot Clinical Trials to support up to and to the ALS field or senior postdoctoral fellows including phase II clinical trials that are establishing their own independent position . associated with a comprehensive biomarker y Milton Safenowitz Postdoctoral Fellowships program to test novel, high-potential treatment to encourage and facilitate promising new approaches in people with ALS . scientists to enter the ALS field . Fellows work y Managing ALS Awards to fund research for with a senior mentor and receive extensive improving clinical, psychological, and social exposure to the ALS research community management of ALS, focusing on both people through meetings and presentations . living with ALS and their caregivers . y Strategic Challenges are crowdsourcing initiatives such as the ALS Assistive Technology For questions, please contact Challenge to help people living with ALS [email protected]. communicate with ease (partnered with 4/9/21 FOR INTERNAL USE ONLY Prize4Life) and the TDP43 PET Tracer Grand Challenge to discover a biomarker to track TDP43 in the body (partnered with ALS Finding a Cure®) . y Strategic Initiatives that invite researchers to ALS PROOF submit collaborative projects that address research gaps, areas of high risk-high reward, and/or areas that provide novel opportunities . This grant program encompasses large, collaborative research programs . For more information, visit the strategic initiative page and refer to the strategic initiative talking points . y Clinical Development Fellowships, in partnership with the American Academy of Neurology (AAN), to support ALS clinician-

2021 ALS Association Mission Toolkit 1-17 ADVANCES IN ALS RESEARCH The ALS Association is the largest private funder of ALS research in the world. We advance ALS science by building research infrastructure and funding individual research projects and engaging the patient community in the search for treatments and a cure. The ALS Association is proud to provide funding for these exciting innovations: Healey Center establishes Investing in infrastructure and data first platform trial access accelerates ALS research. $3M Helped support the Healey With over $1.2M for the Platform Trial to speed up PRO-ACT (Pooled Resource drug development by reducing the cost of Open-Access ALS Clinical research by 30% and decreasing the trial Trials) database which is the time by 50% . world’s largest ALS clinical data repository . Since its launch in 2012, over Starting a new effort to stop ALS as 900 researchers from 68 countries in both for-profit and non-profit sectors, have ob- soon as possible tained access and analyzed these datasets Starting a new effort to stop ALS even resulting in 32 scientific publications . before people get sick by funding the first ever development of genetic testing and counseling guidelines for ALS to help with ALS Bringing effective treatments and their families identify and manage their closer to approval and genetic risk guide treatment decisions Including AMX0035 from Amylyx on emerging gene-based therapies . We are Pharmaceuticals that was shown also partnering with CDC on a workshop to 4/9/21 FORto extendINTERNAL life and USE slow diseaseONLY progression, problem-solve and translate research about and continued progress on Tofersen, a genetic ALS causes into preventive approaches and therapy for SOD1-ALS in late phase trials, treatments to . reduce risk of ALS along with progress on biomarker research . Continuing the search during ALS PROOF Finding out what matters a global pandemic most to people with ALS and By committing funding for 24 new projects spanning diverse areas their caregivers of research such as biology, therapeutics, Since launching, over 1,450 people have biomarkers, infrastructure, and clinical trials . participated in the ALS Focus . These surveys The Association managed over 162 active bring the perspectives and needs of people projects with a multiyear commitment of over with ALS and caregivers to the forefront of $55M and $113M committed to research from program and policy decision making . To join, the ALS Ice Bucket Challenge . visit www.als.org/research/als-focus .

1-18 2021 ALS Association Mission Toolkit 4/9/21 FOR INTERNAL USE ONLY Mission ALS PROOF Initiatives Our Research THE ALS ASSOCIATION’S ALS ROUNDTABLE PROGRAM ince 2019, The ALS Association participation among Roundtable Program has assembled members of the ALS Smembers of the ALS community for community for whom candid, facilitated discussions that shape the travel is challenging . Association’s strategic planning efforts and find solutions to improve ALS care, advocacy, BENEFITS OF and research . PARTNERING IN ALS PROGRAM OBJECTIVES ROUNDTABLES The Roundtable Program provides a forum for solution-based discussions with ALS GLOBAL COLLABORATION stakeholders to accelerate the development All members of ALS Roundtables benefit of meaningful treatments for ALS and from this collaborative forum, which improve the lives of people with ALS and promotes understanding and builds their caregivers . All Roundtable meetings partnerships with industry peers, clinician include individuals with expertise and and scientific leaders, policy makers, experience from a variety of sectors who regulatory officials, public and private reflect diverse points of view on ALS-related insurance representatives, ALS Association issues . The group identifies challenges 4/9/21 faced FORleadership, INTERNAL and —USE most importantlyONLY — people by people with ALS and their caregivers and with ALS and their caregivers . then recommends action that significantly impacts future care and treatment of the DRIVING PRIORITY PROGRAMS disease . ALS PROOF The Roundtable meetings are a unique While these meetings began as in- opportunity to shape and inform program- person gatherings to build relationships and advocacy-based solutions to challenges and rapport among stakeholders, the faced by the ALS community . COVID-19 pandemic prompted the most 2020 Roundtable meetings to go virtual . INFORMING ALS RESEARCH AND POLICY Though the format has changed, the level of AGENDAS engagement, participation and productivity Roundtable meetings inform agendas and remains exceptional, even allowing greater decision making on topics that are critical to

1-20 2021 ALS Association Mission Toolkit the ALS community, including clinical trial The November 2020 Roundtable issues, regulatory and access evaluations, was centered around decreasing ALS payment determinations for ALS therapies, complications for people with ALS, with and more . the goal of identifying common ALS complications, focusing on those that may be PAST ROUNDTABLE TOPICS preventable . We brainstormed on what can y Setting ALS Association Strategic Priorities be done by the Association in collaboration (spring 2019) with our community (especially with clinicians and care services) to reduce complications . y Access to New Therapies (fall 2019) Decreasing complications could potentially y Reducing Time to Diagnosis (spring 2020) clear the way for more efficient clinical trials y Reducing ALS Complications (fall 2020) and improved quality of life and longer/better lives for people with ALS . The fall meeting 2020 PROGRAM OVERVIEW also provided important updates from the The April 2020 Roundtable was focused Association regarding its strategic planning on reducing time to diagnosis, a critical efforts and ongoing and future work to secure topic for the ALS community that brought access to future therapies . together 69 attendees representing key ALS stakeholders . ALS diagnosis is ALS ROUNDTABLE WORKING complex and is primarily based on the GROUPS physician’s interpretation of clinical Stemming from past Roundtable meetings, symptoms and ruling out other diagnoses . three working groups were formed in 2020 People with ALS present first symptoms 4/9/21 FOR to INTERNALdelve deeper USE into eachONLY topic and put and progress in varied ways adding to solutions into action . the diagnosis complexity . During the meeting, participants mapped out key y The Increasing Clinical Trial Enrollment challenges, potential solutions, and near- working group (formed as result of spring term action steps ALSthe communityPROOF can 2019 Roundtable) met this year . The first take to streamline and accelerate the recommendation the group is pursuing is process of arriving at an ALS diagnosis . to increase enrollment with a system to As an outgrowth from the meeting, the match people with ALS to clinical trials . Association formed two multi-stakeholder y The Time to Diagnosis working group is working groups: time to diagnosis with focused on reducing time to ALS diagnosis a focus on education and awareness for by improving the education and resources general neurologists and on genetic testing for ALS diagnosis, with a focus on general and counseling (see below) . neurologists . The group first aims to

2021 ALS Association Mission Toolkit 1-21 Our Research

develop a multi-stakeholder consensus clinicians and genetic counselors in order statement about the benefits of early to accelerate the genetic characterization diagnosis . of the ALS population . y The Genetic Testing and Counseling WHAT TO EXPECT working group is focused on making a Roundtable meetings focus on a clearly measurable impact on genetic testing defined topic that is selected ahead of time and counseling landscape for people by the Association staff and the Roundtable with ALS and their families . So far, the Advisory Panel . A professional moderator group is working on developing a genetics facilitates and guides the discussion . glossary to emphasize consistent and Everyone has the opportunity to be heard . clear terminology around ALS genetics . After the Roundtable, an executive summary The Association is also supporting the is distributed to participants . Sponsors are development of Genetic Testing and invited to participate in working groups to Counseling Guidelines, which are aimed at further delve into identified challenges to improving genetic testing literacy among advance concrete solutions . THANK YOU TO OUR 2020 ROUNDTABLE SPONSORS

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1-22 2021 ALS Association Mission Toolkit ALS VOICE OF THE PATIENT REPORT he ALS Voice of the Patient Report is a treatments, and their hopes for future ALS groundbreaking document that gives therapies . This information is also providing Tthe FDA and other key stakeholders, context of the disease burden to the FDA including drug developers, health care to inform the Agency’s review of new drug providers, and insurance companies, data applications . This report documents the from people with ALS and caregivers immense unmet medical need we all are about the everyday impact and burden working to confront . The content of the ALS the disease has on peoples’ daily lives, Voice of the Patient Report was derived from their experiences with currently available the IMPACT ALS survey initiative that was conducted in December 2017 . This survey was led by The ALS Association with our partners Cytokinetics, Biogen, Ionis Pharmaceuticals, regulatory experts, ALS clinical thought leaders, and, most importantly, patient and caregiver input .

The vision of the IMPACT ALS survey was to systematically gather and expand available data on the perspectives and experiences 4/9/21 FOR of INTERNALpeople with ALSUSE and ONLY caregivers . We surveyed both people living with ALS and their care partners . A total of 1,534 people participated . Of that, 813 were people with ALS, 74 were people assisting a person with ALS PROOF ALS, and 647 were caregivers . People with ALS were represented from across the U .S ., with representation across states comparable to the portion of the U .S . population in each state .

While the report’s findings will probably resonate with people living with ALS; what is important here is the systematic

2021 ALS Association Mission Toolkit 1-23 Our Research documentation of these findings and their y With current treatments mainly focused formal submission to the FDA . This is the first on supportive care, people with ALS time such data has been gathered to this would like treatment options that stop extent across disease progression . disease progression, improve muscle weakness, and assist with breathing or KEY TAKEAWAYS OF THE respiratory function . ALS VOICE OF THE PATIENT y The majority of people with ALS reported REPORT: having fears about the future . The fears y Individuals with ALS and caregivers most frequently reported were related reported to dying, “leaving significant family too soon,” symptoms of The vision of the followed by “dying fatigue and from respiratory weakness, IMPACT ALS survey failure .” The next followed by was to systematically most reported fear speech problems, was “spending the shortness of gather and expand family’s savings on breath, difficulty available data on medical care .” sleeping, and The IMPACT ALS pain . the perspectives survey is the first of a y These progressive and experiences of series of patient and symptoms result people with ALS and caregiver surveys in a loss of 4/9/21 FOR INTERNAL USE ONLYthrough the ALS independence caregivers. Focus survey and led to less platform initiative, time spent at which is a cross- sector collaboration to place the preferences work or school, socializing,ALS PROOF or traveling . of people with ALS and their caregivers in the y People with ALS do not report high center of treatment and policy development, satisfaction with currently available through surveys and studies . ALS Focus treatments and reported that such will build from the IMPACT ALS survey treatments “somewhat help” with and conduct robust research on patient managing ALS and everyday impacts of and caregiver needs, preferences, and disease . Breathing assistance devices were experiences to produce generalizable and reported to be very inconvenient and open data for the entire ALS community . burdensome, but necessary .

1-24 2021 ALS Association Mission Toolkit ALS FOCUS SURVEY PROGRAM Bringing the perspectives of people with ALS and their caregivers to the forefront of research, care, and advocacy. LS Focus is a patient- and caregiver- and disease led survey program to understand progression . Athe experiences and needs of people y Conduct with ALS and their caregivers, putting their benefit-risk research and other preference perspectives at the center of treatment and studies . policy making . ALS Focus surveys collect data y Use ALS Focus measures and findings to on the symptoms, burdens, and everyday inform: impacts of ALS that people face throughout the disease journey so that the whole ALS – Clinical trial design, clinical community can benefit . All components of ALS endpoints and scales, and regulatory Focus are built on a cross-sector collaboration decisions . between The ALS Association, people with ALS, caregivers, industry, academia, and the federal government .

