OUR MISSION

OUR MISSION TABLE OF CONTENTS OUR MISSION

Our Case for Support ...... iv Annual Impact ...... v Our Vision and Mission ...... vi CHAPTER 1: RESEARCH

The ALS Association Research Program The ALS Association Research Program ...... 1–1 Research ...... 1–5 Our Strategic Initiatives ...... 1–5 Infographics Current Projects ...... 1–13 Accelerating the Search for a Cure ...... 1–14 Research By the Numbers ...... 1–15 Research Strategy ...... 1–16 Funded Research ...... 1–17 Scientific Focus Areas ...... 1–18 Advances in ALS Research ...... 1–20 Research Initiatives The ALS Association’s ALS Roundtable Program ...... 1–22 ALS Voice of The Patient Report ...... 1–24 ALS Focus Survey Program ...... 1–26 Research Staff ...... 1–28 CHAPTER 2: CARE SERVICES

How We Work Summary ...... 2–1 How We Work ...... 2–2 Chapter Membership ...... 2–4

ii 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 — Living with ALS: General Scope ...... 2–22 Education — Living with ALS: Medical Resources ...... 2–24 Assistive Technology — Assistive Technology in the ALS Space ...... 2–26 Children’s Programs and Resources ...... 2–28 The Jane Calmes ALS Scholarship Fund ...... 2–32 ALS Association Care Connection ...... 2–33 Care Services Staff ...... 2–34 CHAPTER 3: ADVOCACY

Introduction To ALS Advocacy Introduction ...... 3–1 History of ALS Advocacy ...... 3–2 History of ALS Advocacy: Timeline ...... 3–3 Advocacy Action Advocacy Guiding Principles ...... 3–5 How We Advocate ...... 3–6 Priorities, Impact, and Coalitions ALS Guidance ...... 3–10 Advocacy Impact ...... 3–12 Advocacy Staff ...... 3–13 How We Collaborate ...... 3–15 APPENDIX

Abbreviated Glossary of Scientific Terms...... APP–2 © 2020 The ALS Association

iii 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 informs 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 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 $20 million in the Department of Defense ALS Research Program and $10 million for the National ALS Registry . Our care services team served more than 20,000 people living with ALS, including 8,920 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 .

iv ANNUAL 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 SUPPORTED FIRST ALS PLATFORM TRIAL PUBLICLY AVAILABLE GENETIC DATA PROPELS INNOVATION The ALS Association invested $3M in Healey ALS platform Genomic sequencing is a process to discover ALS new trial to accelerate drug development . This ground- genes . The Association contributed $3 .3M to fund a breaking trial will test multiple therapies for efficacy centralized, cloud-based, genome-sequence database . at the same time, providing time and cost savings and The first of its kind, it will allow all researchers to share increasing patient access to trials . genomic data and conduct standardized analyses, leading to identification of new ALS drug targets . REGULATORY GUIDELINES ADVANCE ALS THERAPEUTIC DEVELOPMENT • The Association released Principles for Urgent, COLLABORATIONS MOVE ALS RESEARCH PROGRESS FORWARD Patient-Centered ALS Clinical Trials, a series of • The Association teamed up with ALS Finding a recommendations to clinical trial sponsors and Cure and the MDA to co-fund Houston Methodist regulatory bodies worldwide . The principles set out a Neurological Institute and Massachusetts General clear framework for ALS clinical trials that are efficient, Hospital for a first in-human T-regulatory cell (Treg) impactful, and respectful to people with ALS . clinical trial totaling more than $2 .5M . This trial will • The Association worked closely with members of the test whether patients’ own immune Treg cells can be ALS community and the FDA to create a landmark, leveraged to treat ALS . patient-led guidance initiative that helps drive ALS drug • The Association collaborated with Target ALS to fund innovation more quickly from the lab to the patient . The the TDP43 Biomarker Initiative with a $350,000 grant . Association provided feedback to the FDA by hosting a Mutated TPD43 is a major toxic protein found in ALS . national workshop and creating a report that informed This project will lead to development of a biomarker the final guidance document . The guidance provides test to reliably measure TDP43 in human biofluids, regulatory flexibility in trial design and drug approval tremendously impacting ALS clinical research and drug resulting in direct benefits for people with ALS . development . Advocacy Supported over 690 meetings with Members of Fought for $10 million in funding for fiscal year 2020 + Congress at the 2019 Advocacy Fly-In and National ALS $10M for the National ALS Registry at the Centers for 690 Advocacy Conference . Empowered more than 20,000 NATIONAL Disease Control and Prevention to identify genetic MEETINGS ALS Advocates with eleven action alerts ­­—­­ resulting in ALS and environmental factors for ALS, provide support ACTION nearly 73,000 advocacy messages to Congress . REGISTRY to researchers to find treatments and a cure, and 11ALERTS promote access to clinical trials . Enrolled bipartisan congressional champions to Initiated and led the campaign to increase funding for introduce the SMART (Safeguarding Medicare Access the ALS Research Program at the Department of Defense to Respiratory Therapy) Act to ensure Medicare $20 (DOD) to $20 million in fiscal year 2020 — a $10 million beneficiaries with ALS can access noninvasive ventilators increase over the funding levels in previous years . (NIV) by removing NIV from competitive bidding . MILLION Spearheaded the effort to pass the bipartisan ALS Disability Insurance Access Act to waive the 5-month wait for people living with ALS . As of December 2019, 220 Representatives and 50 Senators support the bill .

Care Services people viewed, downloaded, 20,125 2,400 73,461 or ordered our educational people living with ALS veterans received assistance materials including the Living with ALS served through the chapter through our nationwide and Families and ALS resource guides and network in the past year chapter network medical information packets $4,995,832 25,588 8,920 in grants provided through attendees at support groups people served through our Certified our Certified Center Program offered through our vast Treatment Centers of Excellence and chapter network across the U .S . Recognized Treatment Centers

v OUR VISION: A WORLD WITHOUT ALS

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. RESEARCH THE ALS ASSOCIATION RESEARCH PROGRAM THE ALS ASSOCIATION RESEARCH PROGRAM

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

1–1 Our goal is to always ensure impactful We also fund the infrastructure to make ALS work gets funded, even if we do not fund it research more efficient and impactful . These ourselves . When we fund a project, we do so infrastructure projects include repositories with the intention that other larger funders, of specimens, genetic information and such as the federal government (like the NIH) electronic data that make it faster and or the private sector, will be able to carry cheaper for people to study ALS, and a the work forward . This strategy works . For smarter investment for private capital . example, an independent evaluation found We pay particular attention to the our Ice Bucket Challenge research spending infrastructure and capacity for clinical has served as a critical multiplier of funding trials . We are the primary supporter of from other sources . As a direct result of $40 the Northeast ALS Consortium (NEALS), a million in Ice Bucket research funding we network of clinical trial sites that facilitate awarded, ALS scientists reported receiving almost all the ALS trials in the United States . an additional $122 million in follow-on grants We also are supporting the Healy Center ALS from other funders . Platform Trial, which promises to speed ALS clinical trials . All these efforts move forward under the formal stewardship of our research program staff, external reviewers, and Everything we do works Research Committee .

toward discovering ALS MISSION INTEGRATION TO ADVANCE RESEARCH treatments and a cure The Association plays a critical role in research through our other mission areas as well . Our 70 plus Certified Treatment Centers of Excellence, overseen by our Care CAPACITY BUILDING Services program, are critical settings for ALS We work to increase the capacity of human studies and clinical trials in their local ALS research as an enterprise . We fund communities . multiple postdoctoral research fellowships There are many challenges in a person’s (apprenticeships for new PhDs) every year journey living with ALS, and there is a critical through the Milton Safenowitz Postdoctoral need for robust data to inform decisions of Fellowship Program . We are proud that over key opinion leaders to drive new programs 76% of our postdoctoral fellows go on to and policies to improve their lives . We are careers in ALS research, along with a large using our ALS Focus survey platform to percentage of the remaining staying in the help inject empirical patient and caregiver ALS space in industry or nonprofit jobs .