WHAT ARE THE GOALS OF ALS FOCUS? The overarching goal of ALS Focus is to4/9/21 conduct FOR INTERNAL USE ONLY scientifically rigorous patient- and caregiver- focused research to improve their quality of life . Specifically, we aim to: y Distribute surveysALS throughout PROOF the year to the ALS community . y Create a collaborative setting that invites candid feedback and insights from patients and caregivers . y Develop and validate tools such as clinical outcome assessments to measure what is most important to people with ALS and caregivers across the spectrum of disease

2021 ALS Association Mission Toolkit 1-25 Our Research

– Value-based reimbursement models use NeuroGUIDs, such as the National ALS for ALS therapies and products . Registry and clinical trials . – Federal regulators and policy makers in government agencies such as the WHO CAN PARTICIPATE? FDA and Centers for Medicare and People living with ALS Medicaid Services (CMS), as well as Current or past caregivers of people members of Congress . with ALS – Clinical care, care services, and home HOW LONG WILL IT TAKE? health practices . Once registered, each secure online survey WHY IS IT IMPORTANT TO can be completed in approximately 15-25 PARTICIPATE IN ALS FOCUS? minutes . Scientifically rigorous efforts to collect WHERE CAN I PARTICIPATE? experience and preference data from people y Access the survey at als.org/ALSFocus . with ALS and caregivers were largely non- existent before ALS Focus . Through this y Learn more here: www.als.org/ALSFocus program, the Association is committed to and for questions email: ALSFocus@alsa- bringing the voice of the ALS community to national.org the forefront to inform how researchers, health *Participants must be at least 18 years old care providers, policy makers, and payors and reside in the United States . Survey make decisions about improving the lives of instructions and questions are presented in people with ALS and those who care for them . English . In addition, the FDA calls for rigorous input4/9/21 FOR INTERNAL USE ONLY from patients and caregivers to inform drug ALS FOCUS ACHIEVEMENTS development . ALS Focus data address this Since January 2020, ALS Focus achieved the need . following milestones:

Importantly, all de-identifiedALS PROOF data and y Secured funding for ALS Focus through findings from each ALS Focus survey are multiple major corporate sponsorships . free and open to the entire ALS community y Built out a Focus survey platform and data after a six-month embargo period . ALS Focus repository housed at NeuroBANK™ through assigns a neurological global unique identifier the Neurological Clinical Research Institute (NeuroGUID), a secure de-identified code, to at Massachusetts General Hospital . each participant, making it possible to link experience and preference data from Focus y Increased membership of the Patient and with data from other ALS research studies that Caregiver Advisory Committee to 20, which

1-26 2021 ALS Association Mission Toolkit includes people with ALS and current and survey to share through the Focus website past caregivers across the country, all lending and other communication materials . (www. their unique perspectives and expertise . als.org/ALS-Focus/survey-results/) y Since launching, over 1,450 people signed y Conducted the second ALS Focus survey up to participate in ALS Focus . on what matters most to people with ALS . y Conducted the first ALS Focus survey This survey uses the ALS Health Index – Understanding Insurance Needs and Short Form developed by Chad Heatwole, Financial Burdens – along with an ongoing MD, MS-CI at the University of Rochester demographics survey . In total, 530 people through an Association grant . Over 675 with ALS, current caregivers and past people participated . caregivers participated in the demographics y Launched a new health status survey to survey and 440 participated in the gauge where Focus participants are in the insurance survey . disease journey . y Expanded and updated a suite of y Upcoming ALS Focus surveys topics include communication materials to increase Caregiver Burden and Risk Tolerance for awareness and recruitment, People with ALS . including a dedicated Focus web page (www.als.org/ ALS-Focus), social media, national emails, and turn-key information for distribution . 4/9/21 FOR INTERNAL USE ONLY y Prepared the first survey dataset and analysis tools for Focus’ corporate sponsors to accessALS PROOF before the six- month embargo period . y Visualized and interpreted results from the first

2021 ALS Association Mission Toolkit 1-27 Our Research RESEARCH STAFF NEIL THAKUR, service in setting clinical performance PH.D., CHIEF measures . In his post-doctoral-fellowship, he studied the interactions between jails, MISSION OFFICER Medicaid and behavioral health care, and Neil Thakur brings more how changes in health financing impacted than two decades of people’s utilization of these systems . During experience as a public graduate school, he worked throughout health expert to the fight the Connecticut behavioral health system, against ALS . He has led The ALS Association’s helping to implement managed care and mission programs – research, care services, health information systems, and raise tens of and advocacy – since 2018 . millions of dollars in competitive grants .

Prior to joining the Association, Neil served Neil won many awards for his government in the National Institutes of Health (NIH) service, including several NIH Director’s Office of the Director, where he supported Awards, and the Secretary for Health and NIH governance and helped make NIH Human Services’ award for Meritorious research more open and less burdensome . Service, the second highest award that the He managed the world’s largest policy to Secretary can bestow . He holds a Ph .D . in make biomedical research papers publicly Health Policy from Yale University School accessible and co-chaired the White House of Public Health and completed a NIMH taskforce that lead to the requirement that postdoctoral fellowship in mental health 4/9/21 FOR INTERNAL USE ONLY all federal science agencies adopt similar services research at the Cecil G . Sheps Center policies . He also spent a year on detail to for Health Services Research at the University the US Senate Special Committee on Aging, of North Carolina at Chapel Hill . He lives in raising awareness about quality issues in Maryland with his wife Jen . long-term health care, particularly around ALS PROOF Alzheimer’s care and pharmaceuticals . KULDIP DAVE,

Prior to his time at NIH, Neil worked with PH.D., VICE health systems in many capacities . He PRESIDENT, was Assistant Director of Health Services RESEARCH Research and Development at the Dr . Kuldip Dave, Vice Department of Veterans Affairs (VA), leading President, Research, joined an evaluation service for the VA health The ALS Association in 2019 . Reporting to system and represented the VA research Neil Thakur, he oversees the Association’s

1-28 2021 ALS Association Mission Toolkit Research Program . In this role, he develops JILL YERSAK, PH.D., and implements the broader research vision VICE PRESIDENT, by setting annual priorities and department MISSION STRATEGY goals including research funding strategy, research programs, and strategic initiatives . Dr . Jill Yersak, Vice He is also responsible for the operational, President, Mission Strategy, staffing, and budgetary oversight of the joined The ALS Association research program . He engages with various in October 2015 . Reporting stakeholders including key opinion leaders to Neil Thakur, she helps oversee the strategy from academia, government, industry, and of projects and programs that span the other nonprofit organizations . He serves as a Association’s mission – research, care, and key communication lead for programmatic advocacy . In this role, she leads mission efforts by presenting the Association’s programs including the ALS Roundtable perspective and strategy to diverse Program and ALS Focus survey program . She audiences at workshops/conferences/ is also responsible for communicating ALS forums and by developing content for research in an accessible way by developing written publications, blogs, social media, and giving research presentations tailored informational videos, perspective papers, to people living with ALS, their caregivers, and journals . and loved ones . She provides Association- wide support, at the national office and Dr . Dave received his bachelor’s degree throughout the chapter network and in all in Biology from Rutgers University in departments, with research information New Brunswick, NJ . He received his Ph .D . needs . These include donor outreach and degree in Pharmacology and Physiology 4/9/21 FOR researchINTERNAL development USE ONLY training . As a former from Drexel University College of part of the Research Communications team, Medicine in Philadelphia, Pa . He went on she played an integral part in implementing to do postdoctoral fellowship at a small the Association’s blog, research website biotechnology company in Pennsylvania redesign, and the mission toolkit . ALS PROOF where he was first exposed to the drug development process . He went to work for Dr . Yersak received her bachelor’s degree in the pharmaceutical company Wyeth for Biology at Ursinus College in Collegeville, two years before joining The Michael J . Fox Pa . After college, she served as a research Foundation for Parkinson’s research . At MJFF, technician at the Children’s Hospital he oversaw a $30M portfolio and led two of Philadelphia in the department of priority therapeutic areas . After nine years at Human Genetics and Molecular Biology, MJFF, he joined The ALS Association has the focused on a pediatric genetic disease head of the Research program . called 22q11 .2 Deletion Syndrome . She

2021 ALS Association Mission Toolkit 1-29 Our Research then went on to complete her Ph .D . at ASHTON FERRARA, Thomas Jefferson University in Philadelphia, MPH, MANAGER, with a focus on a neurodegenerative RESEARCH disease called Kennedy’s Disease . Dr . Yersak then moved to Providence, R .I . to OPERATIONS complete her postdoctoral fellowship Ashton Ferrara started under the mentorship of Dr . Anne Hart with The ALS Association in the Neuroscience department at in September 2019 . Reporting to Kuldip Brown University . During this time, she Dave, she is responsible for issuing spearheaded a project to generate research contracts and managing contract precise ALS C . elegans models (which negotiations, tracking compliance with are microscopic worms) and co-wrote a funded awards, supporting the development successfully funded ALS Association grant of new research programs and collaborating based on this project . on impact measures of the research program and reporting outcomes . Ashton received SARAH PARVANTA, her bachelor’s degree in Public Health from PH.D. MPH, James Madison University in Harrisonburg, DIRECTOR, ALS VA and a Master of Public Health (MPH) with a concentration in Health Promotion from FOCUS Liberty University in Lynchburg, VA . Prior to Dr . Sarah Parvanta, ALS joining the Association, Ashton worked as a Focus Director, has a Ph .D . Contract Administrator with Patient-Centered in communication from the University Outcomes Research Institute (PCORI), where 4/9/21 FOR INTERNAL USE ONLY of Pennsylvania and a master of public she managed pre and post award activities for health (MPH) from the University of North Healthcare Research, Research Infrastructure Carolina at Chapel Hill . Dr . Parvanta leads (PCORnet) and Task Order based Contracts . development and implementation of ALS Focus, a survey program to understand ALS PROOF SELLAM BIRHANE, the experiences, preferences, and needs MBA, MANAGER, of people with ALS and their caregivers . MISSION She specializes in survey design, data analysis, and studies on health perspectives PROGRAMS and quality of life . In her previous role as Sellam Birhane is the a research analyst at RTI International, Manager for Mission she evaluated the impact of The ALS programs at the ALS Association . She is Association’s research program following responsible for helping to coordinate ALS the ALS Ice Bucket Challenge . mission programs, including overseeing any

1-30 2021 ALS Association Mission Toolkit mission programs that emerge from the ALS Engagement as a Graduate Assistant, mainly Roundtable program . She is also responsible performing the duties of an office manager, for the overall project management of new event planner, and student mentor . She mission programs, tracking and executing also served as a volunteer project manager against milestones and action items, as for a health careers institute, creating a well as management and communications work breakdown structure and doing risk with working groups and committees . She management for the client . After graduation also provides support to existing mission she went on to work as a Business Analyst programs such as ALS Focus and ALS at CGI in Rosslyn, VA . In this capacity she Roundtables . assisted in risk management activities, user troubleshooting, requirements analysis, and Sellam received her bachelor’s degree from basic programming . She also served as a the University of Mary Washington (UMW) Program Coordinator for The ALS Association Fredericksburg, VA . She also graduated with on a temporary basis before becoming the a master’s degree in Business from UMW in Manager for Mission Programs . 2018 . While working on her MBA she worked for the Office of Student Activities and

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ALS PROOF OUR Care Network

“We would not be where we are today without the help of others. We immediately reached out to The ALS Association Evergreen Chapter and they have been immensely helpful in our journey.” – Janetta Michelsen, Caregiver, Seattle, Washington 4/15/21 FOR INTERNAL USE ONLY

ALS PROOF How We Work SUMMARY he ALS Association empowers people The ALS Association becoming the leading affected by ALS by supporting increased organization in program delivery . Taccess to clinical care and support services on a nationwide basis . This includes OUR APPROACH TO CARE a network of chapter-based professionals SERVICES delivering a robust portfolio of care and An ALS diagnosis impacts the individual support programs designed to enhance quality diagnosed, as well as their family and loved of life, a suite of multidisciplinary clinical ones . Thus, the Association’s core care services programs where diagnosed persons can access and programs have been developed to expert clinical care, and comprehensive specifically address common needs in the ALS community and professional educational community . programs offering tools that support families, health care practitioners, and volunteers in the y Access to consistent care, treatment, and delivery of care and support . services y Resource and support services that OUR HISTORY enhance quality of life From its inception, The ALS Association y Quality care and support leveraged by has been the only nationwide nonprofit 4/15/21 FOR INTERNALcollaboration USE with partnersONLY organization fighting ALS on every front, delivering a consistent set of core programs and services to families living with ALS . The early years focused on in-person volunteer information and supportALS PROOF services . The portfolio of services has seen significant growth in recent years, including the initiation of a formal multidisciplinary Clinical Program of Excellence . Chapters expanded information, education, and support services delivered by professionally- prepared staff with expertise in all phases of the ALS journey . Strategic outreach and collaboration with medical providers, allied health professionals, service support organizations, and vendors have resulted in

2021 ALS Association Mission Toolkit 2-1 Our Care Network HOW WE WORK s a result of our commitment to processes to actively assess the needs of the clinical care and education, families communities we serve in order to address Ahave access to information they gaps in service delivery . This serves as the may need to develop a care plan that foundation for developing and delivering will result in enhanced quality of life and innovative services and for increasing access optimal personal and family outcomes . to care services . Surveys, focused conversations, and advisory y Designing, implementing, and monitoring groups — which include diagnosed persons, certification and other clinical programs caregivers, families, and care teams — help that support consistency and quality the Association identify unmet needs . in services provided through Certified Regular analysis of models of delivery, usage, Treatment Centers of Excellence, and outcomes helps the Association refine its Recognized Treatment Centers, affiliated programs, services, and processes to identify providers, and other delivery models — priorities and maximize positive outcomes . all based on established best practices The Association uses continuous feedback and standards of care, mechanisms and quality improvement y Building upon our educational resource libraries — including written publications, videos, web pages, and other media 4/15/21 FOR INTERNAL USE ONLY — for the provision of consistent information for our communities . ALS PROOF y Engaging in quality improvement reviews and training that supports an organizational culture that is attractive to a highly motivated and competent

2-2 2021 ALS Association Mission Toolkit workforce serving our community, assumes a lead role in developing including professional and knowledge collaborative relationships and partnerships development, management, team with the communities we serve, including building, professionalism, interpersonal medical providers, other like-minded communication, and work-life balance . organizations, researchers, policymakers, y Hosting a biennial National Clinical regulatory agencies, and others to strengthen Conference services provided to engage and initiatives more than 500 The Association undertaken . health care assumes a lead The ALS Association professionals supports a wide who work on ALS role in developing breadth of specific and other motor collaborative fields of study neuron diseases that are critical to discuss best relationships and to advancing ALS practices and partnerships with research . Active guidelines, participation of multidisciplinary the communities we people living with team care and serve... ALS in these studies coordination, is paramount 4/15/21 FOR INTERNAL USE ONLY ALS-relevant to their success programs and provides the and services, research and technology foundation for continued work in areas that updates, and other topics critical to the are of keen interest and show a promising field . ALS PROOF positive impact on people as they live with y Hosting monthly educational webinars this disease . presented by experts for people living The Association’s Clinical Management Grant with ALS, their caregivers and family Program supports clinical management members, chapter staff, and professionals studies in order to improve care for people who work with people affected by ALS . with ALS . The program focuses on the full The Association continues to strengthen spectrum of clinical management, including relationships internal to the Association gaps in the delivery of care, the development while acknowledging the interdependencies of telemedicine, assistive technology, and among chapter network, Care Services, mental health care for people living with ALS Research, Public Policy, and national office and their caregivers . support departments . The Association

2021 ALS Association Mission Toolkit 2-3 Our Care Network CHAPTER MEMBERSHIP PROGRAMS AND SERVICES OFFERED THROUGH OUR CHAPTER NETWORK (SPECIFIC PROGRAMS MAY VARY BY CHAPTER) CUMULATIVE NUMBER OF PEOPLE REGISTERED WITH EACH CHAPTER