1–2 preference data into these decisions . We counts the number of people with ALS but are making these data available to the collects data and funds scientists throughout entire ALS community without charge, the country to better understand the causes and interoperable with other ALS research of ALS . We fight successfully for increased tools through global unique identifiers for federal research resources for ALS, and NIH people with ALS and their caregivers . Our spends about $5 for every $1 we directly ALS Roundtable Program is geared toward fund . We also recently fought to double the identifying challenges and coming to creative Department of Defense commitment to solutions with leaders in the ALS community ALS research to $20 million . Our Advocacy – in government, industry, and most and Research programs also collaborate on importantly, people with ALS and caregivers . engagement with the FDA, which has led This program has already identified the to many improvements in the conduct of disconnect between FDA treatment approval clinical trials, including the new ALS Clinical standards compared to insurance coverage Trial Guidelines released in 2019 . and payment standards . Our focus on the overall ALS research Our Advocacy program supports the National enterprise, and efforts to steward the ALS ALS Registry for ALS research, which not only research community to greater capacity,

1–3 efficiency, and collaboration, are working . In in ALS research simply would not have been 2014, we were able to support 42 principal possible without the infrastructure and investigators, and by the end of 2018 that broad collaboration we have supported over support had grown five-fold, to 237 principal time . We were able, for example, to fund investigators . In addition, the number of multiple gene studies and databanks that researchers collaborating on ALS-related pooled information and talent . More than scientific papers has nearly doubled since 250 scientists around the world analyzed over 2014 . 100,000 samples to identify a new genetic link to ALS, the KIF5A gene . Collaboration in the ALS space has already borne fruit . Some of the recent discoveries MOVING FORWARD Our research strategy is robust and successful . There is an ever-growing portfolio of exciting drug targets and genes, clinical studies, scientists, and companies new to the ALS space . As the ALS clinical trial enterprise grows larger and faster, we will have to pay closer attention to preclinical safety and toxicity projects, to ensure there are enough promising candidates to test . And as these clinical studies bear fruit and practice starts to change, we will have to shift resources to study new treatments in real world settings, to ensure these new treatments are being used effectively . Some of these expanded areas of study might be disease modifying therapies in clinic . They may also be vital assistive technology and care giving practices that need data to drive both better service delivery and policy . And we will be supporting research to detect and treat ALS earlier and earlier in the disease process, in the hopes of preventing disability altogether . We are just getting started .

1–4 RESEARCH Our Strategic Initiatives

he ALS Association has announced Learn more about each strategic initiative nine important research strategic here: https://www.als.org/research/research- Tinitiatives since the ALS Ice Bucket we-fund/partnerships-initiatives/strategic- Challenge to advance the search for initiatives treatments and a cure for the disease . In this document, we have included details PROJECT MINE on the goals of each initiative, along with $1.4 million original commitment partnered information on The ALS Association’s role with Greater New York and Georgia Chapters. and funding commitments . Total commitment since 2014 is over $2.1 Strategic initiatives are large global million. collaborations focused on the understanding An international, large-scale research of the disease, targeting new therapies, initiative devoted to discovering genetic expediting clinical trials, and making RNA and causes of ALS and to ultimately finding a DNA sequencing data available to the entire cure . The goal is to identify GENES associated ALS research community . These initiatives with ALS by performing whole genome will generate data and resources available for sequencing on at least 15,000 ALS patients researchers globally . Central to all the major plus 7,500 healthy controls worldwide, new collaborations are the people living with resulting in an open-source ALS genome ALS . database, in conjunction with the collection

1–5 of skin samples to make ALS patient- GENOMIC TRANSLATION FOR induced pluripotent stem cell (iPSC) lines . ALS CARE (GTAC) Our funding supports the U .S . arm of this $3.5 million commitment partnered with initiative, led by Jonathan Glass, M .D . (Emory Greater New York Chapter University) and John Landers, Ph .D . (University of Massachusetts Medical School) . Original A collaboration between Biogen and funding announced in October 2014 . Columbia University Medical Center (CUMC) to better identify new targets for treatment NEW YORK GENOME CENTER– development, in order to understand how CENTER FOR GENOMICS different genes contribute to various clinical OF NEURODEGENERATIVE forms of ALS . This will translate into clinical trials that are more focused . This project will DISEASES (NYGC CGND) follow 1,500 people with ALS and collect In 2015, The ALS Association funded $2.5 detailed clinical data, sequence their DNA million in our original commitment and and store blood cell samples to generate partnered with the Greater New York iPSCs . This study will allow correlation of Chapter. That commitment was matched ALS clinical symptoms to genetic causes and with an additional $2.5 million contributed help stratify patients for future clinical trials . by the Tow Foundation. In 2019, the Funding announced in August 2015 . Association committed an additional $3.5 million, including a $1 million commitment CReATe from the Association’s Greater New York Clinical Research in ALS and Related Disorders Chapter. That brings our total contribution for Therapeutic Development (CReATe) to $6 million. Consortium: $450K commitment for biomarker study and biorepository and an $835,937 A consortium that is a collaboration commitment to Drs. Paul Taylor, Jinghui Zhang, between numerous global laboratories and Michael Benatar for DNA sequencing . capable of generating and analyzing thousands of DNA sequences from people A Rare Diseases Clinical Research Consortium with ALS . The goal is to discover new genetic (RDCRC) that forms part of the National contributors of ALS to then translate into Institutes of Health (NIH) Rare Diseases Clinical clinical solutions for ALS . It houses all data Research Network . The goal of CReATe is to in a central repository that is freely available identify new genes and novel disease pathways to the research community worldwide . linked to ALS and related disorders . In addition Original funding announced in October to sequencing samples collected from study 2014 . Additional funding announced in participants, CReATe is building a resource February 2019 . of biosamples that have attached detailed clinical information, providing a unique and

1–6 critical resource for biomarker development . computational biology . The goal is to use iPSC The biorepository will enable the discovery lines from ALS patients and healthy controls and validation of biomarkers relevant to and OMICS methods to identify unique cell therapy development for patients with ALS and signatures that are specific to various subtypes related disorders . In partnership with The ALS of motor neuron diseases, in order to better Association, CReATe is funding pilot biomarker develop therapies and design clinical trials . projects using this resource, as well as other Funding announced July 2016 . biorepositories, including the Northeast ALS Consortium (NEALS) biorepository supported ALS ACCELERATED by The ALS Association . Funding announced THERAPEUTICS (ALS ACT) September 2015 . Original $10 million commitment matched with an additional $10 million contributed by y CReATe Connect: All ALS organizations ALS Finding a Cure® associated with CReATe are a part of Connect: A part of the Rare Diseases A novel academic-foundation-industry Clinical Research Network (RDCRN) partnership with ALS Finding a Cure, initiated Contact Registry, CReATe Connect is with researchers from General Electric (GE) an international online system to help Healthcare and four academic NEALS sites facilitate communication between doctors/ to accelerate treatments for people living scientists and patients and their families . with ALS . It is using the following strategies to CReATe Connect provides a means for develop new therapeutic approaches for ALS: patients with these rare diseases (and supporting development of neuroimaging their family members) to indicate their tools as potential ALS biomarkers; willingness to be contacted in the future supporting projects focused on decreasing about clinical research opportunities and to the production of misfolded proteins, and receive updates on the progress of research reversing neuroinflammation, two major and new educational opportunities contributors to the disease process; supports sponsored by CReATe . NeuroBANKTM (see below); and supporting Phase IIA pilot clinical trials with relevant NEUROLINCS biomarkers aimed at developing novel high- $2.5 million commitment partnered with the potential ALS treatments . A TDP 43 PET Tracer Greater Philadelphia Chapter Grand Challenge was launched as part of ALS A partnership with NIH’s National Institutes of ACT . and was awarded to Dr . Timothy Miller in Neurological Disorders and Stroke (NINDS) . 2017 to develop a novel TDP-43 biomarker to This National Institutes of Health (NIH)-funded track disease progression over time, improve collaborative effort is between various research diagnosis time, and understand whether a drug groups with expertise in iPSC technology, is hitting its target . Original funding announced disease modeling, OMICS methods, and October 2014 .

1–7 y NeuroBANKTM: original funding of openly shared; the Cleveland Laboratory $200,000 under ALS ACT – further at the University of California San Diego, expanded in August 2016 for an which will spearhead the development additional $1,679,091, bringing our total of antisense therapy against the C9orf72 commitment to $1,879,091. A patient- gene, the most common genetic cause of centric clinical research platform and ALS; and the Finkbeiner laboratory at the central repository that sets the framework Gladstone Institutes, which is affiliated with to allow for standardization of ALS University of California San Francisco, which patient information (including proteomic, will further develop robotic technology for genomic, and clinical data) that is linked screening drugs in motor neuron cell culture . across simultaneously-running research The Cleveland Laboratory is collaborating studies, locations, and modalities . It is with Martin Marsala, M .D ., at the University designed to host, curate, and disseminate of California San Diego and Brian Kaspar, this information . Global Unique Identifier Ph .D ., at the Research Institute at Nationwide (GUID) technology generates a patient- Children’s Hospital in Ohio . In 2014, The ALS specific character string that securely Association Golden West Chapter, along with identifies a patient without revealing their Advisory Trustees Jim Barber and Linda Della, true identity . NeuroBANKTM is part of partnered with the National ALS Association NYGC projects, GTAC, Answer ALS, and to build the Neuro Collaborative concept . For other ALS research projects . more information, click here. Original funding announced October 2014. NEURO COLLABORATIVE A $4.8 million commitment in October ANSWER ALS 2014 - funding through The ALS Association The ALS Association contributed to its with contributions from the Orange County development/business plan and is a partner and Wisconsin Chapters. To date, we have with Team Gleason and others to advance committed a total of $7.8 million. this initiative. We plan to contribute funds as An initiative founded as a collaboration the program evolves. between three leading California laboratories An initiative spearheaded by Steve Gleason aimed at discovering and developing new to challenge ALS researchers to come up potential ALS therapies that can be delivered with a solid plan to find a cure for ALS . Its to pharmaceutical companies for further strategy includes two impact goal arms . One development . The three laboratories are the is designed for immediate impact to help Svendsen Laboratory at Cedars-Sinai in Los ALS patients live more productive lives by Angeles, which will develop and maintain a supporting affordable assistive technologies Motor Neuron Core Facility to create iPSC and services . The other arm is designed to lines from people with ALS that will be contribute to the ultimate impact to fund