Alabama Chapter 220 Arizona Chapter 606 Arkansas Chapter 175 Central & Southern Ohio Chapter 495 Connecticut Chapter 321 DC/MD/VA Chapter 928 Evergreen Chapter 597 Florida Chapter 1200 Georgia Chapter 670 Golden West Chapter 1834 Greater Chicago Chapter 465 Greater New York Chapter 1041 Greater Philadelphia Chapter 1596 Greater Sacramento Chapter 289 Greater San Diego Chapter 255 Indiana Chapter 515 Iowa Chapter 275 Kentucky Chapter 305 Louisiana/Mississippi Chapter 362 Massachusetts Chapter 385 Michigan Chapter 613 Mid-America Chapter 756 MN/ND/SD Chapter 628 Nevada Chapter 167 New Mexico Chapter 120 4/15/21 FOR INTERNAL USE ONLY North Carolina Chapter 827 Northern New England Chapter 209 Northern Ohio Chapter 409 Oklahoma Service Area 111 Orange County Chapter 240 Oregon and SW Washington Chapter 535 Rhode IslandALS Chapter PROOF94 Rocky Mountain Chapter 595 South Carolina Chapter 255 St. Louis Regional Chapter 347 Tennessee Chapter 423 Texas Chapter 1207 Upstate New York Chapter 398 West Virginia Service Area 116 Western Pennsylvania Chapter 355 Wisconsin Chapter 606 0 200 400 600 800 1000 1200 1400 1600 1800 2000

2-4 2021 ALS Association Mission Toolkit INFORMATION AND RESOURCE including diagnosed persons, caregivers, REFERRALS families, medical professionals, and community health care providers . y Maintaining a physical, electronic, and y video inventory of educational and Providing in-service educational informational materials related to ALS, programming to community health including diagnosis, prognosis, treatments, care agencies or providers as part of research and clinical care options, a strategically planned professional common associated challenges, typically outreach program, such as local home accessed equipment and services, health or hospice organizations . insurance and benefit information, quality y Presenting relevant information regarding of life issues, and associated potential ALS and chapter resources to the general solution options . community as part of a strategically- y Offering a packet of information for planned community awareness program people new to the chapter, containing (for example, partnering with a local information critical to those newly Rotary Club) . diagnosed with ALS, including the value y Participating in state, regional, and of multidisciplinary clinical care, the value national health care professional of developing a strategic care plan, and conferences as subject matter experts, the benefits available through accessing or as an exhibitor or platform presenter (for example, at a State SLP Conference, chapter programs and services . 4/15/21 FOR INTERNAL USE ONLY y Producing a medical information packet Hospice Association, the Paralyzed to be given to first response professionals Veterans of America Annual Summit, and medical providers in urgent and or an American Academy of Neurology emergent medical situations . Meeting) . ALS PROOF y Providing referrals to local, regional, and CHAPTER CLINICAL LIAISON national resources including appropriate PROGRAMS medical care in the clinic, home, or y Creating partnerships and coordinating hospice environment; access to medical care and services between The ALS equipment and health care services; Association chapter and local ALS insurance and benefit options; veteran’s Association Certified Treatment Centers of services; and psychosocial services . Excellencesm, ALS Association Recognized EDUCATION PROGRAMS Treatment Centerssm, or Affiliated Clinics . y Hosting programs that meet the various y Facilitating chapter care services staff educational needs of stakeholders, attending ALS clinics to provide support

2021 ALS Association Mission Toolkit 2-5 Our Care Network

services to patients, families, and the local for diagnosed persons, caregivers, and ALS community . families . y Providing financial or ‘in-kind’ support y Developing targeted support or to ALS Association Certified Treatment program opportunities focused on the Centers of Excellencesm and ALS specific needs of constituents, including Association Recognized Treatment caregivers, children, or homebound Centerssm . persons . y Cultivating relationships with community y Providing navigation assistance as health care practitioners including diagnosed persons and their families speech-language pathologists, physical transition from private insurance to public therapists, occupational therapists, insurance programs, applying for disability respiratory therapists, social workers, and benefits, or accessing other community, home health, hospice, and other health regional, and nationwide resources . care providers . y Supporting grant funding to offset the cost SUPPORT PROGRAMS of professional counseling services . y Facilitating understanding of various care y Coordinating and facilitating support modalities, including treatment, palliative group meetings to provide education, care, and hospice care . information, and networking opportunities y Supporting family end-of-life care planning with a focus on respecting 4/15/21 FOR INTERNAL USE ONLY care determination; personal, cultural, and religious values; and beliefs ALS PROOF as practiced by individuals and families . CAREGIVER PROGRAMS y Providing expanded services to address unique needs of ALS

2-6 2021 ALS Association Mission Toolkit family caregivers, including caregiver-only y Providing navigation assistance to families support or resource groups . investigating options and applying for y Facilitating caregiver self-care and caregiver resource benefits . instructional education programs . y Providing referrals to professional service y Hosting caregiver acknowledgment or providers as needed . retreat programs . DURABLE MEDICAL y Supporting access to The ALS Association EQUIPMENT (DME) PROGRAMS Care Connection Program, which utilizes y Facilitating independence and quality of an online calendar to coordinate family life for people living with ALS by providing needs with available family, friends, or information and education related to the community volunteers . benefits of durable medical equipment y Supporting grant funding to offset the cost (DME) . of caregiver respite services . y Providing appropriate referrals to qualified y Participating in National Family Caregiver DME providers . Month activities . y Implementing a DME Loan Program y Offering a Bereavement Engagement consistent with national DME Loan Program, respectfully acknowledging program policies enabling persons with a loss and reaching out to survivors no or limited resources timely access to on a time-specified basis to offer 4/15/21 FOR INTERNALappropriate DMEUSE equipmentONLY on loan . encouragement and support . y Initiating and cultivating relationships CARE MANAGEMENT with qualified DME providers interested GUIDANCE AND in providing service excellence to the ALS CONSULTATIONS community, including collaborating with ALS PROOF an identified vendor to support operation y Providing routine communication with of DME Loan Program . diagnosed persons and families regarding progression-appropriate information, and ASSISTIVE TECHNOLOGY AND anticipated challenges and needs . AUGMENTATIVE & ALTERNATIVE y Providing guidance to support diagnosed COMMUNICATION PROGRAMS persons and families in accessing health (AT/AAC) care services and making informed health y Providing education and information care choices based on knowledge of ALS, related to how ALS impacts health care resources, insurance, financial, communication and what types personal, and community resources . of interventions are available to

2021 ALS Association Mission Toolkit 2-7 Our Care Network

support augmentative or alternative Loan program policies enabling persons communication . with no or limited resources timely access y Initiating and cultivating relationships with to appropriate DME equipment on loan, augmentative/alternative communication such as an iPad or speech-generating (AAC) equipment vendors and speech- device (SGD) . language pathologists to facilitate access y Providing expanded program services to appropriate evaluation, selection and including employing or contracting with training in the use of communication a speech language pathologist (SLP) or equipment . assistive technology professional (ATP) to y Implementing an assistive technology (AT) deliver comprehensive AT/AAC support Loan Program consistent with national services . assistive technology/augmentative/ HOME VISIT PROGRAMS alternative communications (AT/AAC) y Providing personalized in-home or virtual home visits to consult with families in the comfort of their own homes regarding the disease, typical progression, anticipated common challenges, available resources, and guidance in developing a personalized strategic health care plan . y Developing collaborative relationships with existing community health care 4/15/21 FOR home-visitINTERNAL providers, USE ONLYsuch as state or county health services, home health, Visiting Nurse Association, and hospice to maximize positive outcomes of service delivery . ALS PROOF GENERAL GRANT PROGRAMS y Mitigating the enormous financial costs associated with addressing the challenges associated with maintaining quality of life during an ALS journey by providing grant funds to offset out-of-pocket expenses . y Developing a general or specific grant funding program which may include

2-8 2021 ALS Association Mission Toolkit the following eligible expenses: youth and expanding other general durable medical equipment (DME), services — including education, support assistive technology/augmentative/ groups, conferences, and retreats — alternative communications (AT/AAC), and programs to include and address caregiver respite, transportation, home children and youth, subject to age- modification, medical or health care appropriate communications and copays, or skilled or non-skilled home activities . health care . y Providing targeted educational and SPECIFIC GRANT PROGRAMS informational projects for children and youth, such as backpacks, information y Cultivating relationships with qualified packets, children’s newsletter, family days, agencies dedicated to providing excellent picnics, and youth camps . in-home health services at a preferred y Collaborating with recognized children contracted rate, including nursing, and youth content experts to develop physical therapist (PT), occupational youth-focused conferences or supported therapist (OT), speech-language activities designed to provide age- pathologist (SLP), and certified nursing appropriate information related to assistant (CNA) services . Support services ALS progression and care, along with via grant funding as available . empowering networking opportunities, y Initiating and cultivating relationships with such as DME Youth Conference and ALS 4/15/21 FOR INTERNAL USE ONLY qualified transportation organizations Youth Bowling Day . able to provide appropriate transportation to clinics and to medical, chapter, and VOLUNTEER quality-of-life activities . Support services PROGRAMS via grant funding as available . ALS PROOF y Developing a professionally-directed y Initiating and cultivating relationships program that connects community with organizations qualified to provide volunteers to individual families living appropriate home modifications, enabling with ALS . diagnosed persons to more safely access y Collaborating with existing community and navigate their home environment . organizations to increase awareness Support services via grant funding as of unmet volunteer needs of families available . experiencing a journey with ALS . YOUTH PROGRAMMING y Recruiting and training appropriate y Recognizing unique needs of ALS volunteers to provide direct, non-medical families that consist of children and support for families .

2021 ALS Association Mission Toolkit 2-9 4/15/21 FOR INTERNAL USE ONLY How We ALS PROOF Support Clinical Care SUPPORTING CLINICAL CARE person living with ALS and their education, care services, and emotional loved ones face a variety of support . challenges along the journey . As A y The ALS Association’s Clinical Programs the disease progresses and new symptoms currently consists of 73 Certified and difficulties arise, the number of people Treatment Centers of Excellence, 21 involved in providing care increases as Recognized Treatment Centers, and 94 well . Clear communication among the care Affiliated Clinics . team and coordination of care and services becomes vital . The Association’s Certified y The Association (National Office and Treatment Centers of Excellence (CTCE) and Chapters) provided $4,885,387 in grants Recognized Treatment Centers (RTC) provide to our Certified Centers and Recognized compassionate care in a supportive, family- Treatment Centers ($4,525,981 to CTCEs oriented atmosphere . Centers that achieve and $359,406 to RTCs) in 2020 . either of these designations meet program y 9,296 people were served through our requirements and follow recommended CTCEs and RTCs . best practices as outlined in the American Academy of Neurology Practice Parameters and collaborate 4/15/21 FOR INTERNAL USE ONLY with their local Association chapter to offer care and support to people living with ALS and their families . People withALS PROOF ALS can maintain independence longer and experience an improved quality of life when provided with options for symptom management, assistive technology, adaptive equipment,

2021 ALS Association Mission Toolkit 2-11 Our Care Network

THE ALS ASSOCIATION CERTIFIED TREATMENT CENTERS OF EXCELLENCE The ALS Association’s nationwide network of Certified Treatment Centers of Excellencesm provides evidence-based, multidisciplinary ALS care and services in a supportive atmosphere with an emphasis on hope and quality of life . To become certified as a Center of Excellence, an ALS clinic must meet clinical care and treatment standards, and all program requirements and processes to participate in ALS- related research, and successfully complete a comprehensive site review . CRITERIA AND REQUIREMENTS y Active relationship with, and support from, the local ALS Association chapter y Organized ALS clinic monthly or more frequently as needed y Clinic medical director, well-qualified in the field of neuromuscular neurology with a commitment to ALS y Existing multidisciplinary or interdisciplinary ALS clinic, which should have been established for a minimum of one year with a substantial track record of institutional support before the certification application process begins y Availability of neurological diagnostic tools and other necessary medical services, including gastroenterology or interventional radiology and pulmonology y Established caseload of people living with ALS (recommended minimum average of 50) and a pattern of new people with ALS joining the clinic y Collaboration with other ALS Association 4/15/21 TreatmentFOR INTERNAL Centers, for USE example ONLY in academic pursuits, educational pursuits, or IRB-related projects y Full multidisciplinary team, which includes licensed and certified professionals present in clinic on ALS clinic days, including but not limited to:

– ALS/NeuromuscularALS PROOF Neurologist – Nursing professional – ALS Association chapter liaison – Social Worker – MSW preferred – Speech Language Pathologist – Registered Dietitian – Occupational Therapist – Physical Therapist

2-12 2021 ALS Association Mission Toolkit – Pulmonologist or Respiratory Therapist (RT) * „ Available in the medical center and able to see the person(s) living with ALS or family during the clinic visit for unanticipated needs . Professional is not required to be physically present the entire clinic . „ If absent, another team member must be capable of performing required testing and assigned to conduct the pulmonary assessment and pulmonary function tests (PFTs) . y When known respiratory needs exist, the pulmonologist or RT must be present during the clinic to see the person(s) with ALS with the identified needs . y Mental Health Professional* – Ph .D . in psychology or psychiatrist available in the medical center and able to see the person(s) with ALS or family during clinic for unanticipated psychological or psychiatric needs . Doesn’t need to be present for the entire clinic . – If absent, another team member, such as a master’s prepared nurse or social worker, must be capable of performing required consultations, evaluations, recommendations, and referrals . – If known psychological or psychiatric needs exist, there must be a timely plan for a psychologist or psychiatrist to meet with the person(s) or family to address issues . y Active involvement in ALS-specific research (IRB-approved), to include at least one of the following: 4/15/21 FOR INTERNAL USE ONLY – Clinical Trials (Interventional) – Clinical Studies (Observational, Biomarker Studies, Natural History) – Genetic Screening, Sequencing, or Counseling Research – Basic Research/Drug Discovery Research ALS PROOF – Telemedicine/Telehealth Research – Assistive Technology/Device Development Research – Other ALS Specific, IRB Approved Research THE ALS ASSOCIATION RECOGNIZED TREATMENT CENTERS The ALS Association Recognized Treatment Centers have the same high- quality approach to multidisciplinary care as The ALS Association Certified Treatment Centers of Excellence and provide services through a multidisciplinary care team . These centers, however, do not directly participate in ALS research

2021 ALS Association Mission Toolkit 2-13 CERTIFIED CENTERS Certified Treatment Centers of Excellence (CTCE) and Recognized Treatment Centers (RTC)

Requirements for the CTCE or RTC CTCE RTC Active relationship with, and support from, the local ALS Association chapter ✓ ✓ Organized ALS clinic monthly or more frequently as needed ✓ ✓ Clinic medical director, well-qualified in the field of neuromuscular neurology with a commitment to ALS ✓ ✓

Existing multi/interdisciplinary ALS clinic established for a minimum of one year, with a substantial track record of institutional support ✓ ✓

Availability of neurological diagnostic tools and other necessary medical services (to include Gastroenterology and/or Interventional Radiology and Pulmonology) ✓ ✓

Established caseload of people living with ALS (recommended minimum average of 50) and a pattern of new people with ALS joining the clinic ✓ ✓

Collaboration with other ALS Association Treatment Centers (e .g . in academic pursuits, educational pursuits, IRB-related projects) ✓ ✓

1 . Full multidisciplinary team (licensed and certified professionals present in clinic on ALS clinic days, including but not limited to): – ALS/Neuromuscular Neurologist ✓ ✓ – Nursing professional ✓ ✓ – ALS Association chapter liaison ✓ ✓ – Social Worker – MSW preferred 4/15/21 FOR INTERNAL USE ONLY ✓ ✓ – Speech-Language Pathologist ✓ ✓ – Registered Dietitian ✓ ✓ – Occupational Therapist ✓ ✓ – Physical Therapist ✓ ✓ ALS PROOF Pulmonologist or Respiratory Therapist (RT) (at clinic or on as-needed basis) ✓ ✓ – Mental Health Professional (at clinic or as a referral) ✓ ✓ Active involvement in ALS-specific research (IRB-approved), to include at least one of the following: y Clinical Trials (Interventional) y Clinical Studies (Observational, Biomarker Studies, Natural History) y Genetic Screening, Sequencing, or Counseling Research ✓ y Basic Research/Drug Discovery Research y Telemedicine/Telehealth Research y Assistive Technology/Device Development Research y Other ALS Specific, IRB Approved Research

2-14 2021 ALS Association Mission Toolkit WHERETHE ALSWE ASSOCIATION SUPPORT CARE Team Members (as of January 2021) CLINICAL PARTNER NETWORK (as of January 2021)

THE ALS ASSOCIATION CERTIFIED TREATMENT CENTERS OF EXCELLENCESM – 73

THE ALS ASSOCIATION RECOGNIZED TREATMENT CENTERS – 21 OTHER ALS ASSOCIATION AFFILIATED CLINICS – 94

THE ALS ASSOCIATION THE ALS OTHER 73 CERTIFIED TREATMENT 214/15/21ASSOCIATION FOR INTERNAL USE94 ONLYALS ASSOCIATION CENTERS OF RECOGNIZED AFFILIATED CLINICS SM EXCELLENCE TREATMENT It is important to note that other The ALS Association’s nation-wide CENTERS models of care are necessary to meet network of Certified Treatment The ALS Association Recognized the needs of all people living with Centers of ExcellenceSM provides Treatment Centers have the same ALS. These include, but are not evidence-based, multidisciplinaryALS PROOF ALS high-quality approach to limited to, neurology group practices care and services in a supportive multidisciplinary care as The ALS and solo practitioners across the atmosphere with an emphasis on Association Certified Treatment country. The ALS Association chapters hope and quality of life. Centers of ExcellenceSM; however, they may provide educational and other do not offer onsite ALS research. support to these practitioners and To become certified as a Center of their patients in their local Excellence, each clinic must: community. However, these models of • Adhere to The ALS Association’s care are not an official part of The ALS clinical care and treatment Association Certified Center Program. standards, based on AAN Practice Parameters • Participate in ALS-related research • Successfully complete a comprehensive site review

The ALS Association Core Values: COMPASSION. INTEGRITY. URGENCY ALS.org

01/12/2021 2021 ALS Association Mission Toolkit 2-15 Our Care Network

CERTIFIED TREATMENT CENTERS OF EXCELLENCE To find a Certified Treatment Center in your area, visit http://www.als.org/community/centers-clinics/

Clinic Name ST Clinic Name ST The ALS Care Clinic at Crestwood Medical M Health Fairview ALS Clinic MN AL Center Minneapolis VA Health Care System MN Arizona Mayo Clinic AZ University of Missouri Healthcare ALS MO Barrow AZ Treatment Center Phoenix Neurological AZ SLUCare ALS Clinic MO Cedars-Sinai CA Atrium Health ALS Center NC Forbes Norris CA Duke University ALS Center NC Keck Medicine of USC ALS Clinic CA Wake Forest University NC University of California — Davis CA University of Nebraska NE University of California — Irvine CA Dartmouth–Hitchcock Medical ALS Center NH University of California — San Diego CA University of New Mexico NM University of California — San Francisco CA Rutgers University – Robert Wood Johnson NJ Loma Linda University CA Hospital for Special Surgery NY Hospital for Special Care CT Mount Sinai NY VA Connecticut CT Stony Brook NY George Washington University Hospital DC ALS Association Program at Columbia University NY Jacksonville Mayo Clinic FL State University of New York Upstate NY Phil Smith Neuroscience Institute at Holy Cross FL OhioHealth ALS Clinic OH Hospital ALS Clinic Cleveland Clinic OH University of Miami FL Louis Stokes – Cleveland VA Clinic OH University of South Florida FL Portland – VA Clinic OR Universtiy of Florida Jacksonville ALS Clinic FL Providence OR Augusta University ALS Clinic GA 4/15/21 FOROregon INTERNAL Health & Science USE University ONLY OR Emory ALS Center GA ALS Center at Allegheny Health Network PA University of Chicago IL Penn State University– Hershey PA University of Illinois IL Jefferson Weinberg ALS Center PA Indiana University ALS Clinic at IU Health IN Medical University of South Carolina (MUSC) SC University of Kansas KS Baylor University TX Universtiy of Kentucky KY ALS PROOF Houston Methodist TX Ochsner ALS Clinic LA University of Texas – San Antonio TX University of Maryland MD University of Utah UT Curt and Shonda Schilling ALS Clinic at Lahey MA Richard R . Dart ALS Clinic (University of Virginia) VA Clinic University of Vermont VT Henry Ford MI ALS Center at the Swedish Neuroscience WA Mercy Health MI Institute University of Michigan MI Puget Sound – VA Clinic WA Spectrum Health ALS Multidisciplinary Clinic MI Virginia Mason WA The ALS Center of Excellence at Hennepin MN Froedtert Hospital ALS Center WI County Medical Center Clement Zablocki Milwaukee VA Medical WI Mayo Clinic Rochester ALS Clinic MN Center

2-16 2021 ALS Association Mission Toolkit RECOGNIZED TREATMENT CENTERS

Clinic Name ST Clinic Name ST TidalHealth Peninsula Regional Medical Alabama Neurology Associates AL MD Center Kaiser Permanente Los Angeles Medical CA Center Essentia Health Duluth ALS Clinic MN Kaiser San Rafael ALS Multidisciplinary CA VA St. Louis Health Care System ALS Clinic MO Clinic Hackensack Meridian ALS Clinic at Jersey Kaiser San Francisco ALS Multidisciplinary NJ CA Shore University Medical Center Clinic Lehigh Valley ALS Comprehensive Care Kaiser Permanente South Bay PA CA Center Multidisciplinary ALS Clinic Geisinger Medical Center ALS Clinic PA San Francisco VA CA The Louise Wilcox ALS Center RI Colorado Neurological Institute CO Greenville Health System SC Iowa City VA Health Care System IA Avera Brain and Spine Institute SD University of Louisville Physician’s ALS KY Clinic at Frazier Rehab The ALS Clinic of the MidSouth TN Louisiana State University ALS Clinic LA/MS Emory Bellard ALS Clinic TX

The ALS Association’s Centers have full multidisciplinary teams Nursing of ALS specialists at the clinic who Professional will work collaboratively with the 4/15/21 FORMental INTERNAL Health USE ONLY ALS Professional Neurologist patient and their family . The ALS Association Centers are designed ALS to provide a regular, thorough, Respiratory Association and interdisciplinary evaluation; Therapist Chapter ALS PROOF Liaison answers to questions; and potential Person with solutions to problems . Typically, a ALS clinic visit involves a full morning or Physical Social afternoon and occurs every three Therapist Worker months . Speech Occupational Language Therapist Pathologist Registered Dietitian

2021 ALS Association Mission Toolkit 2-17 Our Care Network

SUPPORT THROUGH identifying appropriate methods of sharing . EDUCATION y Supporting the development and An ALS diagnosis can immediately leave delivery of quality learning resources people feeling distraught and struggling to and opportunities for multiple age cope . Our educational priorities focus on groups (including children), for various providing critical information to people living educational settings, respecting reading with ALS, their families and caregivers, and abilities, and in at least two languages . medical professionals — who may or may not, y Developing educational resources that are have prior experience with ALS . relevant . y y Providing educational programs and Creating resources that can be materials in a myriad of strategic forms, repurposed locally, nationally, and including face-to-face, workshops, internationally . symposiums, print, digital, and video . y Informing caregivers about options to y Making sure content is accessible care for their loved ones, especially as the regardless of where a person lives and person living with ALS loses the ability to their access to available technology . do so themselves . y y Involving our intended audiences during Providing information on disease the needs assessments, development and progression, symptoms and their design of materials and programs, and management, and considerations for decision-making that can help prepare individuals and families for addressing situations during the course of the disease . 4/15/21 FOR INTERNAL USE ONLY y Educating medical professionals about ALS treatment best practices for those who have limited experience or who have never encountered the disease before . ALS PROOF y Identifying opportunities to collaborate with other professionals and like-minded organizations to reduce redundancy, increase efficiency, and build stronger unity including assessment and evaluation of programs and materials against a given set of key indicators to determine their effectiveness as part of ongoing quality improvement activities .

2-18 2021 ALS Association Mission Toolkit 4/15/21 FOR INTERNAL USE ONLY

ALS PROOF Impact & Resources CARE SERVICES IMPACT People living with ALS come first in everything we do. The ALS Association is dedicated to providing people with ALS and their families and friends with the critical information, support, and resources necessary to live a full life and better meet daily challenges. 21,545 9,296 people living with ALS served through people registered with our Chapter the chapter network in the past year network & served through our Certified Treatment Centers of Excellence and Recognized Treatment Centers 12 CEU approved educational training sessions for healthcare providers 2,295 veterans received assistance through our nationwide chapter network $470,000 in higher educational scholarships was awarded to 94 students through the 12 Jane Calmes Scholarship Fund videos addressing the role of the multidisciplinary team in respiratory care throughout the course of the 2,397 disease times our 12 monthly educational webinars were viewed live or on- $4,885,387 demand 4/15/21 FOR INTERNALin grants providedUSE ONLY through our Certified Center Program 505,456 unique page views on the Care Services 25,831 attendees at support groups offered webpages onALS www.als.org PROOF through our vast chapter network across the U .S . 42,696 people viewed, downloaded, or 190+ ordered our educational materials relationships with clinical partners, including the Living with ALS and incorporating best practices as Families and ALS resource guides and established by the American Academy medical information packets of Neurology

2-20 2021 ALS Association Mission Toolkit FILLING THE GAP In Education, Knowledge, and Understanding THE NEED understand what to expect and how to cope . Furthermore, education materials for n ALS diagnosis can immediately medical professionals, who may not have leave people feeling helpless and prior experience with ALS, have been found struggling to cope . There is a great A to be extremely useful in promoting the need for new and innovative educational quality of care that people with ALS receive . materials to support this population . As ALS is a progressive disease, information is THE ALS ASSOCIATION’S ROLE needed at every stage of the disease to help people with ALS, families, and their caregivers The ALS Association is the leading organization in the ALS community, committed to finding treatments and a cure, as well as providing support and educational materials for people living with ALS . Our educational mission priorities include:

y Commitment to being the number one resource for people living with ALS by 4/15/21 FOR INTERNAL USE ONLY providing educational programs and materials in myriad forms, including print, digital, and video . y Informing caregivers about options to ALS PROOF care for their loved ones, especially as the person living with ALS loses the ability to do so themselves . y Educating medical professionals about ALS treatment best practices for those who have limited experience or who have never encountered the disease before . y Creating public awareness of ALS with the general population to build support for the ALS Association’s many initiatives .

2021 ALS Association Mission Toolkit 2-21 Our Care Network EDUCATION Living with ALS: General Scope THE ALS ASSOCIATION’S y Functioning When Mobility is Affected by LIVING WITH ALS RESOURCE ALS GUIDES y Adjusting to Swallowing Changes and Nutritional Management in ALS he ALS Association’s Living with ALS Resource Guides are a module series y Changes in Speech and Communication Tthat cover the progression of ALS and the Solutions many issues and considerations surrounding y Adapting to Changes in Breathing When the disease, from diagnosis to end-of-life . These You Have ALS guides cover rapidly expanding information y Approaching End of Life in ALS and research in the clinical management of It is anticipated that the guides will be ALS and were designed to inform and educate accessed online over 16,000 times over the people about ALS in a comprehensive and next year . The printed resources are available easily understood format . The resource through the Association’s order portal and guides address the most common challenges, throughout the ALS Association chapter concerns, and issues facing people living with network, resulting in thousands of printed ALS . They are extremely popular among the guides being delivered to those in need . ALS community due to the breadth of subjects covered in each . TRANSLATION OF RESOURCE 4/15/21 FOR INTERNAL USE ONLY The guides cover the following topic areas: GUIDES y What is ALS? An Introductory Resource The Hispanic, Spanish-speaking population Guide for Living with ALS in the United States reached 59 million people nationwide in 2017 and is continuing y After the ALS Diagnosis: Coping with the ALS PROOF to grow annually . Based on known “New Normal” prevalence rates, there are an estimated y Changes in Thinking and Behavior in ALS 3,400 Spanish-speaking people with ALS, y Living with ALS: Planning and Making although, due to underreporting, this Decisions number is likely higher . The ALS Association y Understanding Insurance and Benefits recently translated all Living with ALS When You Have ALS Resource Guides into Spanish to ensure that this population can access information y Managing Symptoms of ALS about ALS .