1–8 a collaborative effort to bring together initiatives to maximize collaborations to find the world’s best ALS researchers to find a treatments and a cure for ALS. treatment or a cure in the next five-10 years . An initiative founded by Kevin Gosnell, a As part of this initiative, all DNA samples from person who lost his battle to ALS, to bring participants will be sequenced by the New together leading neurology experts and care York Genome Center (NYGC), which will be specialists in Massachusetts to leverage their funded through ALS Association research institutions’ strengths to expedite progress programs . In addition, NeuroBANKTM toward finding a treatment for ALS by 2020 will be an integral part of the program . while improving care now . Institutional Projects funded as part of ALS ACT, the partnerships include Massachusetts General Neuro Collaborative, and NeuroLINCS form Hospital, the ALS Therapy Development an important foundation for Answer ALS . Institute (ALS TDI), the University of Partnership announced in September 2015. Massachusetts Medical School, and ALS ONE – MASSACHUSETTS Compassionate Care ALS . Under the ALS ONE umbrella, we funded research projects of Dr . ALS PARTNERSHIP Steven Perrin from ALS TDI, Dr . Nazem Atassi The ALS Association partnered with ALS from Mass General, and Dr . Robert Brown ONE and ALS Finding a Cure to fund $2 from U . Mass Medical School . million each for specific clinical and research

1–9 The Association’s funding to ALS TDI patient datasets, which are accessible to helped move its lead compound AT-1501 clinicians and scientists throughout the from preclinical studies into a phase I ALS research community . The project will clinical trial through TDI’s affiliate Anelexis be led by Dr . Iya Khalil, chief commercial Pharmaceuticals . Partnership announced officer and co-founder of GNS Healthcare . January 2016. Funding announced in The ALS Association’s partnership with GNS November 2016. Healthcare will transform Answer ALS’s petabytes of patient data into mechanistic GNS HEALTHCARE models, connecting genetic, molecular, and The ALS Association contributed $500,000 biochemical variables to clinical outcomes to GNS Healthcare to use artificial that will allow in silico experiments to be intelligence (AI) to create a comprehensive performed at a rapid rate on the computer . disease model to advance research into ALS. These rapid, high-throughput computational experiments will explore the numerous GNS Healthcare will use its powerful factors in the REFS Answer ALS data models machine learning platform, REFS, in that drive disease progression and drug conjunction with the rich Answer ALS response . Discoveries will then be evaluated and validated with wet lab experiments and, eventually, clinical studies . Funding announced July 2018 .

ALS REPRODUCIBLE ANTIBODY PLATFORM (ALS RAP) The ALS Association contributed $200,000 in partnership with the Motor Neurone Disease Association and the ALS Society of Canada for a total contribution of $600,000 to support ALS-RAP.

ALS-RAP was created as a public-private partnership comprised of world experts in antibody generation and validation, including Structural Genomics Consortium (SGC) and its associated labs at the Montreal Neurological Institute (MNI) McGill University in Montreal (Canada), the University of Oxford (UK), and the Karolinska Institute (Sweden) . The funding will support the creation of an open-

1–10 access pipeline to validate antibody research on Aging, National Institutes of Health and provide the ALS research community Read more here: https://www.als.org/stories- with the highest quality reliable, renewable news/als-association-contributes-33-million- antibodies for ALS genes . Standard operating fund-groundbreaking-genome-sequencing- procedures will be established to characterize database ALS antibodies – both commercially available and newly created – to ensure these exceed ALS PLATFORM TRIAL SGC and the Neuro’s stringent quality criteria The ALS Association invested $3M over three to establish a public list of “gold-standard” years in the ALS platform trial to accelerate antibodies that will ultimately lay a solid drug development. foundation for successful ALS therapies . Funding announced in March 2018. This groundbreaking trial will test multiple therapies for efficacy at the same time, GENOME-SEQUENCING providing time and cost savings and DATABASE increasing patient access to trials . The trial will take place at the Sean M . Healey & AMG The Association contributed $3.3M to fund a Center for ALS at Massachusetts General centralized, cloud-based, genome-sequence Hospital in collaboration with the Barrow database, including $100,000 from The ALS Neurological Institute and the Northeast Association Georgia Chapter. ALS Consortium (NEALS) . This investment The first of its kind database will allow all builds on the Association’s long history of researchers to share genomic data and supporting research at Mass General – over conduct standardized analyses, leading to $9 million just in the last 10 years . identification of new ALS drug targets . The Read the news: Association’s investment will fund cloud storage, cloud computing, and the genomic y https://www.als.org/stories-news/als- sequencing of 3,000 people living with ALS . association-invests-3-million-first-ever- als-platform-trial-speed-clinical-trial The creation of a central repository was conceived by three researchers: y https://www.massgeneral.org/neurology/ als/news/2020-01-14-ALS-association- y John Landers, Ph .D ., University of makes-commitment-healey-platform-trial Massachusetts Medical School Learn more about the ALS Platform Trial here: y Jonathan Glass, M .D ., Department of https://www.massgeneral.org/neurology/als/ Neurology & Pathology, Emory University research/platform-trial y Bryan Traynor, M .D ., Ph .D ., National Institute

1–11 INFOGRAPHICS CURRENT PROJECTS The ALS AssociationCURRENT is Accelerating PROJECTS Progress Towards Treatments The ALS Association is Accelerating Progress Toward Treatments

The ALS Association's Global Research Program

OR 3 MN MI NY NH RI WI 2 4 PA 19 1 3 3 8 MD MA CT 33 IL OH 14 2 2 CO 2 VA CA 1 MO 3 13 6 NC TN 1 2 AZ 5 GA 2 TX 4 LA

FIN 1 UK 1 FL CAN 4 5 GER BEL 9 IRL 2 1 3 FR SWI 1 ITL 2 4

MYS 1 Active Projects by Location

CHL UNITED STATES 2 SAF AUS 1 1 Arizona - 5 Missouri - 6 California - 13 North Carolina - 1 Colorado - 1 New Hampshire - 1 Connecticut - 2 New York - 19 Florida -9 Ohio - 2 Georgia - 2 Oregon - 3 Active Illinois - 2 Pennsylvania - 8 Louisiana - 1 Rhode Island - 3 Research Massachusetts - 33 Tennessee - 2 Maryland - 14 Texas - 4 171Projects Michigan - 4 Virginia - 3 Minnesota - 2 Wisconsin - 3 In an effort to accelerate progress toward finding treatments and a cure WORLDWIDE for ALS, as of 06/26/2020, The ALS Australia -1 France - 1 South Africa - 1 Association is currently funding 171 Belgium - 1 Germany - 2 Switzerland - 2 active research projects total. There Canada - 5 Ireland - 3 United Kingdom -4 are 143 within the United States. Chile - 2 Italy - 4 Finland - 1 Malaysia - 1

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

06/26/20201–13 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 disease and identify new therapeutic targets through gene sequencing, omics analyses, and epidemiological studies .

Therapeutic Development: Develop drugs and technologies against novel and genetic targets to test for preclinical efficacy and safety .

Clinical Phases (I/II/III): Test drugs for safety and efficacy in people with ALS . (Please note: The ALS Association does not fund phase III trials) .

Managing ALS: Impact lives of people with ALS today through funding research in developing assistive technology, measuring patient/caretaker burden, and improving quality of life .

Research Tools: Generate, characterize, and distribute standardized tools and animal models for preclinical use by the research community .

Biomarkers: Discover, validate, and develop new ALS biomarkers . Biomarkers are needed to improve measurement of ALS diagnosis, prognosis, and a person’s response to treatment .

Patient Cohorts: Support infrastructure to run clinical trials, support registry and recruitment for clinical trials, and enable open source sharing of data and biospecimens for research .

Community Engagement: Integrate patient voice and perspective, and engage stakeholders across the community including other nonprofits, industry, investors, government, and regulators .

Training & Education: Support training through postdoctoral and clinical fellowships . Provide training for principal investigators, trial coordinators, and clinical sites for optimal clinical trials .

1–14 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 .

2019 AWARDED NEW GLOBAL research RESEARCH PROJECTS IN 8 COUNTRIES $21.5 MILLION budget 70 The ALS Association Committed $21.5 million to 70 Promising Research Initiatives Worldwide

Clinician Scientist new $111 Development Postdoctoral Fellows Funded MILLION 2 Awards in 2019 6 dedicated to research since the in 2019 ALS Ice Bucket Challenge to advance treatments and a cure

new global strategic initiatives 11 NEW ALS GENES launched or (MATR3, CHCHD10, TUBA4A, TBK1, NEK1, C21orf2, 9supported ANXA11, TIA1, KIF5A, GLT8D1, and LGALSL) identified since the 2014 ALS Ice Bucket Challenge each representing new therapeutic targets

ALS drug trials and potential new ALS antisense drugs 33+ observational and numerous other drugs on the studies are horizon aimed to slow or stop the 32+ actively 3 progression of ALS enrolling*. *(as of 6/26/2020)

1–15 RESEARCH STRATEGY A Collaborative and Global Approach That Equals Immense Impact

Working with Top ALS Experts Attracting Bright New We collaborate with the world’s Scientists experts in ALS, whether in industry, We encourage new scientists to clinics, labs or government, as well as with enter and remain in ALS research experts in related disease areas to lead an and are dedicated to their continued success . exceptional research program . Impact: Over 76 percent of our postdocs Impact: Our highly competitive research remain in ALS research to start their own labs program funds the most ALS research dollars and mentor more new researchers . The other 14 percent hold jobs in industry, non-profit, of any ALS nonprofit, with a $17 million and medical writing . research budget in 2019, and is held to rigorous standards to drive innovation . Collaborating for Greater Impact Championing People Living with ALS Collaboration is the cornerstone of our research program . We partner with People living with ALS are at the academia, industry, government, and other center of everything we do and must nonprofit organizations . receive the best care and support possible . Impact: We lead by spurring long-lasting Impact: In 2019, we funded seven Managing collaborations among researchers across ALS projects focused on improving care for all sectors, leading to globally shared data, people with ALS and their families . protocols, and research samples to accelerate research progress . Investing in ALS Clinical Trials We sponsor ALS clinical trials Leveraging Investments to accelerate drugs through the We secure matching gifts to pipeline as quickly as possible . significantly increase donor Impact: We have funded numerous clinical investment from the ALS Ice Bucket trials over the years and have helped Challenge and beyond . countless drugs move from ideas into trials . Impact: Our original $1 .5 million investment Our support of the Healey ALS Platform trial in antisense technology infused over $100 is just one example, which will test multiple million and one FDA-approved drug for spinal muscular atrophy, SPINRAZA™, and therapies at the same time – saving time and two potential new ALS drugs targeting the money . SOD1 and C9orf72 genes .

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

1–17 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 and their caregivers, along with clinical trials Assistive Technology Challenge winners are to accelerate treatments through the drug dedicated to achieving this! development pipeline .

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

1–18 GENETICS STEM CELLS

The number of genes identified to Stem cell technology is progressing cause familial ALS has multiplied rapidly, and the ALS Association is since the discovery of SOD1 . Many efforts are spearheading work on several critical fronts underway to identify more ALS genes and to advance this key research tool . target them for therapy . The ALS Association continues to make significant investments in DISEASE MODELS identifying new genes and has supported all The ALS Association’s research the major 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 ALS therapeutics, and we are on the cutting edge, funding this exciting technology .

DRUG DEVELOPMENT The ALS Association 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 .

1–19 ADVANCES IN ALS RESEARCH A significant number of research innovations, advancements in drug development and clinical trials, new collaborations, and more, all aimed at finding treatments and a cure for ALS, accelerated the pace of discovery. We’re excited to share some of our most newsworthy research headlines that gave us and people living with ALS hope this year! The ALS Association is proud to provide funding for these exciting innovations.

HEALEY CENTER ESTABLISHES FIRST ALS PLATFORM TRIAL SIGNIFICANT ADVANCEMENTS FOR ALS CLINICAL TRIALS The ALS Association invested $3M in Healey ALS y Amylyx Pharmaceuticals, Inc, . (funded by the platform trial to accelerate drug development . This Association) announced that AMX0035 demonstrated ground-breaking trial will test multiple therapies for statistically significant treatment benefit for people efficacy at the same time, providing time and cost with ALS in the CENTAUR phase 2 trial . savings and increasing patient access to trials . y Biogen announced encouraging positive results for the drug tofersen* showing reduction of toxic levels of SOD1 protein and slowing disease progression in REGULATORY GUIDELINES ADVANCE ALS THERAPEUTIC people with ALS . Phase 3 trial to confirm its efficacy DEVELOPMENT and safety is underway . y The Association released Principles for Urgent, *The Association funded the drug’s underlying Patient-Centered ALS Clinical Trials, a series of technology. recommendations to clinical trial sponsors and regulatory bodies worldwide . The principles set out a COLLABORATIONS MOVE ALS RESEARCH PROGRESS FORWARD clear framework for ALS clinical trials that are efficient, y The Association teamed up with ALS Finding a impactful, and respectful to people with ALS . Cure and the MDA to co-fund Houston Methodist y The Association worked closely with members of the Neurological Institute and Massachusetts General ALS community and the FDA to create a landmark, Hospital for a first in-human T-regulatory cell (Treg) patient-led guidance initiative that helps drive ALS clinical trial totaling more than $2 .5M . This trial will drug innovation more quickly from the lab to the test whether patients’ own immune Treg cells can be patient . The Association provided feedback to the leveraged to treat ALS . FDA by hosting a national workshop and creating a y The Association collaborated with Target ALS to report that informed the final guidance document . fund the TDP43 Biomarker Initiative with a $350,000 The guidance provides regulatory flexibility in trial grant . Mutated TPD43 is a major toxic protein found design and drug approval resulting in direct benefits in ALS . This project will lead to development of a for people with ALS . biomarker test to reliably measure TDP43 in human biofluids, tremendously impacting ALS clinical PUBLICLY AVAILABLE GENETIC DATA PROPELS INNOVATION research and drug development . Genomic sequencing is a process to discover ALS new genes . The Association contributed $3 .3M to fund a INVESTMENT IN INFRASTRUCTURE PROTECTS AND ACCELERATES centralized, cloud-based, genome-sequence database . ALS RESEARCH The first of its kind, it will allow all researchers to share y The Association continued support of the Northeast genomic data and conduct standardized analyses, ALS Consortium (NEALS), the largest consortium of leading to identification of new ALS drug targets . ALS clinical researchers in the world . This funding will support new initiatives and ongoing programs to increase the quality and efficiency of ALS clinical trials . ALS FOCUS ESTABLISHED TO DRIVE MISSION STRATEGY y The Association funded Massachusetts General The Association launched ALS Focus, a survey program Hospital and Barrow Neurological Institute with that captures the needs and perspectives of people over $600,000 to improve clinical trial operational with ALS and their caregivers, placing them at the infrastructure and support initiatives to enhance center of ALS treatment and policy decision-making . clinical trial access and increase trial participant All data is open and free to the entire ALS community . recruitment and retention .

1–20 RESEARCH INITIATIVES THE ALS ASSOCIATION’S ALS ROUNDTABLE PROGRAM ince 2019, The ALS Association Roundtable Program has assembled Smembers of the ALS community for candid, facilitated discussions that shape the Association’s strategic planning efforts and find solutions to improve ALS care, advocacy, and research .

PROGRAM OBJECTIVES The Roundtable Program provides a forum for solution-based discussions with ALS stakeholders to accelerate the development of meaningful treatments for ALS and improve the lives of people with ALS and their caregivers . All Roundtable regulatory officials, public and private meetings include individuals with expertise insurance representatives, ALS Association and experience from a variety of sectors leadership, and — most importantly — people who reflect diverse points of view on with ALS and their caregivers . ALS-related issues . The group identifies challenges faced by people with ALS and DRIVING PRIORITY PROGRAMS their caregivers and then recommends A seat at the Roundtable meetings offers action that significantly impacts future care sponsors a unique opportunity to shape and treatment of the disease . and inform program-based solutions to challenges faced by the ALS community . BENEFITS OF PARTNERING IN ALS ROUNDTABLES INFORMING ALS RESEARCH AND POLICY GLOBAL COLLABORATION AGENDAS Roundtable meetings inform agendas and All members of ALS Roundtables benefit decision making on topics that are critical to from this collaborative forum, which the ALS community, including clinical trial promotes understanding and builds issues, regulatory and access evaluations, partnerships with industry peers, clinician payment determinations for ALS therapies, and scientific leaders, policy makers, and more .

1–22 EXAMPLES OF POTENTIAL 2020 time with the collaboration of the Association ROUNDTABLE TOPICS staff and the Roundtable Advisory Panel . A professional moderator is present to facilitate y Increasing Clinical Trial Participation and guide the discussion . Everyone will be y Home Health heard . After the Roundtable, an executive y Evidence Generation for Insurance summary is distributed to participants . Coverage Decisions Sponsors also have the opportunity to y Reducing Time to Diagnosis participate in working groups to further y Essential Coverage Benefits delve into identified challenges to advance y Patient and Caregiver Value Assessment concrete solutions .