2-22 2021 ALS Association Mission Toolkit EDUCATION Living with ALS: Medical Resources COLLECTION OF MEDICAL The Medical RECORDS: MEDICAL Information Packet has been developed INFORMATION PACKET to serve as a tool When providing emergency care, health to inform medical professionals like paramedics and emergency providers caring for room staff will have many questions about people with ALS, a person’s medical condition . In these as well as provide stressful situations, it is often helpful to have insurance and family information, in a an organized set of information, which can centralized location . The sheets may be used help inform medical providers about specific as a packet or as individual pages, based on issues and considerations . one’s preference and need .

IN CASE OF EMERGENCY: KEY MEDICAL INFORMATION CARD

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ALS PROOF

The Key Medical Information Card has been developed to include those fundamentally important key considerations that are necessary to know in an emergency situation . It can be printed and folded to fit easily in a wallet, pocket, car glove compartment, etc .

2021 ALS Association Mission Toolkit 2-23 Our Care Network

EDUCATION ensure proper care for the person living with ALS . In conjunction with the caregiver LIVING WITH ALS: CAREGIVERS RESOURCES printed materials, The ALS Association has as How-To Video Series for Caregivers a programmatic priority to create a ‘How To’ Video Series . Often, caregivers lack the basic ALS not medical training and education to assist with only the growing medical needs of their loved affects ones . The How-To video series will focus those on the subject matters relevant to meeting diagnosed the daily needs of a person living with ALS, with the through presentations from subject matter disease, experts . The goal of the video series is to but also those who care for them . While the ensure that caregivers are confidently able to person living with ALS has to adjust their provide the best care . Videos will be based life to live with the disease, their family on information contained in the Living with members, or other caregivers, also have to ALS Resource Guides . make significant changes in their lives to

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ALS PROOF

2-24 2021 ALS Association Mission Toolkit ASSISTIVE TECHNOLOGY Assistive Technology in the ALS Space

or some, ALS will completely take away communication (AAC) devices) may be their their ability to communicate — verbally, only link to the outside world; their only Fwritten, bodily . Without this ability to way to stay in touch with family and friends . communicate, ALS can leave a person feeling This vastly improves their quality of life, as trapped in their own body . This situation is it provides them with the ability to gather particularly significant as people with ALS information for decision-making, and offers are often homebound due to the difficulties opportunities for a variety of interactions . of leaving their residence . THE ALS ASSOCIATION’S ROLE Technology continues to play a key role in enabling ALS patients to maintain a level Often, ALS patients have difficulty in using of independence . As a prime example, their devices and may have limited access assistive speech technologies allow patients to their clinic speech-language pathologist to communicate when they lose their between clinic visits . One of the challenges ability to speak . Computer access (via a The ALS Association faces is that, too often, communication device or adapted computer, Care Services chapter staff are neither trained also known as augmentative and alternative in how to use these devices nor able to 4/15/21 FOR INTERNALinstruct USE patients ONLY on how to best use them . As a result, the person may have the technology available, but is unable to fully utilize its capabilities . There is a critical ALS PROOF need for specialists who have the expertise and knowledge within the assistive technology spectrum . This includes both high-tech and low-tech assistive speech devices . A trained specialist is able to conduct more structured outreach to people living with ALS and family members regarding options for alternative forms of communication and can provide ongoing support .

2021 ALS Association Mission Toolkit 2-25 Our Care Network

One of The ALS Association’s goals is to staff, constituents, and people living with hire Technology Integration Specialists (TIS) ALS on utilizing technological opportunities to support Association chapters . As The that benefit the ALS community as a whole . Association builds its technology offerings, This specialist will also work in conjunction the implementation plan requires the hiring with Speech-Language Pathologist (SLP) in of TIS’s to support multiple chapter service ALS certified centers and clinics, vendors, areas . distributors, and other organizations providing communication devices throughout The TIS’s primary goal is to build up a their service area . This will be critical to chapter’s capacity to support technological ensuring the creation of a culture in which advancements, including augmentative appropriate resources and solutions are technology, for people living with ALS in identified, planned for, sustained, and used their geographic region . The TIS will assist in safely and effectively . developing strategies focused on engaging

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ALS PROOF

2-26 2021 ALS Association Mission Toolkit CHILDREN’S PROGRAMS AND RESOURCES oping with ALS can be especially restricted in how they may be able to spend difficult for the children of a parent their time, such as feeling responsible for Cor other close family member living helping to care for the diagnosed loved with ALS, as children do not have the same one, rather than socializing with friends . The intellect and emotional maturity as adults . need for them to provide direct caregiving And, in most cases, they are not given (sometimes in intimate situations, such as the same age-appropriate educational bathing a parent), combined with the need resources to help them understand . This is a for them to provide emotional support to widespread concern, as a significant number adults and siblings, can lead to feelings of people living with ALS report having of isolation from peers, depression, and a child who is directly impacted by the decreases in self-worth and self-esteem . disease . THE ALS ASSOCIATION’S ROLE Children often don’t understand what is happening to a loved one with ALS, leaving The ALS Association understands these them feeling scared of the unknown and young caregivers receive little of the

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ALS PROOF

2021 ALS Association Mission Toolkit 2-27 Our Care Network attention given to their adult counterparts, FAMILIES AND ALS, CHILDREN’S leaving them a fragmented and relatively AGE-BASED, AND AT-SCHOOL unknown caregiving population . In many PROFESSIONAL RESOURCE instances, the isolation experienced by young caregivers restricts them from reaching out GUIDES for assistance . With no known cure and minimal known treatments, ALS can be a difficult subject The ALS Association continues to develop to explain to children . These resource resources and programs that support efforts guides are the result of many years of to educate and assist children who have a clinical social work practice and formal loved one with ALS . Children handle trying research with families, children, and youths times differently than adults and The ALS affected by neurological illness . While Association aims to ensure that they receive much attention is given to the person living age and developmentally appropriate with ALS and their adult family member/ services .

CAMPS SPECIFICALLY DESIGNED FOR CHILDREN AFFECTED BY ALS Children impacted by ALS often have trouble connecting with their peers who have not experienced similar circumstances . The ALS Association collaborates with different camp programs throughout 4/15/21 the FOR INTERNAL USE ONLY US that provide specialized programs where children can interact and connect with other children impacted by ALS while participating in organizedALS PROOF group encounters . Youths attending such camp programs all have something in common (the significant impact of ALS on their lives) . They can build new friendships and establish contacts, participate in sharing sessions, feel less alone in their challenges, and have an ongoing communication plan .

2-28 2021 ALS Association Mission Toolkit caregivers, children are often voiceless, Older youth/young adult guide, ages 19-25 . despite experiencing much of the same This book takes a more serious tone, again shock, sadness, and grief as their adult following several young adults as they counterparts . manage their own lives with the care needed for their loved one with ALS, school and Families and ALS — This guide was created relationships . It is minimally illustrated and primarily for families living with ALS, again uses actual stories and quotes . including parents, grandparents, siblings, and other family members . The goal of At School: A Guide to Supporting Students the Families and ALS resource guide is to Who Have Been Affected by ALS — These facilitate discussions surrounding ALS, so professionals recognize the limited family members can begin answering many information and the need to receive of the questions that will be asked . guidance in order to lessen the potential social isolation felt by kids in families CHILDREN’S AGE-BASED with ALS — particularly in terms of how GUIDES these issues affect school performance Real Kids Talk About ALS: Feeling Normal, and attendance . This guide was created Sad, and Different - Graphic novel for youth, ages 7-12 . Delivered in a graphic novel format, this book uses direct quotes from youth in the ALS research projects . These quotes4/15/21 help FOR INTERNAL USE ONLY situate the learning style and experiences of children in ALS .

The ALS Experience: It’s Different and Hard — Choose your own adventure guide, middle ALS PROOF school youth, ages 13-18 . This book follows the stories of three youth, providing options for how they talked to people, who they talked to, and what was the outcome . The book allows other youth to choose who they might want to talk about ALS and their experiences . A companion online version has been developed .

School, Friends, Work, and ALS: A Young Adult Guide to Balancing Life with ALS —

2021 ALS Association Mission Toolkit 2-29 Our Care Network to address the needs of school staff and their lack of skill . In a study of young personnel working with children impacted caregivers for ALS, 68 percent had no by ALS . training or education in providing care, despite having to handle an average of ASSISTING WITH ACTIVITIES 11 tasks, including complicated assistive OF DAILY LIVING: DURABLE devices . These children expressed fear of MEDICAL EQUIPMENT TRAINING harming their family member with ALS, yet had no one to talk with, underscoring their Coping with ALS can be difficult for the anxiety and concern in their lack of skill . many children of a family member living with ALS, given differences in emotional THE ALS ASSOCIATION’S ROLE maturity and lack of age-appropriate educational and supportive resources . This In order to assist with the education and is a widespread concern, as a significant training of youths in caregiving including the number of people living with ALS report correct use of durable medical equipment, having a child who is impacted by the the ALS Association will create a specific disease . Moreover, children are tasked program in conjunction with Dr . Melinda with the physical care of a person living S . Kavanaugh, PhD, from the University of with ALS, yet had little to no training or Wisconsin, who has conducted significant support, leading to anxiety and concern in research on young caregivers and children who are impacted by a loved one’s ALS diagnosis . This project will result in the creation of the manuals that will provide guidance, skill development, and support 4/15/21 FOR INTERNAL USE ONLY for children and youth caregivers who provide care to their loved ones living with ALS . This project specifically addresses our mission priority to provide persons ALS PROOF with ALS and their family caregivers access to high quality consistent and compassionate support services . By working across Association chapters, this project strengthens the ability of the chapters to provide targeted, evidence-based, and rigorously tested caregiver educational programs, which will be made available to the ALS community nationwide .

2-30 2021 ALS Association Mission Toolkit THE JANE CALMES ALS SCHOLARSHIP FUND Helping People Affected by ALS Continue Their Education

he ALS Association established the Jane Calmes ALS Scholarship fund to provide support for students affected by the financial hardship of living with ALS . Mark Calmes, vice chair Tof the ALS Association National Board of Trustees developed the fund in honor of his wife Jane, who lived with ALS for eight years until her passing in August 2017 . This fund has allowed students to study a variety of majors, including medicine, biology, business and social services . 45 SCHOLARSHIPS WERE AWARDED IN 2019; 94 SCHOLARSHIPS WERE AWARDED IN 2020 With the help from The Jane Calmes ALS Scholarship I will be able to finish my bachelor’s degree debt free and for that I am forever grateful.

Chandler Brestel, studying healthcare management, University of Arkansas

Relationship to ALS Scholarship Recipients by College Type Scholarship Recipients by Year in School 2% Junior/Community College 35% 81 4/15/21 FOR INTERNAL USE32% ONLY 22%

98% 4-Year College/ 11% 8 ALS PROOF Grad School 2 2 1 Undergraduate Undergraduate Undergraduate Undergraduate Parent Grandparent Self Uncle Sibling Freshman Sophomore Junior Senior

In addition to the physical and emotional toll faced by people impacted by ALS, the financial strain of the disease can devastate a family’s ability to plan for future expenses . Families impacted by ALS often do not have the financial means to support the pursuit of college degrees or vocational certificates .

To learn more about the scholarship please visit www.als.org/get-involved/jane-calmes-als-scholarship-fund.

The Jane Calmes ALS Scholarship Fund is administered through Scholarship America.

2021 ALS Association Mission Toolkit 2-31 Our Care Network ALS ASSOCIATION CARE CONNECTION he Care Connection program is simple: it uses a private online calendar to bring together volunteers from the community — friends, neighbors and service groups — Tto provide help and support for people living with ALS and their families . For some families living with ALS, Care Connection is the only option to give loving caregivers a break from their day-to-day responsibilities .

Visit alsa.lotsahelpinghands.com/ to find tools that will help you organize a Care Connection Community for a family affected by ALS .

Step-by-step instructions assist a designated family Care Connection Coordinator with connecting the many family, friends, and community volunteers who want to help, with identified family needs . 4/15/21 FOR INTERNAL USE ONLY

ALS PROOF

2-32 2021 ALS Association Mission Toolkit CARE SERVICES STAFF KIM MAGINNIS, LORI BANKER- SENIOR VICE HORNER, PRESIDENT, CARE DIRECTOR, SERVICES CLINICAL Kimberly Maginnis brings PROGRAMS experience, energy, Lori has been working with enthusiasm and an appreciation for the ALS Community for over 23 years . She respectful relationships to her role as Senior began with the Southeast Wisconsin Chapter Vice President, Care Services, at The ALS in 1997 and was instrumental in the growth Association . She has worked in the healthcare of Care Services as the Chapter expanded to industry for more than three decades, and serving the entire state . The development of her resume includes positions at the Veterans partnerships with ALS multidisciplinary clinics Administration and Harvard Community was key to that growth ensuring people Health Plan in Boston, Massachusetts . living with ALS had access to the highest Prior to joining The ALS Association, Maginnis standard of care in the state . She served on worked at Inova Health System in Falls several National ALS Association committees and task forces including the Care Services Church, VA for more than 20 years as4/15/21 senior FOR INTERNAL USE ONLY director of Corporate Health Services, Urgent Committee and the Clinic Certification/ Care Centers and Employee Occupational Recertification Sub-team . Lori joined the Health . Some of her key accomplishments in National ALS Association in 2019 where she this role include her developing successful, brings her experience to her role . In managing collaborative relationshipsALS PROOF with her colleagues the National Certified Center Program, she in other departments; achieving income works directly with both Chapters and ALS targets while managing risk and achieving multidisciplinary clinics to obtaining and strong operational controls; developing successfully maintaining designation as a strategic business plans that yielded defined Certified Treatment Center of Excellence financial and operational successes . or Recognized Treatment Center . She works closely with all mission areas including Maginnis holds a B .S . in Hospital and Health Research and Advocacy . Lori received her Services Administration, Ithaca College, BA in Psychology and Adult Education from Ithaca, New York and an M .B .A . in Business Alverno College . She has been a Licensed Administration/Finance from American Practical Nurse for over 30 years serving in University, Washington, D .C . many areas of health care .