PROGRAM COMMUNITY THANK YOU TO OUR 2019 ENGAGEMENT IN 2019 ROUNDTABLE SPONSORS The March 2019 Roundtable meeting held in Washington, D .C ., brought together 37 participants representing people with ALS, caregivers, clinicians, researchers, regulatory officials, and industry partners to provide input on the Association’s strategic priorities for the next three to five years . Participants collectively defined challenges and proposed solutions in key areas: reducing time to diagnosis, alleviating caregiver burden, increasing clinical trial participation, and enhancing home health .

The second Roundtable meeting held in October 2019 convened 44 participants, including a broad representation from public and private insurance . With many promising potential ALS treatments in clinical trials, the meeting focused on the topics of assessing value and ensuring access to drug therapies for people with ALS .

WHAT TO EXPECT Roundtable meetings will be focused on a clearly defined topic that is selected ahead of

1–23 ALS VOICE OF THE PATIENT REPORT

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

The vision of the IMPACT ALS survey was to systematically gather and expand available data on the perspectives and experiences of people with ALS and 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, 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 documentation of these findings and their formal submission to the FDA . This is the first time such data has been gathered to this extent across disease progression .

1–24 KEY TAKEAWAYS OF THE would like treatment options that stop ALS VOICE OF THE PATIENT disease progression, improve muscle weakness, and assist with breathing or REPORT: respiratory function . y Individuals with ALS and caregivers y The majority of people with ALS reported reported significant symptoms of fatigue having fears about the future . The fears and weakness, followed by speech most frequently reported were related to problems, shortness of breath, difficulty dying, “leaving family too soon,” followed sleeping, and pain . by “dying from respiratory failure .” The y These progressive symptoms result in a next most reported fear was “spending loss of independence and led to less time the family’s savings on medical care .” spent at work or school, socializing, or The IMPACT ALS survey is the first of a series traveling . of patient and caregiver surveys through the y People with ALS do not report high ALS Focus survey platform initiative, which satisfaction with currently available is a cross-sector collaboration to place the treatments and reported that such preferences of people with ALS and their treatments “somewhat help” with caregivers in the center of treatment and managing ALS and everyday impacts of policy development, through surveys and disease . Breathing assistance devices were studies . ALS Focus will build from the IMPACT reported to be very inconvenient and ALS survey and conduct robust research on burdensome, but necessary . patient and caregiver needs, preferences, and y With current treatments mainly focused experiences to produce generalizable and on supportive care, people with ALS open data for the entire ALS community .

1–25 ALS FOCUS SURVEY PROGRAM Bringing the perspectives of people with ALS and their caregivers to the forefront of research, care, and advocacy.

he ALS Association strives to improve ALS drug development, clinical trial Tdesign, clinical care, payment models, regulatory decisions, home health, and more . ALS Focus is a rigorous survey program that tackles these challenges by asking people with ALS and caregivers about their needs and experiences throughout the disease journey . Feedback is delivered to key decision-makers to continue the fight to improve quality of life for people with ALS . Importantly, all data collected is free and open to the entire ALS community . WHAT IS ALS FOCUS? ALS Focus is a survey program that captures the experiences and perspectives of people with ALS and their caregivers, putting the preferences of people affected by ALS at the center of ALS decision-making . The ALS Association is recruiting people with ALS and their caregivers for the ALS Focus survey program as they meet the challenges of ALS .

Information from this survey will help us fight for access to care and financial security for people with ALS and their families .

1–26 JOIN US - YOUR VOICE MAKES AN IMPACT Participate in research in addition to clinical trials. See your survey responses compared to the rest of the ALS community.

THE SURVEY DATA WE COLLECT IS: y Open and free to the entire ALS community y Protected – All data and findings are de-identified using a unique code called a global unique identifier (GUID) y Combined with other ALS research studies that use a GUID, such as the National ALS Registry and clinical trials, to broaden the impact of your participation y Actionable! Data will be used to inform decisions and strengthen programs and policies around: 9 Drug development 9 Drug payment and reimbursement 9 Clinical trial design 9 Clinical care 9 Regulatory review 9 Home health, and more

WHO CAN PARTICIPATE? People living with ALS Current or past caregivers of people with ALS HOW LONG WILL IT TAKE? Once registered, each secure online survey can be completed in approximately 15-25 minutes .

WHERE CAN I PARTICIPATE? Access the survey at als.org/ALSFocus .

*Participants must be at least 18 years old and reside in the United States . Survey instructions and questions are presented in English .

Learn more here: www.als.org/ALSFocus and for questions email: [email protected]

1–27 RESEARCH STAFF

NEIL THAKUR, Department of Veterans Affairs (VA), leading PH.D., CHIEF an evaluation service for the VA health system and represented the VA research MISSION OFFICER service in setting clinical performance Neil Thakur became measures . In his postdoctoral fellowship, Executive Vice President he studied the interactions between jails, of Mission Strategy in Medicaid and behavioral health care, and May 2018 . He leads the how changes in health financing impacted advocacy, care services, and research people’s utilization of these systems . During programs for the Association . graduate school, he worked throughout Prior to joining the Association, Neil served the Connecticut behavioral health system, in the National Institutes of Health (NIH) helping to implement managed care and Office of the Director, where he supported health information systems, and raise tens of NIH governance and helped make NIH millions of dollars in competitive grants . research more open and less burdensome . Neil won many awards for his government He managed the world’s largest policy to service, including several NIH Director’s make biomedical research papers publicly Awards, and the Secretary for Health and accessible and co-chaired the White House Human Services’ award for Meritorious taskforce that lead to the requirement that Service, the second highest award that the all federal science agencies adopt similar Secretary can bestow . He holds a Ph .D . in policies . He also spent a year on detail to Health Policy from Yale University School the U .S .Senate Special Committee on Aging, of Public Health and completed a NIMH raising awareness about quality issues in postdoctoral fellowship in mental health long-term health care, particularly around services research at the Cecil G . Sheps Center Alzheimer’s care and pharmaceuticals . for Health Services Research at the University Prior to his time at NIH, Neil worked with of North Carolina at Chapel Hill . He lives in health systems in many capacities . He Maryland with his wife, Jen . was Assistant Director of Health Services Research and Development at the

1–28 KULDIP DAVE, the pharmaceutical company Wyeth for PH.D.,VICE two years before joining The Michael J . Fox Foundation for Parkinson’s research . At MJFF, PRESIDENT, he oversaw a $30M portfolio and led two RESEARCH priority therapeutic areas . After nine years at Dr . Kuldip Dave, Vice MJFF, he joined The ALS Association has the President, Research, joined head of the Research program . The ALS Association in 2019 . Reporting to Neil Thakur, he oversees the Association’s JILL YERSAK, PH.D., Research Program . In this role, he develops VICE PRESIDENT, and implements the broader research vision MISSION STRATEGY by setting annual priorities and department Dr . Jill Yersak, Senior goals including research funding strategy, Director, Mission Strategy, research programs, and strategic initiatives . joined The ALS Association He is also responsible for the operational, in October 2015 . Reporting staffing, and budgetary oversight of the to Neil Thakur, she helps oversee the strategy research program . He engages with various of projects and programs that span the stakeholders including key opinion leaders Association’s mission – research, care, and from academia, government, industry, and advocacy . In this role, she leads mission other nonprofit organizations . He serves as a programs including the ALS Roundtable key communication lead for programmatic Program and ALS Focus survey program . She efforts by presenting the Association’s is also responsible for communicating ALS perspective and strategy to diverse audiences research in an accessible way by developing at workshops/conferences/forums and by and giving research presentations tailored developing content for written publications, to people living with ALS, their caregivers, blogs, social media, informational videos, and loved ones . She provides Association- perspective papers, and journals . 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 research development 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 . where he was first exposed to the drug Dr . Yersak received her bachelor’s degree in development process . He went to work for Biology at Ursinus College in Collegeville,