2021 ALS Association Mission Toolkit 2-33 Our Care Network

CYNTHIA LESLIE RYAN, KNOCHE, RRT, MSPT, DIRECTOR, BBA, DIRECTOR, EDUCATION AND CHAPTER CARE PROFESSIONAL SERVICES DEVELOPMENT Cynthia has served PROGRAMS Association stakeholders since 2007 by Leslie joined the ALS Association in 2019 . In providing consultation, guidance, and support her position, she oversees the development to the Association’s nationwide network of and implementation of educational chapters with respect to the wide range of materials and programs for people living care and support programs they deliver . Her with ALS, the community, and healthcare clinical background as RRT, and management providers . She collaborates with partners experience in the healthcare business across the association to ensure relevant arena support successful assessment and and timely information is shared with the development of program services . Cynthia ALS community . Prior to joining the national holds credentials as a Registered Respiratory office, Leslie worked at The ALS Association Therapist with the National Board for Rocky Mountain Chapter as the Care Services Respiratory Care, is licensed as a RRT in Director for 15 years . She was instrumental Florida, and holds a Bachelor of Business in building valuable care services Administration degree from the University programming as the chapter expanded its of North Florida . Cynthia works closely with reach to cover 3 states and partnered with our advocacy mission team to support multidisciplinary clinics throughout the 4/15/21 FOR INTERNAL USE ONLY meaningful legislative and regulatory benefits region . Leslie developed relationships with for those living with ALS . In addition, Cynthia industry partners and helped to establish leads professional outreach efforts as an the high level of excellence the chapter is invited guest speaker, presenting to allied known for in the community . Serving on practice healthcare ALSand servicePROOF organizations the national Care Services Committee and on a state and nationwide level . various task forces over the years gave her the opportunity to share her expertise throughout the association . She received her Bachelor of Science in Health Studies and a Master of Science in Physical Therapy from Boston University, and practiced clinical care in a variety of settings before transitioning to the nonprofit sector .

2-34 2021 ALS Association Mission Toolkit MIRIAM BRODKIN, Her specialty areas are communication, MANAGER, computer access, electronic aids for daily living and home modifications . She is a RESOURCE strong advocate for patient rights with CONNECTION elected officials, insurance companies, and Miriam joined the ALS other healthcare providers . Association in 1997 after Alisa is 2000 graduate of the California her mother passed away from ALS . It was State University Northridge with a Graduate important to her to help others with going Certificate in Assistive Technology . She has through the same experience . In her role, she a Bachelor of Science Degree in Hospitality communicates with people living with ALS, Management and a Bachelor of Arts Degree their families, healthcare professionals, and in History from East Stroudsburg University . the general public through email and phone She is a Certified Assistive Technology inquiries . Miriam helps connect people to Professional (ATP) through the Rehabilitation appropriate ALS Association chapter and Engineering and Assistive Technology Society community services and programs to help of North America (RESNA) and a Certified them address important-day-to-day decisions Aging in Place Specialist (CAPS) through the and to find solutions and options for managing National Association of Home Builders . the symptoms and journey of ALS . Miriam also helps people from other countries identify MARKEYA MARTIN resources in their own communities 4/15/21. FORCOORDINATOR, INTERNAL USE ONLY ALISA BROWNLEE, MISSION AREAS ATP, CLIPP, CAPS, Markeya joined the ALS WSP, CLINICAL Association in 2018 . In ALS PROOF this role she assists with MANAGER, coordinating and planning ASSISTIVE for the three mission areas; Care Services, TECHNOLOGY Advocacy/Public Policy, and Research . She SERVICES also assists with organizing and overseeing Alisa is a 24 year staff member of the ALS major grants and programs provided . Prior to Association working for both the Greater joining the national office, Markeya worked at Philadelphia Chapter and the National LifeSpan Network in Columbia, Maryland as a Office . Alisa speaks, blogs, writes articles grant coordinator . She received her Bachelor and uses social media to increase awareness of Science in Community Health from Hofstra of ALS and the use of assistive technology . University .

2021 ALS Association Mission Toolkit 2-35

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ALS PROOF OUR Advocacy

“It brought tears to my eyes when I saw the news about the bill. It was an honor to participate in ALS Advocacy Day.” —Tabitha Wandell ALS Advocate and Caregiver, Tennessee 4/9/21 FOR INTERNAL USE ONLY Introduction ALS PROOF To ALS Advocacy INTRODUCTION he ALS Association empowers advocates to take action on issues important to people living with ALS . Our goal is to educate policymakers and the public and to drive smart Tdecisions related to research, treatments, and access to care that benefit people with ALS and their families .

We are the largest and most influential national advocacy organization in the United States focused solely on ALS . We are a nonpartisan organization committed to working across party lines to identify solutions for ALS . We empower and support people living with ALS to make a difference in the policy process to change laws, regulations, and policies .

Every day, policymakers at all levels of government make decisions that impact funding for ALS research and programs that serve people living with ALS . Our advocates educate and advocate with elected officials to make it an urgent priority to find cures and treatments and improve services for people living with ALS . The Association and advocates meet with members of Congress, call and write emails and letters to get their message across .

The ALS Association plans and directs strategic campaigns to achieve results: 4/9/21 FOR INTERNAL USE ONLY y develop and promote legislation; y develop and expand the number of congressional champions; y lobby key members of Congress to ALS PROOF improve services and increase research funding; y engage Association chapters in lobbying their congressional delegations; y mobilize a vibrant grassroots network; and y partner with patient advocacy, physician and health professional organizations on cross-cutting issues such as pre-existing conditions .

2021 ALS Association Mission Toolkit 3–1 Our Advocacy HISTORY OF ALS ADVOCACY ver the past two decades, The ALS Association has been at the forefront of public policy efforts to better the lives of people with ALS . The ALS Association is the only Onational nonprofit organization fighting ALS on every front through research, care services, public education, and public policy — giving help and hope to those affected by ALS .

We bring the ALS community together to speak with one voice and advocate on issues important to the community at the national level . This includes advocating before Congress, the White House, and with key federal agencies — including the National Institutes of Health (NIH), Department of Defense (DOD), the Centers for Disease Control and Prevention (CDC), the Centers for Medicare and Medicaid Services (CMS), the Food and Drug Administration (FDA), Department of Veterans Affairs (VA), and the Social Security Administration (SSA) .

Our chapters and advocates are essential to our success . The Association empowers a national grassroots network of approximately 40,000 advocates 4/9/21 FOR INTERNALwith theUSE information, ONLY tools, and expertise needed to speak out and play an active role in the fight for treatments and a cure . In 2020, ALS chapters and ALS PROOF advocates accomplished over 630 meetings with members of Congress . Many more meetings took place throughout the year in states and congressional districts . Throughout 2020, advocates mobilized through 20 action alerts that generated more than 85,000 messages to Congress .

3–2 2021 ALS Association Mission Toolkit TIMELINE OF EVENTS In 2001, we successfully lobbied Congress to waive a 24-month wait for Medicare for people with ALS on Social Security Disability Insurance . 2001 In 2003 we successfully prompted the Social Security Administration to publish new rules that made it easier 2003 for people living with ALS to qualify for Social Security Disability Insurance (SSDI) and added ALS to the list of In 2006, we supported passage of the Lifespan Respite conditions that automatically qualify for presumptive Care Act, which led to $15 million in funding per year disability payments under SSDI . for the kinds of respite care programs that are urgently 2006 needed by people with ALS .

In 2007 we spearheaded efforts to establish a more In 2008, we helped to implement historic regulations at focused, coordinated, and better-funded approach the Department of Veterans Affairs that designate ALS 2007 for studying ALS, which led to the creation of the ALS as a service-connected disease, ensuring that veterans 2008 Research Program (ALSRP) at the Department of Defense with ALS and their survivors have access to VA benefits, (DOD) as part of a $5 million appropriation in the FY2007 including health care and disability benefits . Defense Appropriations Bill . We also led the fight to establish the National ALS Registry and Biorepository, which secured federal funding to design, build, and implement the Registry and Biorepository at the Centers for Disease Control and Prevention (CDC), the In 2012 we helped enact the FDA Safety and Innovation single largest ALS research project ever created . Act (FDASIA), which strengthened the Fast Track and 2012 Accelerated Approval processes, required FDA to partner more closely with patient organizations representing those In addition, we partnered with FDA to convene the first- with rare diseases, and provided additional flexibility to ever ALS-specific public hearing and urged the FDA to FDA to approve new treatments quicker through the use partner with the ALS community to help expedite the 2013 of biomarkers and other surrogate endpoints . drug development and approval process and bring new treatments to patients as soon as possible . In 2016 we helped push Congress to pass the 21st Century Cures Act, which included provisions that will improve the 2016 process of developing therapies targeting rare diseases The ALS Association brought the ALS community together 4/9/21 FOR INTERNALand authorizedUSE $4 .ONLY8 billion over 10 years for the NIH . to create the first patient-focused guidance for ALS drug development ever submitted to the FDA . It has the potential 2017 to speed access, reduce costs, ensure more effective use of resources, and incentivize the pharma/biotech industry to enter the ALS market and develop new treatments for ALS . We also worked with Congress to increase funding for the DOD ALSRP from $7 .5 to $10 million, bringing the total The ALS Association led the charge that secured $10 funding for the ALSRP toALS nearly $80PROOF million since the million in funding for the DOD ALSRP and $10 million for program’s inception in 2007 . 2018 the CDC National ALS Registry and Biorepository . In addition, we played a key role in securing a bill to protect We also secured the first-ever Senate “Dear Colleague” access to customized wheelchairs and accessories that letter circulated for signature by senators and sent to caused the Medicare program to reverse its decision to add the Appropriations Committee supporting funding for these complex devices to the Competitive Bidding Program . the DOD ALSRP and the CDC National ALS Registry and Biorepository .

The ALS Association initiated and led the successful campaign to increase funding for the DOD ALSRP to $20 million — a $10 million increase — through direct lobbying, 2019 In 2020, we initiated and led the successful campaign to chapter advocacy, grassroots mobilization and engaging boost funding for the DOD ALSRP from $20 to $40 million . other ALS organizations . 2020 We also ended the SSDI 5-month waiting period for people The ALS Association spearheaded and co-led coalition living with ALS by spearheading successful efforts to pass efforts to protect access to noninvasive ventilators (NIV) and enact into law the bipartisan ALS Disability Insurance in collaboration with physicians and clinicians . This Access Act . included meetings with Medicare program officials and the introduction of the Safeguarding Medicare Access to In addition, we protected access to noninvasive ventilators Respiratory Therapy (SMART) Act of 2019 . (NIV) for Medicare beneficiaries with ALS by fighting the Medicare program’s decision to add NIV to the Competitive Bidding Program .

2021 ALS Association Mission Toolkit 3–3 4/9/21 FOR INTERNAL USE ONLY Advocacy ALS PROOF In Action ADVOCACY GUIDING PRINCIPLES s legislative, regulatory, and other and support services . policy proposals are considered, y Preserve and increase support for ALS Athey must be measured against the research. Funding and support must be mission and values of The ALS Association: provided for research in all phases to To discover treatments and a cure for ALS, discover new life-extending therapies and to serve, advocate for, and empower and technologies for people with ALS . people affected by ALS to live their lives to The ALS Association is dedicated to the fullest . ensuring laws, regulations, and incentives The ALS Association’s public policy priorities relating to the therapy development are grounded in our advocacy guiding process effectively and expeditiously principles which maintain that policy promote new interventions and, proposals must preserve and/or enhance ultimately, a cure for ALS . health care, benefits, and services for those y Fight for people living with ALS on who have it, provide coverage to those a wide range of issues. People with who do not, and lower costs and improve ALS should be protected against quality for all . This must be achieved at the discrimination in all settings, including 4/9/21 FOR INTERNAL USE ONLY federal and state level and in both public in the workplace, when purchasing and private settings . Research funding must health insurance, or receiving veterans’ be preserved and increased, people with benefits . The ALS Association monitors ALS must not be discriminated against, and legislation and regulation regarding resources and supportALS forPROOF caregivers must health care, disability rights, taxation, be increased . and more to ensure the ALS community is represented . PRINCIPLES y Increase resources and support for y Protect and expand access to health families and caregivers of people with care, benefits, and services. People ALS. Families and caregivers are the with ALS and their families must have lifeline for people with ALS and must health care that is accessible, affordable, be recognized and supported . The ALS adequate and understandable . The ALS Association believes federal, state, Association is committed to fighting for and private programs must do more people with ALS on every front to break for families and caregivers in terms of down barriers to health care, benefits, programs, supports, and financial aid .

2021 ALS Association Mission Toolkit 3–5 Our Advocacy HOW WE ADVOCATE he Association works closely with with lawmakers . Once someone enrolls as volunteer leaders, ALS advocates, an advocate through our action center, they Tresearchers, and partners on the federal can engage at key moments to contact their and state level to advocate for policies that senators and representatives on important are supportive of and responsive to the needs issues . of people living with ALS and their families . The Association values a collaborative Our network of over 40,000 grassroots approach to advocacy and belongs to several advocates is mobilized regularly through formal and informal coalitions . Coalitions action alerts to contact their elected officials allow us to leverage the combined expertise on ALS priorities . These volunteers also come of the broader patient advocacy community together for conferences and signature events to make sure the ALS perspective is accounted throughout the year to discuss and learn for when decisions are made . Lawmakers about these priorities and advocate directly have limited resources and time, and they rely on coalitions to provide policy solutions developed through consensus by diverse groups .

THE ROLE OF THE ASSOCIATION’S 4/9/21 FOR INTERNALCHAPTER USE ONLY NETWORK IN ADVOCACY Our chapter network plays a vital role in our public policy efforts . Chapters serve as a ALS PROOF resource for what people with ALS need and form the backbone of our long-term grassroots strategy .

While our grassroots advocacy grew as a result of the ALS Ice Bucket Challenge, it is through our chapters that we expand our grassroots

3–6 2021 ALS Association Mission Toolkit through their local connections and the the Association’s public policy priorities work that they do on the ground every day . and meet with lawmakers and their staff to This helps us continue to build our roster discuss our priorities . of grassroots advocates, who help amplify the ALS community’s priorities . Leaders at NATIONAL ALS ADVOCACY CONFERENCE the chapter level also serve as our grasstops Every summer, the Association holds its advocates — building the Association’s long- largest public conference providing advocates term relationships with members of Congress from across the country an opportunity to and their staff and bringing the local voice to come together to network, share their stories, Capitol Hill . and meet with members of Congress . People with ALS come with their family members and THE ALS ASSOCIATION chapter staff to learn about ALS public policy EMPLOYS SEVERAL TOOLS AS priorities and what Congress needs to do . The PART OF OUR GRASSROOTS conference also provides program sessions to ADVOCACY STRATEGY: update the community on the latest research and care services innovations . Almost 1,000 ACTION ALERTS AND CONTACTING CONGRESS people attended this event virtually in 2020, The Association maintains an action center including over 260 people with ALS – the on our website that allows us to reach out to most to ever participate . more than 40,000 advocates with updates 4/9/21 FOR INTERNAL USE ONLY on our public policy priorities and the latest OPPORTUNITIES FOR ADVOCACY AT developments from Capitol Hill, and to SIGNATURE EVENTS encourage them to contact their members In addition to the Association Fly-In and of Congress and participate in the legislative National ALS Advocacy Conference, The process . In a matterALS of minutes, PROOF our advocates Walk to Defeat ALS provides an excellent can send a personalized email to their opportunity where chapters expand their members of Congress . roster of advocates by engaging a large audience of people who already have an ANNUAL ASSOCIATION CHAPTER FLY-IN interest in fighting ALS . Advocacy tents Every year, The ALS Association hosts an or tables serve as gathering spaces where event where our chapter executives and chapters sign-up new advocates and advocate leaders get a chance to participate reengage returning advocates . Chapters also in a day of meetings and training at the invite members of Congress to participate height of the appropriations process . in The Walk, which gives the member an Chapter leaders engage in discussion with opportunity to address the audience and to national office staff and policy experts on interact directly with people with ALS and

2021 ALS Association Mission Toolkit 3–7 Our Advocacy their supporters and see how this disease registry for people living with ALS, the impacts their community . Registry connects patients directly with clinical trials, fuels research as a repository THE NATIONAL ALS REGISTRY of data for scientists, and empowers patients AND BIOREPOSITORY to make invaluable contributions to a future The ALS Association led the fight to establish freed from ALS . In January 2017, ATSDR the National ALS Registry and Biorepository launched the National ALS Biorepository as at the CDC by working with Congress to part of the Registry to enable researchers enact the ALS Registry Act in 2008 . The to request samples from both living and legislation provided the Agency for Toxic deceased (post-mortem) persons with ALS . Substances and Disease Registry (ATSDR), a ASSOCIATION branch of the CDC, with the authorization and guidance necessary to create a National COLLABORATION WITH ALS Registry . The Association partnered with FEDERAL AGENCIES the CDC to begin identifying cases of ALS on The ALS Association has played a pivotal a nationwide basis and collecting information role in expanding the CDC’s investigations urgently needed for ALS research – work that into ALS prevention and environmental risk continues to this day . factors, with the ultimate goal of translating The Registry and Biorepository are the single CDC’s early stage research into a more largest ALS research project ever undertaken . definitive list of causes and preventing future As the world’s largest population-based cases of ALS .

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ALS PROOF

3–8 2021 ALS Association Mission Toolkit 4/9/21 FOR INTERNAL USE ONLY

ALS PROOF Priorities, Impacts & Coalitions Our Advocacy ADVOCACY IMPACT With the support of over 40,000 ALS advocates, we secure resources to support treatment and research for the entire ALS community. Our nationwide network of 39 chapters and two service areas are essential to our efforts at the federal, state and local levels in advancing our mission to discover treatments and a cure for ALS, and to serve, advocate for, and empower people affected by ALS to live their lives to the fullest. Here are recent successes in ALS advocacy! Protected access to noninvasive Ended the SSDI 5-month waiting ventilators (NIV) for Medicare period for people living with ALS beneficiaries with ALS by fighting by spearheading successful efforts the Centers for Medicare and to pass and enact into law the Medicaid Services (CMS) decision bipartisan ALS Disability Insurance to add NIV to the competitive Access Act . bidding program . Boosted funding for the ALS Engaged the ALS community in a Research Program at the campaign to bring AMX0035 to $40 Department of Defense (DOD) people living with ALS as quickly from $20 to $40 million in 2020 as possible by collecting over MILLION to support the creation of more 50,000 signatures on a petition to clinical trials that will lead to the FDA and Amylyx . discovery of treatments and a Joined with other leading patient cure . organizations in the fight to preserve protections for pre- Ensured $10 million in continued 4/9/21 FOR INTERNAL USE ONLY existing conditions for people $10M funding for the National ALS with ALS under all insurance NATIONAL Registry and Biorepository plans, including Medicare, ALS to identify genetic and Medicaid, and private insurance . REGISTRY environmental factors for ALS, provide support to researchers to SupportedALS over PROOF 630 meetings with find treatments and a cure, and 630+ members of Congress and over promote access to clinical trials . MEETINGS 500 calls to Congress during the 2020 Virtual Advocacy Fly-In and Supported increases in funding 20 National ALS Virtual Advocacy $105M for the National Institutes of ACTION Conference . Empowered more Health, resulting in increased ALERTS than 40,000 ALS advocates funding for ALS research from through 20 action alerts — NIH $83 million in fiscal year 2018 to resulting in over 85,000 advocacy $105 million in fiscal year 2019 . messages to Congress .

3–10 2021 ALS Association Mission Toolkit HOW WE COLLABORATE The Value of Coalitions

hen organizations join forces action . As new issues emerge, coalition efforts and work together to impact enable The ALS Association to join with Wpolicy, they maximize access to others to examine complex and cross-cutting policymakers and increase the likelihood of issues that impact patients . advocacy success . Members of Congress and administration officials, who have limited COLLABORATIONS WITH resources and time, rely on coalitions to OTHER ALS ORGANIZATIONS provide policy solutions where there are The ALS Association works in collaboration shared concerns . with a number of organizations, including the Belonging to a coalition in no way limits Muscular Dystrophy Association, I AM ALS, the ability of the ALS Association to act Team Gleason, and Les Turner ALS Foundation . independently . It does amplify access to policymakers and the influence of The ALS MEMBERSHIP ORGANIZATIONS Association in representing the concerns of y National Health Council (NHC): NHC people living with ALS . It also ensures that is the only organization that brings the ALS Association is at the table when together all segments of the health 4/9/21 FOR INTERNAL USE ONLY consensus is developed and important community to provide a united voice for decisions are made . the more than 133 million people with chronic diseases and disabilities and The ALS Association only belongs to their family caregivers . Made up of more coalitions that are nonpolitical and ALS PROOF than 100 diverse national health-related committed to working with Republicans organizations and businesses, the NHC’s and Democrats in a nonpartisan fashion . core membership includes the nation’s Participation by member organizations in leading patient advocacy organizations, any coalition activity is always voluntary . which control its governance and Decisions about whether to take a position policy-making process . Other members on specific policy positions are made on include professional and membership a case-by-case basis — after thoughtful associations; nonprofit organizations with consideration of the pros and cons . an interest in health; and representatives Coalitions allow member organizations to from the pharmaceutical, generic drugs, benefit from combined expertise which, health insurance, device, biotechnology, when leveraged, enables timely analysis and and communications industries .

2021 ALS Association Mission Toolkit 3–11 Our Advocacy y Consortium for Citizens with Disabilities Affordable Care Act (ACA) and favors a (CCD): CCD is the largest coalition of bipartisan approach to reforming the national organizations working together healthcare system . to advocate for federal public policy y Alliance for a Stronger FDA: The Alliance that ensures the self-determination, for a Stronger FDA works to ensure annual independence, empowerment, integration appropriations that will adequately fund and inclusion of children and adults with the FDA’s essential missions . A science- disabilities in all aspects of society . based and effective FDA supports y Defense Health Research Consortium biomedical innovations as well as approves (DHRC): Comprised of more than two safe and effective drugs and medical dozen diverse organizations, DHRC devices for people living with ALS and focuses on continued Congressional all Americans . The Alliance unites a support for dozens of medical research broad and diverse spectrum of patient programs at the Department of Defense . groups, consumer advocates, biomedical These programs, funded annually in the researcher, health professionals and Defense Appropriations Act, make up the companies . “Congressionally Directed Medical Research Programs” (CDMRPs), which include the INFORMAL COALITIONS IN WHICH THE ALS vital ALS Research Program (ALSRP) . ASSOCIATION PARTICIPATES: y National Organization for Rare Disorders y Independence Through Enhancement of (NORD): A patient advocacy organization Medicare and Medicaid (ITEM) Coalition: dedicated to individuals with rare The ITEM Coalition is devoted to raising diseases and the organizations that 4/9/21 FOR awarenessINTERNAL and USE building ONLY support for policies serve them . NORD, along with its more that will enhance access to assistive than 260 patient organization members devices, technologies, and related services is committed to the identification, for people with disabilities and chronic treatment, and cure of rare disorders conditions . The coalition is consumer-led ALS PROOF through education, advocacy, research, and includes a diverse set of disability and patient services . organizations, aging organizations, other y Partnership to Protect Coverage: A consumer groups, voluntary health coalition of the nation’s leading patient associations, and non-profit provider advocacy organizations which have associations . The ALS Association is a collectively opposed health care reform member of ITEM’s steering committee . legislation that would negatively impact y Safeguarding Medicare Access to patients . The coalition led efforts on Respiratory Therapy (SMART) Coalition: the repeal/replacement efforts on the The coalition was formed to protect

3–12 2021 ALS Association Mission Toolkit access to non-invasive ventilators with in which every provider in America is support from respiratory therapists in the permitted to deliver and be adequately Medicare program . A majority of people compensated for providing safe, high living with ALS require access to ventilators quality care using connected care (e .g . at some point to maintain their quality telemedicine and telehealth) at their of life . Working with national associations discretion, regardless of care delivery of respiratory physicians and therapists, location or technological modality . the Association drafted and lobbied Members include leading companies for immediate passage of Safeguarding from across the health care and Medicare Access to Respiratory Therapy technology spectrum, representing (SMART) Act (H .R . 4945) . insurers, retail pharmacies, technology y Ad Hoc Group for Medical Research: Ad and telecommunications companies, Hoc Group for Medical Research is a and health care entrepreneurs . The coalition of patient and voluntary health Alliance is advised by a distinguished groups, medical and scientific societies, group of patient and provider academic and research organizations, organizations . and industry that support enhancing the federal investment in the biomedical, behavioral, and population-based research conducted and supported by the NIH . 4/9/21 FOR INTERNAL USE ONLY y Medicare Access for Patients Rx (MAPRx): MAPRx is a coalition of patient, family caregiver and health professional organizations committed to safeguarding the well-being ofALS patients PROOF with chronic diseases and disabilities who rely on Medicare Prescription Drug Coverage . MAPRx members represent every segment of Medicare beneficiaries and join together to advocate on their behalf and collaborate with national and state policymakers to ensure all beneficiaries have access to needed medications . y Alliance for Connected Care: Alliance for Connected Care was formed to create a statutory and regulatory environment

2021 ALS Association Mission Toolkit 3–13 Our Advocacy y Partnership to Improve Patient Care and supporting services . In pursuit of (PIPC): PIPC is at the forefront of applying that goal, NCART works with consumers, principles of patient-centeredness to clinicians, and physicians along with federal, the nation’s health care system – from state and private policy makers to establish the generation of comparative clinical and protect appropriate coverage, coding, effectiveness research at the Patient- supplier standards, and funding policies . Centered Outcomes Research Institute y Medical Expense Deduction (MedEx) (PCORI), to the translation of evidence Coalition: MedEX is an AARP-convened into patient care in a manner that achieves coalition of more than 60 organizations value to the patient . Having driven the that supports retention of the medical tax concepts of patient-centeredness and deduction that allows taxpayers to claim patient engagement in the conduct of an itemized deduction for high out-of- research, PIPC looks forward to bringing pocket medical expenses . the voices of patients and people with disabilities to the discussion of how to advance patient-centered principles throughout an evolving health care system . y National Coalition for Assistive & Rehab Technology (NCART): NCART is a national organization of suppliers and manufacturers of Complex Rehab 4/9/21 FOR INTERNAL USE ONLY Technology (CRT) products and services used by individuals with significant ALS PROOF disabilities and chronic medical conditions . NCART seeks to ensure these individuals have adequate access to CRT products

3–14 2021 ALS Association Mission Toolkit ADVOCACY STAFF KATHLEEN Substance Abuse and Mental Health Services SHEEHAN, VICE Administration . Kathleen also possesses expertise in public policy issues related to the PRESIDENT, development and approval of pharmaceuticals PUBLIC POLICY at the Food and Drug Administration . Kathleen brings over 20 years of government ABRAM BIELIAUSKAS, relations experience on health care issues ASSOCIATE to The ALS Association . As an advocate and DIRECTOR, policy expert, she has extensive knowledge of GOVERNMENT patient concerns as well as the complexities of Medicare, Medicaid, private insurance, AFFAIRS and pharmaceuticals . Throughout her career, Abram joined The ALS Kathleen has championed access to care for Association in 2017 and patients, as well as increased appropriation works with Kathleen Sheehan to lobby Capitol for health science research at NIH and other Hill on the Association’s various public policy federal agencies . Kathleen is a respected priorities . Mr . Bieliauskas previously served as health care policy strategist known for her Government Affairs and Advocacy Specialist at 4/9/21 FOR INTERNAL USE ONLY bipartisan approach and ability to create the Pancreatic Cancer Action Network, where effective relationships across the political he managed logistics for that organization’s spectrum . Hallmarks of her career include Advocacy Day conference, oversaw a national vibrant engagement with chapters and grassroots network of advocates strategically volunteer leaders, asALS well PROOFas the development located in key congressional districts, and of effective collaborations with other national advocated through influencing Congress to organizations . Kathleen has held leadership support increased cancer research funding . positions with the American College of Abram also brings more than a year of Capitol Cardiology, the Visiting Nurse Associations of Hill experience, having worked in both the America, the Maryland Homecare Association, US House and Senate . He is leveraging his as well as national organizations focusing appropriations experience to support CDC on substance abuse and mental health . and ALSRP . Originally from Cincinnati, Ohio, Her leadership has resulted in numerous Abram moved to Washington, DC, after legislative victories, including securing a key graduating cum laude with a bachelor’s provision in the Affordable Care Act and degree in Political Science Pre-Law from Ohio crafting key sections of reauthorization for the University .