1–29 Pa . After college, she served as a research Focus, a survey program to understand the technician at the Children’s Hospital of experiences, preferences, and needs of Philadelphia in the department of Human people with ALS and their caregivers . She Genetics and Molecular Biology, focused on specializes in survey design, data analysis, and a pediatric genetic disease called 22q11 .2 studies on health perspectives and quality of Deletion Syndrome . She then went on to life . In her previous role as a research analyst complete her Ph .D . at Thomas Jefferson at RTI International, she evaluated the impact University in Philadelphia, with a focus on a of The ALS Association’s research program neurodegenerative disease called Kennedy’s following the ALS Ice Bucket Challenge . Disease . Dr . Yersak then moved to Providence, R .I . to complete her postdoctoral fellowship ASHTON FERRARA, under the mentorship of Dr . Anne Hart in MPH, MANAGER, the Neuroscience department at Brown RESEARCH University . During this time, she spearheaded OPERATIONS a project to generate precise ALS C . elegans Ashton Ferrara started models (which are microscopic worms), co- with The ALS Association wrote a successfully funded ALS Association in September 2019 . Reporting to Kuldip grant based on this project, and mentored Dave, she is responsible for issuing several graduate and undergraduate research contracts and managing contract students . Dr . Yersak is dedicated to mentoring negotiations, tracking compliance with and outreach . She served on the Board funded awards, supporting the development for the Association for Women in Science of new research programs and collaborating (Philadelphia) and volunteered for The ALS on impact measures of the research program Association Rhode Island Chapter and at The and reporting outcomes . Ashton received Louise Wilcox ALS Clinic in Providence, R .I . her bachelor’s degree in Public Health from SARAH PARVANTA, James Madison University in Harrisonburg, PH.D. MPH, VA and a Master of Public Health (MPH) with a concentration in Health Promotion from DIRECTOR, ALS Liberty University in Lynchburg, VA . Prior FOCUS to joining the Association, Ashton worked Dr . Sarah Parvanta, ALS as a Contract Administrator with Patient- Focus Director, has a Centered Outcomes Research Institute Ph .D . in communication from the University (PCORI), where she managed pre and post of Pennsylvania and a master of public award activities for Healthcare Research, health (MPH) from the University of North Research Infrastructure (PCORnet) and Task Carolina at Chapel Hill . Dr . Parvanta leads Order based Contracts . development and implementation of ALS

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

1–31

CARE SERVICES HOW WE WORK SUMMARY

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

2–1 HOW WE WORK

s a result of our commitment to The Association uses continuous feedback clinical care and education, families mechanisms and quality improvement Ahave access to information they processes to actively assess the needs of the may need to develop a care plan that communities we serve in order to address will result in enhanced quality of life and gaps in service delivery . This serves as the optimal personal and family outcomes . foundation for developing and delivering Surveys, focused conversations, and advisory innovative services and for increasing access groups — which include diagnosed persons, to care services . caregivers, families, and care teams — help y Designing, implementing, and monitoring the Association identify unmet needs . certification and other clinical programs Regular analysis of models of delivery, usage, that support consistency and quality and outcomes helps the Association refine its in services provided through Certified programs, services, and processes to identify Treatment Centers of Excellence, priorities and maximize positive outcomes . Recognized Treatment Centers, affiliated

2–2 providers, and other delivery models — all The Association continues to strengthen based on established best practices and relationships internal to the Association while standards of care, acknowledging the interdependencies among y Building upon our educational resource chapter network, Care Services, Research, libraries — including written publications, Public Policy, and national office support videos, web pages, and other media — for departments . The Association assumes a lead the provision of consistent information for role in developing collaborative relationships our communities . and partnerships with the communities we y Engaging in quality improvement serve, including medical providers, other reviews and training that supports an like-minded organizations, researchers, organizational culture that is attractive policymakers, regulatory agencies, and to a highly motivated and competent others to strengthen services provided and workforce serving our community, initiatives undertaken . including professional and knowledge The ALS Association supports a wide breadth development, management, team of specific fields of study that are critical to building, professionalism, interpersonal advancing ALS research . Active participation communication, and work-life balance . of people living with ALS in these studies is y Hosting a biennial National Clinical paramount to their success and provides the Conference to engage more than 500 foundation for continued work in areas that health care professionals who work on are of keen interest and show a promising ALS and other motor neuron diseases positive impact on people as they live with to discuss best practices and guidelines, this disease . multidisciplinary team care and The Association’s Clinical Management Grant coordination, ALS-relevant programs and Program supports clinical management services, research and technology updates, studies in order to improve care for people and other topics critical to the field . with ALS . The program focuses on the full y Hosting monthly educational webinars spectrum of clinical management, including presented by experts for people living gaps in the delivery of care, the development with ALS, their caregivers and family of telemedicine, assistive technology, and members, chapter staff, and professionals mental health care for people living with ALS who work with people affected by ALS . and their caregivers .

2–3 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 573 Arkansas Chapter 135 Central & Southern Ohio Chapter 441 Connecticut Chapter 369 DC/MD/VA Chapter 943 Evergreen Chapter 630 Florida Chapter 1,267 Georgia Chapter 613 Golden West Chapter 1,743 Greater Chicago Chapter 427 Greater New York Chapter 1,060 Greater Philadelphia Chapter 1,159 Greater Sacramento Chapter 285 Greater San Diego Chapter 234 Indiana Chapter 434 Iowa Chapter 267 Kentucky Chapter 267 Louisiana/Mississippi Chapter 301 Massachusetts Chapter 489 Michigan Chapter 525 Mid-America Chapter 699 MN/ND/SD Chapter 604 Nevada Chapter 52 New Mexico Chapter 130 North Carolina Chapter 799 Northern New England Chapter 198 Northern Ohio Chapter 366 Oklahoma Service Area 109 Orange County Chapter 232 Oregon and SW Washington Chapter 559 Rhode Island Chapter 99 Rocky Mountain Chapter 584 South Carolina Chapter 235 St. Louis Regional Chapter 327 Tennessee Chapter 339 Texas Chapter 1,125 Upstate New York Chapter 353 West Virginia Service Area 112 Western Pennsylvania Chapter 342 Wisconsin Chapter 479 0 200 400 600 800 1,000 1,200 1,400 1,600 1,800 2,000

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

2–5 Association Recognized Treatment CAREGIVER PROGRAMS sm Centers . y Providing expanded services to address y Cultivating relationships with community unique needs of ALS family caregivers, health care practitioners including including caregiver-only support or speech-language pathologists, physical resource groups . therapists, occupational therapists, y Facilitating caregiver self-care and caregiver respiratory therapists, social workers, and instructional education programs . home health, hospice, and other health y Hosting caregiver acknowledgment or care providers . retreat programs . SUPPORT PROGRAMS y Supporting access to The ALS Association y Coordinating and facilitating support Care Connection Program, which utilizes group meetings to provide education, an online calendar to coordinate family information, and networking opportunities needs with available family, friends, or for diagnosed persons, caregivers, and community volunteers . families . y Supporting grant funding to offset the cost y Developing targeted support or of caregiver respite services . program opportunities focused on the y Participating in National Family Caregiver specific needs of constituents, including Month activities . caregivers, children, or homebound y Offering a Bereavement Engagement persons . Program, respectfully acknowledging y Providing navigation assistance as a loss and reaching out to survivors diagnosed persons and their families on a time-specified basis to offer transition from private insurance to public encouragement and support . insurance programs, applying for disability benefits, or accessing other community, CARE MANAGEMENT GUIDANCE regional, and nationwide resources . AND CONSULTATIONS y Supporting grant funding to offset the cost y Providing routine communication with of professional counseling services . diagnosed persons and families regarding progression-appropriate information, and y Facilitating understanding of various care anticipated challenges and needs . modalities, including treatment, palliative care, and hospice care . y Providing guidance to support diagnosed persons and families in accessing health y Supporting family end-of-life care care services and making informed health planning with a focus on respecting care care choices based on knowledge of ALS, determination; personal, cultural, and health care resources, insurance, financial, religious values; and beliefs as practiced personal, and community resources . by individuals and families .

2–6 y Providing navigation assistance to families y Initiating and cultivating relationships investigating options and applying for with qualified DME providers interested resource benefits . in providing service excellence to the ALS y Providing appropriate referrals to community, including collaborating with professional service providers as appropriate . an identified vendor to support operation of DME Loan Program . DURABLE MEDICAL EQUIPMENT (DME) PROGRAMS ASSISTIVE TECHNOLOGY AND y Facilitating independence and quality of AUGMENTATIVE & ALTERNATIVE life for people living with ALS by providing COMMUNICATION PROGRAMS information and education related to the (AT/AAC) benefits of durable medical equipment y Providing education and information (DME) . related to how ALS impacts y Providing appropriate referrals to qualified communication and what types DME providers . of interventions are available to y Implementing a DME Loan Program support augmentative or alternative consistent with national DME Loan communication . program policies enabling persons with y Initiating and cultivating relationships with no or limited resources timely access to augmentative/alternative communication appropriate DME equipment on loan . (AAC) equipment vendors and speech-

2–7 language pathologists to facilitate access HOME VISIT PROGRAMS to appropriate evaluation, and selection y Providing personalized in-home or virtual and training in the use of communication home visits to consult with families equipment . in the comfort of their own homes y Implementing an assistive technology (AT) regarding the disease, typical progression, Loan Program consistent with national anticipated common challenges, available assistive technology/augmentative/ resources, and guidance in developing a alternative communications (AT/AAC) personalized strategic health care plan . Loan program policies enabling persons y Developing collaborative relationships with no or limited resources timely access with existing community health care to appropriate DME equipment on loan, home-visit providers, such as state or such as an iPad or speech-generating county health services, home health, device (SGD) . Visiting Nurse Association, and hospice y Providing expanded program services to maximize positive outcomes of service including employing or contracting with delivery . a speech language pathologist (SLP) or assistive technology professional (ATP) to GENERAL GRANT PROGRAMS deliver comprehensive AT/AAC support y Mitigating the enormous financial costs services . 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 the following eligible expenses: durable medical equipment (DME), assistive technology/augmentative/ alternative communications (AT/AAC), caregiver respite, transportation, home modification, medical or health care copays, or skilled or non-skilled home health care . SPECIFIC GRANT PROGRAMS y Cultivating relationships with qualified agencies dedicated to providing excellent in-home health services at a preferred