2021 ALS Association Mission Toolkit 3–15 Our Advocacy

ASHLEY SMITH, ADAM BAKER, ASSOCIATE MANAGER, DIRECTOR, PUBLIC POLICY GRASSROOTS INITIATIVES ADVOCACY Adam serves as the liaison Ashley designs and directs to the Center for Disease nonpartisan grassroots activities to advance Control (CDC) and oversees all aspects of The ALS Association’s priorities and mission . The ALS Association’s contract relating to Prior to joining The ALS Association, Ashley the promotion of the National ALS Registry, led the grassroots efforts of the National as well as other issues concerning disease League of Cities (NLC) by crafting advocacy surveillance . He has served in this role since strategies that supported NLC’s federal April 2018 . Prior to his time at the Association, policy agenda and empowered city leaders Adam worked for Senator Jon Tester and the to engage directly with federal elected Democratic Senatorial Campaign Committee . officials . At M+R, she facilitated state- and He earned his BA in English and Philosophy federal-level advocacy activities for clients from Kenyon College . including the American Lung Association, the MacArthur Foundation, and the American Public Health Association . Ashley earned her BA in Political Science and American Studies from the University of Kansas and an MA in Government, with a Political Communications 4/9/21 FOR INTERNAL USE ONLY Concentration, from Johns Hopkins University .

ALS PROOF

3–16 2021 ALS Association Mission Toolkit 4/9/21 FOR INTERNAL USE ONLY

ALS PROOF Appendix Appendix ABBREVIATED GLOSSARY OF SCIENTIFIC TERMS A Bulbar Muscles: The muscles that control speech, chewing, and swallowing . Agonist: A drug that increases neurotransmitter activity by directly stimulating the nerve cell receptors . C Central Nervous System (CNS): The brain and ALS Functional Rating Scale – Revised spinal cord combined . (ALSFRS-R): A survey of questions that assesses the impact of als on activities of daily living . It is Cerebrospinal Fluid (CSF): A clear fluid that often used as a primary outcome measure of als covers and protects the brain and spinal cord . clinical trials . Chromosome: A visible carrier of the genetic Amino Acid: One of the 20 building blocks of information . protein . Corticospinal Tract: The bundle of nerves that Antibody: A defense protein that binds to foreign reach from the motor area of the brain (see molecules to allow their elimination . cortex) to the spinal cord, connecting to the nerves that go out to control the muscles . Antigen: A substance that is capable of causing the production of antibodies . Antigens may or CRISPR/Cas9: Genome editing technology that may not lead to an allergic reaction . allows the permanent modification of genes within an organism . By delivering the Cas9 Antioxidant: A chemical compound or substance nuclease bound to a synthetic guide RNA into a that inhibits oxidation . cell, the cell’s genome can be cut at the designed/ Assay: An investigative procedure (i e. ., experiment) desired location . This allows existing genes to in the laboratory . 4/9/21 FOReither INTERNAL be removed USEor added ONLY in . CRISPR stands Ataxia: Loss of balance . for Clustered Regulatory Interspaced Short Palindromic Repeats . Atrophy: The progressive loss of muscle mass, or wasting, caused by reduction in the size or number of muscle cells . It is one of the later D symptoms of als . ALS PROOF DNA: Deoxyribonucleic acid . Hereditary material that encodes genetic information . Axon: The long, hairlike extension of a nerve cell that carries a message to the next nerve cell . Dysarthria: Impaired speech and language due to weakness or stiffness in the muscles used for B speaking . Blood-Brain Barrier (BBB): A protective barrier Dyskinesia: Abnormality or impairment of formed by the blood vessels and glia of the brain . voluntary movement . It prevents some substances in the blood from Dysregulation: Dysregulate: An impairment of a entering brain tissue . physiological regulatory mechanism; to cause a Bradykinesia: Slowness of movement . dysfunctional level of an activity or chemical in an

APP-2 2021 ALS Association Mission Toolkit organism by disrupting normal function . Genotype: The genetic makeup (i .e ., DNA code) of an individual . Dysphagia: Difficulty in swallowing . Glutamate: Glutamate is one of the most Dystonia: A slow movement or extended spasm in common amino acids found in nature . It is a group of muscles . the main component of many proteins, and is present in most tissues . Glutamate is also E produced in the body and plays an essential role Electroencephalogram (EEG): A method of in human metabolism . It is a primary excitatory recording the brain’s continuous electrical activity neurotransmitter in the human CNS . by means of electrodes attached to the scalp . Embryonic Stem Cells: Embryonic stem cells H are the “blank slates” of an organism, capable of Hyperreflexia: Excessive response of muscle developing into all types of tissue in the body . reflexes when a normal stimulus is applied . Enzyme: A protein that acts as a catalyst in Hyporeflexia: Weak or absent muscle response mediating and speeding a specific chemical when a normal stimulus is applied . reaction . Excitotoxic: An agent that excities neurons which I can, over time, lead to neuronal death . Immune System: A complex system that is responsible for distinguishing us from everything F foreign to us and for protecting us against Fasciculation: Small, involuntary, irregular, visible infections and foreign substances . The immune contractions of individual muscle fibers . Often system works to seek and kill invaders . seen in the legs, arms, and shoulders of 4/9/21persons FOR Incidence:INTERNAL The occurrenceUSE ONLY of new cases of a with ALS . This is often described by people with condition . The incidence rate describes the ALS as “persistent rolling beneath the skin .” frequency with which cases are identified . Forced Vital Capacity (FVC): The amount of air Incidence is commonly measured in new cases that can be forcibly exhaled from the lungs after per 1,000 (or 100,000) of population at risk per year . taking the deepest breathALS possiblePROOF . It is measured by a test called spirometry, a type of pulmonary Induced Pluripotent Stem Cells (iPSCs): A type function test . The percent force vital capacity of pluripotent stem cell that can be generated is often used as criteria to participate in an ALS directly from adult cells . clinical trial . Inflammation: The nonspecific immune response Free radicals: Chemicals that are highly reactive that occurs in reaction to any type of bodily and can oxidize other molecules (i .e ., superoxide) . injury . It is a stereotyped response that is identical whether the injurious agent is a pathogenic G organism, foreign body, ischemia, physical trauma, Gene: Genes are the basic biological units of ionizing radiation, electrical energyor extremes of heredity . They are composed of DNA . temperature . Genome: All of the genetic information; all of the Inflammatory Disease: A disease that is hereditary material possessed by an organism . characterized by activation of the immune system

2021 ALS Association Mission Toolkit APP-3 Appendix to abnormal levels that lead to disease . Mutation: A permanent change, a structural alteration, in the DNA or RNA . Mutations can be Intrathecala: Injection into the innermost caused by many factors, including environmental membrane surrounding the central nervous insults such as radiation and mutagenic chemicals . system . Usually done by lumbar puncture . Mutations are sometimes attributed to random Interventional Trial: Type of trial or clinical research chance events . study in which exposure to a potential therapy Myelin: A fatty substance that surrounds and or drug is assigned and being tested . It is used insulates the axon of some nerve cells to help to determine the effectiveness and safety of a speed nerve transmission . It is important for potential treatment . proper function of the nervous system . Investigator: A person who carries out a scientific study . A researcher . N In Vitro: In an artificial environment outside the Nerves: Bundles of fibers that use electrical and living organism, such as in a dish or test tube in chemical signals to transmit sensory and motor the laboratory . information from one body part to another . In Vivo: In a living organism, such as a mouse or Nervous System: The system of cells, tissues, human . and organs that regulates the body’s responses to internal and external stimuli . In vertebrae it L consists of the brain, spinal cord, nerves, ganglia, Lower Motor Neurons: Nerve cells (motor neurons) and parts of the receptor and effector organs . originating in the spinal cord that connect Neuron: Neurons are the nerve cells which make to muscles, conduct signals to allow muscle up the central nervous system . They consist of a movement . nucleus, a single axon which conveys electrical signals to other neurons and a host of dendrites M which deliver incoming signals . Molecule: The smallest unit of a substance 4/9/21 that FORNeurodegenerative: INTERNAL USE The progressiveONLY loss of the can exist alone and retain the character of that structure and function of the nervous system, substance . especially neurons . Motor Neuron: A neuron that conveys impulses Neuroprotective: If an agent provides protection initiating muscle contraction or glandular to any part of the body’s nervous system, it is said secretion . ALS PROOF to provide neuroprotection . Motor Neuron Disease (MND): A group of Neurotransmitters: Chemical substances that disorders in which motor nerve cells (neurons) carry impulses from one nerve cell to another; in the spinal cord and brain stem deteriorate found in the space (synapse) that separates the and die . ALS is the most common motor neuron transmitting neuron’s terminal (axon) from the disease . receiving neuron’s terminal (dendrite) . Muscle Atrophy: Loss of muscle fiber volume characterized by a visible decrease in muscle size . O This occurs because muscles no longer receive Observational Study: Type of trial in which impulses or signals from nerve cells . enrolled participants are observed . Outcome

APP-4 2021 ALS Association Mission Toolkit measures (i .e . measures of strength or function) Precision Medicine: A tailoring of medical may be part of the observation . No treatment/ treatment to the individual characteristics of drug is given . It is often used to learn about trends each person, while taking into account individual of symptoms, the course of disease, and can variability in genes, environment, and lifestyle include biomarker studies . for each person . In precision medicine programs, researchers aim to learn as much as possible from Oxidative Stress: Accumulation of destructive each unique person living with ALS . molecules called free radicals can lead to motor neuron death . Free radicals damage components Protein: Proteins are large molecules required of the cells’ membranes, proteins or genetic for the structure, function, and regulation of the material by “oxidizing” them – the same chemical body’s cells, tissues, and organs . Each protein reaction that causes iron to rust . has unique functions . Proteins are essential components of muscles, skin, bones, and the body P as a whole . Protein is also one of the three types of nutrients used as energy sources by the body . Phenotype: The observable characteristics of an individual resulting from the expression of genes . Proteomics: The study and identification of the This may be directly observable (eye color) or proteins produced by the genetic instructions apparent only with specific tests (blood type) . carried by a cell . Some phenotypes, such as the blood groups, are Protocol: A precise and detailed plan for the study completely determined by heredity, while others of a biomedical problem or for a regimen of an are readily altered by environmental agents . experimental therapy . Pluripotent Stem Cells: Human pluripotent stem cells are a unique scientific and medical resource . Q They can develop into most of the specialized Qualitative: Relating to measuring or cells and tissues of the body, such as muscle4/9/21 cells, FOR INTERNAL USE ONLY measurement of the quality of something, such as nerve cells, liver cells, and blood cells . They are its size, appearance, etc . self-renewing, making them readily available for research and, potentially, for treatment purposes . Quantitative: Relating to measuring or Scientists derive these unique cells from human measurement of the quantity (amount) of something . embryos, from fetal ALStissue, orPROOF from adult tissue (in the case of induced pluripotent stem cells (iPSCs)) . R Positron Emission Tomography (PET) Scan: A RNA: Ribonucleic acid . The primary function of computer-based imaging technique that provides RNA is to act as a messenger carrying instructions a picture of the brain’s activity rather than its from DNA for controlling protein synthesis within structure . The technique detects levels of injected a cell . glucose labeled with a radioactive tracer . Potassium Channel: A type of ion channel that S forms potassium-selective pores that span the cell Sclerosis: A hardening within the nervous system, membrane, thereby helping transport potassium especially of the brain and spinal cord, resulting across the cell membrane . They are found in most from degeneration of nervous elements such as cell types and control a variety of cell functions . the myelin sheath .

2021 ALS Association Mission Toolkit APP-5 Appendix

Sialorrhea: Drooling . impulse in the next neuron . Spinal Cord: Part of the central nervous system Synergistic: Interaction or cooperation between extending from the base of the skull from the two or more substances or organizations to brain stem through the vertebrae of the spinal produce a greater combined effect . column . It carries information from the body’s nerves to the brain and signals from the brain to T the body . Toxicity: The extent, quality or degree of being Stem Cells: Cells that can differentiate into many poisonous . different cell types when subjected to the right Transgenic: An organism whose sperm or egg biochemical signals . Stem cells are a promising contains genetic material originally derived from new therapeutic approach to treating central an organism other than the parents or in addition nervous system disorders . The most versatile stem to the parental genetic material . cells, called pluripotent stem cells, are present in the first days after an egg is fertilized by sperm . Translational Research: Studies that apply findings Researchers believe they can coax stem cells to from basic science discovered in the lab to become whatever tissues patients need . Stem relevant disease therapies that enhance patient cells come from embryos, bone marrow, and well-being . umbilical cords . View the stem cell glossary to Trophic Factor: One of a class of proteins that help learn more . keep cells healthy . Stratify: To arrange or classify . Superoxide Dismutase: An enzyme that destroys U superoxide, which is a highly reactive form Upper Motor Neurons: Nerve cells (motor of oxygen . With ALS, 20 percent of the total neurons) originating in the brain’s motor cortex population of patients have mutations in the gene and running through the spinal cord . for copper/zinc superoxide dismutase type SOD1 . SOD1 normally breaks down free radicals ,4/9/21 but FORV INTERNAL USE ONLY mutant SOD1 is unable to perform this function . Vector: The agent used (by researchers) to carry Synapse: A tiny gap between the ends of nerve new genes into cells . Plasmids currently are fibers across which nerve impulses pass from one the vectors of choice, though viruses and other neuron to another; at the synapse, an impulse bacteria are increasingly being used for this causes the release of a neurotransmitter, which purpose . ALS PROOF diffuses across the gap and triggers an electrical For more ALS vocabulary, visit The ALS Association glossary online, found at http://www.alsa.org/research/our-approach/glossary.html

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The ALS Association 1300 Wilson Boulevard Suite 600 Arlington, VA 22209 www.als.org