2–8 contracted rate, including nursing, y Providing targeted educational and physical therapist (PT), occupational informational projects for children and therapist (OT), speech-language youth, such as backpacks, welcoming pathologist (SLP), and certified nursing packets, children’s newsletter, family days, assistant (CNA) services . Support services picnics, and youth camps . via grant funding as available . y Collaborating with recognized children y Initiating and cultivating relationships with and youth content experts to develop qualified transportation organizations youth-focused conferences or supported able to provide appropriate transportation activities designed to provide age- to clinics and to medical, chapter, and appropriate information related to quality-of-life activities . Support services ALS progression and care, along with via grant funding as available . empowering networking opportunities, y Initiating and cultivating relationships such as DME Youth Conference and ALS with organizations qualified to provide Youth Bowling Day . appropriate home modification, enabling VOLUNTEER PROGRAMS diagnosed persons to more safely access y Developing a professionally-directed and navigate their home environment . program that connects community Support services via grant funding as volunteers to individual families living available . with ALS . YOUTH PROGRAMMING y Collaborating with existing community y Recognizing unique needs of ALS organizations to increase awareness families that consist of children and of unmet volunteer needs of families youth and expanding other general experiencing a journey with ALS . services — including education, support y Recruiting and training appropriate groups, conferences, and retreats — and volunteers to provide direct, non-medical programs to include and address children support for families . and youth, subject to age-appropriate communications and activities .

2–9 HOW WE SUPPORT CLINICAL CARE SUPPORTING CLINICAL CARE

person living with ALS and their with ALS can maintain independence longer loved ones face a variety of and experience an improved quality of life Achallenges along the journey . As when provided with options for symptom the disease progresses and new symptoms management, assistive technology, adaptive and difficulties arise, the number of people equipment, education, care services, and involved in providing care increases as emotional support . well . Clear communication among the care y The ALS Association’s Clinical Programs team and coordination of care and services currently consists of 73 Certified becomes vital . The Association’s Certified Treatment Centers of Excellence, 22 Treatment Centers of Excellence (CTCE) and Recognized Treatment Centers, and 87 Recognized Treatment Centers (RTC) provide Affiliated Clinics . compassionate care in a supportive, family- y The Association (National Office and oriented atmosphere . Centers that achieve Chapters) provided $5,189,183 in grants either of these designations meet program to our Certified Centers and Recognized requirements and follow recommended Treatment Centers ($4,995,832 to CTCEs best practices as outlined in the American and $193,832 to RTCs) in 2018 . Academy of Neurology Practice Parameter and collaborate with their local Association y 8,920 people were served through our chapter to offer care and support to people CTCEs and RTCs . living with ALS and their families . People

There is a difference between curing and healing. Unfortunately, at this time, there isn’t a cure for ALS. We do, however, believe we can heal the patient. This can be done by addressing the fact that ALS is not just a physical disease, but an emotional, spiritual, and psychological disease as well. The multiple aspects of ALS are best addressed in the setting of a multidisciplinary clinic that acknowledges the unique challenges that ALS patients and their families face.

– Scott D. Miller, MD, The ALS Association Recognized Treatment Center at Kaiser Permanente South Bay Medical Center

2–11 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 Treatment Centers, for example 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/Neuromuscular Neurologist – Nursing professional – ALS Association chapter liaison – Social Worker – MSW preferred – Speech Language Pathologist – Registered Dietitian – Occupational Therapist – Physical Therapist – Pulmonologist or Respiratory Therapist (RT) *

2–12 „ 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: – Basic science or bench research – Translational research – Clinical trials – Clinical research studies – Genetics – Other IRB-approved ALS-specific projects 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 .

2–13 CERTIFIED CENTERS Certified Treatment Centers of Excellence (CTCE) and Recognized Treatment Centers (RTC)

Requirements for the CTCE or RTC CTC 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 ✓ ✓ – Speech-Language Pathologist ✓ ✓ – Registered Dietitian ✓ ✓ – Occupational Therapist ✓ ✓ – Physical Therapist ✓ ✓ 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: • Basic science/bench research • Translational research • Clinical trials ✓ • Clinical research studies • Genetics • Other IRB-approved ALS-specific projects

2–14 WHERE WETHE SUPPORTALS ASSOCIATION CARE CHAPTER NETWORK Team Members (as of June 2020) AND CLINICAL PARTNERS (as of June 2020)

ALS ASSOCIATION CHAPTERS – 39

THE ALS ASSOCIATION CERTIFIED TREATMENT CENTERS OF EXCELLENCESM – 73

THE ALS ASSOCIATION RECOGNIZED TREATMENT CENTERS – 22 OTHER ALS ASSOCIATION AFFILIATED CLINICS – 87

THE ALS ASSOCIATION THE ALS OTHER 73 CERTIFIED TREATMENT 22 ASSOCIATION 87 ALS 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, multidisciplinary 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

06/12/2020 2–15 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 U Missouri Healthcare ALS Treatment Center MO Barrow AZ SLUCare ALS Clinic MO Phoenix Neurological AZ Atrium Health ALS Center NC Cedars-Sinai CA Duke University ALS Center NC Forbes Norris CA Wake Forest NC Keck Medicine of USC ALS Clinic CA U of Nebraska NE UC Davis CA Dartmouth-Hitchcock Medical ALS Center NH UC Irvine CA University of New Mexico NM UC San Diego CA Rutgers – Robert Wood Johnson NJ UC San Francisco CA Hospital for Special Surgery NY Loma Linda University CA Mount Sinai NY Hospital for Special Care CT Stony Brook NY VA Connecticut CT ALS Association Program at Columbia NY George Washington University Hospital DC University Jacksonville Mayo Clinic FL SUNY Upstate NY Phil Smith Neuroscience Institute at Holy Cross OhioHealth ALS Clinic OH FL Hospital ALS Clinic Cleveland Clinic OH U of Miami FL Louis Stokes – Cleveland VA Clinic OH U of South Florida FL Portland – VA Clinic OR U of Florida Jacksonville ALS Clinic FL Providence OR Augusta University ALS Clinic GA Oregon Health & Science University OR Emory ALS Center GA ALS Center at Allegheny Health Network PA U of Chicago IL Penn State – Hershey PA U of Illinois IL Jefferson Weinberg ALS Center PA Indiana University ALS Clinic at IU Health IN Medical University of South Carolina (MUSC) SC U of Kansas KS Baylor TX U of Kentucky KY Houston Methodist TX Ochsner ALS Clinic LA U of Texas – San Antonio TX U of Maryland MD U of Utah UT Curt and Shonda Schilling ALS Clinic at Lahey Richard R . Dart ALS Clinic (University of MA VA Clinic Virginia) Henry Ford MI U of Vermont VT Mercy Health MI ALS Center at the Swedish Neuroscience WA University of Michigan MI Institute Spectrum Health ALS Multidisciplinary Clinic MI Puget Sound – VA Clinic WA The ALS Center of Excellence at Hennepin Virginia Mason WA MN County Medical Center Froedtert Hospital ALS Center WI Mayo Clinic Rochester ALS Clinic MN Clement Zablocki Milwaukee VA Medical WI Center

2–16 RECOGNIZED TREATMENT CENTERS

Clinic Name ST Clinic Name ST Alabama Neurology Associates AL Pennisula Regional Medical Center MD Kaiser Permanente Los Angeles Medical Essentia Health Duluth ALS Clinic MN CA Center VA St . Louis Health Care System ALS Clinic MO Kaiser San Rafael ALS Multidisciplinary CA Clinic Dartmouth-Hitchcock Manchester Clinic NH Kaiser San Francisco ALS Multidisciplinary Hackensack Meridian ALS Clinic at Jersey CA NJ Clinic Shore University Medical Center Kaiser Permanente South Bay Lehigh Valley ALS Comprehensive Care CA PA Multidisciplinary ALS Clinic Center 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 U Louisville Physician’s ALS Clinic at Frazier KY Rehab The ALS Clinic of the MidSouth TN LSU 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 Mental Health ALS Professional Neurologist patient and their family . The ALS Association Centers are designed ALS to provide a regular, thorough, Respiratory Association Therapist Chapter and interdisciplinary evaluation; 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

2–17 SUPPORT THROUGH y Supporting the development and EDUCATION delivery of quality learning resources and opportunities for multiple age n ALS diagnosis can immediately groups (including children), for various leave people feeling distraught and educational settings, respecting reading struggling to cope . Our educational A abilities, and in at least two languages . priorities focus on providing critical y Developing educational resources that are information to people living with ALS, relevant . their families and caregivers, and medical professionals — who may or may not, have y Creating resources that can be prior experience with ALS . repurposed locally, nationally, and internationally . y Providing educational programs and y Informing caregivers about options to materials in a myriad of strategic forms, care for their loved ones, especially as the including face-to-face, workshops, person living with ALS loses the ability to symposiums, print, digital, and video . do so themselves . y Making sure content is accessible y Providing information on disease regardless of where a person lives and progression, symptoms and their their access to available technology . management, and considerations for y Involving our intended audiences during decision-making can help prepare the needs assessments, development and individuals and families for addressing design of materials and programs, and situations during the course of the disease . identifying appropriate methods of sharing . y Educating medical professionals about ALS treatment best practices for those who have limited experience or who have never encountered the disease before . 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 IMPACT & RESOURCES CARE SERVICES IMPACT People 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. 20,125 8,920 people living with ALS served through people served through our Certified the chapter network in the past year Treatment Centers of Excellence and Recognized Treatment Centers 500+ health care professionals attended the 2,400+ ALS Association’s Clinical Conference, veterans received assistance providing information on best practices through our nationwide chapter and guidelines, multidisciplinary team network care and coordination, ALS-relevant programs and services, technology for therapies, respiratory devices and 3 augmentative communication, along youth education books were developed with providing opportunities for CEUs for children, teens, and young adults, and professional networking addressing the many aspects of having a loved one living with ALS 73,461 $4,995,832 people viewed, downloaded, or in grants provided through our Certified ordered our educational materials Center Program including the Living with ALS and Families and ALS resource guides and medical information packets 25,588 attendees at support groups offered 2,387 through our vast chapter network times our 9 monthly educational across the U .S . webinars were viewed live or on-demand 150+ relationships with clinical partners, 661,246 incorporating best practices as unique page views on the Care Services established by the American Academy webpages on www.als.org of Neurology

2–20 FILLING THE GAP In Education, Knowledge, and Understanding

THE NEED 3 . Educating medical professionals about ALS treatment best practices for those n ALS diagnosis can immediately who have limited experience or who have leave people feeling helpless and never encountered the disease before . Astruggling to cope . There is a great need for new and innovative educational 4 . Creating public awareness of ALS with the materials to support this population . As general population to build support for ALS is a progressive disease, information is the ALS Association’s many initiatives . needed at every stage of the disease to help people with ALS, families, and their caregivers YOUR ROLE understand what to expect and how to cope . From print to our digital online library of Furthermore, education materials for medical educational materials, The ALS Association professionals, who may not have prior is looking to partner with you to make experience with ALS have been found to be important educational materials available . extremely useful in promoting the quality of care that people with ALS receive . SOME OPPORTUNITIES FOR PARTNERSHIP INCLUDE: THE ALS ASSOCIATION’S ROLE y Content and co-branding on print and The ALS Association is the leading digital materials . Our online educational organization in the ALS community, resources were accessed over 660,000 committed to finding treatments and a cure, times in the previous year and we have as well as providing support and educational a wide-reaching audience throughout materials for people living with ALS . Our the chapter network, with approximately educational mission priorities include: 14,000 people with ALS and their families 1 . Commitment to being the number one receiving services at any given time . resource for people living with ALS by y Building public goodwill through media providing educational programs and campaigns that demonstrate how your materials in myriad forms, including print, company supports an important cause . digital, and video . y Demonstrating to your employees that 2 . Informing caregivers about options to your company cares about the public care for their loved ones, especially as the causes that matter to them: the social person living with ALS loses the ability to piece of business . do so themselves .

2–21 EDUCATION Living with ALS: General Scope

THE ALS ASSOCIATION’S LIVING Nutritional Management in ALS WITH ALS RESOURCE GUIDES y Changes in Speech and Communication Solutions he ALS Association’s Living with ALS y Adapting to Changes in Breathing When Resource Guides are a module series You Have ALS that cover the progression of ALS T y Approaching End of Life in ALS and the many issues and considerations surrounding the disease, from diagnosis It is anticipated that the guides will be to end-of-life . These guides cover rapidly accessed online over 16,000 times over the expanding information and research in next year . The printed resources are available the clinical management of ALS and were through the Association’s order portal and designed to inform and educate people throughout the ALS Association chapter about ALS in a comprehensive and easily network, resulting in thousands of printed understood format . The resource guides guides being delivered to those in need . address the most common challenges, concerns, and issues facing people living with ALS . They are extremely popular among the ALS community due to the breadth of subjects covered in each .

The guides cover the following topic areas: y What is ALS? An Introductory Resource Guide for Living with ALS y After the ALS Diagnosis: Coping with the “New Normal” y Changes in Thinking and Behavior in ALS y Living with ALS: Planning and Making Decisions y Understanding Insurance and Benefits When You Have ALS y Managing Symptoms of ALS y Functioning When Mobility is Affected by ALS y Adjusting to Swallowing Changes and

2–22 The ALS Association is seeking $120,000 to speaking people with ALS, although, due to print 2,000 copies of all resource guides for underreporting, this number is likely higher . distribution to people with ALS, caregivers, The ALS Association recently translated all and professionals around the country. Living with ALS Resource Guides into Spanish to ensure that this population can access TRANSLATION OF RESOURCE information about ALS . GUIDES The ALS Association is seeking $60,000 to The Hispanic, Spanish-speaking population in print 1,000 copies of each Spanish-translated the United States reached 59 million people resource guide for distribution to people with nationwide in 2017 and is continuing to ALS, caregivers and professionals around the grow annually . Based on known prevalence country. rates, there are an estimated 3,400 Spanish-

2–23 EDUCATION Living with ALS: Medical Resources

COLLECTION OF MEDICAL RECORDS: MEDICAL INFORMATION PACKET hen providing emergency care, health professionals like Wparamedics and emergency room staff will have many questions about a person’s medical condition . In these stressful situations, it is often helpful to have an organized set of information, which can It can be printed and folded to fit easily in a help inform medical providers about specific wallet, pocket, car glove compartment, etc . issues and considerations . The ALS Association is seeking $6,000 to The Medical Information Packet has been support the costs to print 5,000 cards for developed to serve as a tool to inform distribution to our chapter network across medical providers caring for people with the country. ALS, as well as provide insurance and family information, in a centralized location . EDUCATION The sheets may be used as a packet or as individual pages, based on one’s preference LIVING WITH ALS: CAREGIVERS RESOURCES and need . How-To Video Series for Caregivers ALS not only affects those diagnosed with The ALS Association is seeking $9,000 to the disease, but also those who care for support the costs of printing 5,000 copies of them . While the person living with ALS has to the Information Packets for distribution to adjust their life to live with the disease, their our chapter network across the country. family members, or other caregivers, also IN CASE OF EMERGENCY: KEY have to make significant changes in their lives MEDICAL INFORMATION CARD to ensure proper care for the person living with ALS . In conjunction with the caregiver The Key Medical Information Card has been printed materials, The ALS Association has as developed to include those fundamentally a programmatic priority to create a ‘How To’ important key considerations that are Video Series . Often, caregivers lack the basic necessary to know in an emergency situation . medical training and education to assist with

2–24 the growing medical needs of their loved The ALS Association is seeking $120,000 to ones . The How-To video series will focus enable development of storyboard, subject on the subject matters relevant to meeting matter expert participation, and production the daily needs of a person living with ALS, of three videos in the upcoming year. Initial through presentations from subject matter priority subjects include “Functioning When experts . The goal of the video series is to Mobility is Affected by ALS”, “Adjusting ensure that caregivers are confidently able to to Swallowing Changes and Nutritional provide the best care . Videos will be based Management in ALS”, and “Changes in on information contained in the Living with Speech and Communication Solutions”. ALS Resource Guides .

2–25 ASSISTIVE TECHNOLOGY Assistive Technology in the ALS Space

or some, ALS will completely take away them . As a result, the person may have the their ability to communicate — verbally, technology available, but is unable to fully Fwritten, bodily . Without this ability to utilize its capabilities . There is a critical need communicate, ALS can leave a person feeling for specialists who have the expertise and trapped in their own body . This situation is knowledge within the assistive technology particularly significant as people with ALS are spectrum . This includes both high-tech and often homebound due to the difficulties of low-tech assistive speech devices . A trained leaving their residence . specialist is able to conduct more structured outreach to people living with ALS and family Technology continues to play a key role in members regarding options for alternative enabling ALS patients to maintain a level forms of communication and can provide of independence . As a prime example, ongoing support . assistive speech technologies allow patients to communicate when they lose their One of The ALS Association’s goals is to ability to speak . Computer access (via a hire Technology Integration Specialists (TIS) communication device or adapted computer, to support Association chapters . As The also known as augmentative and alternative Association builds its technology offerings, communication (AAC) devices) may be their the implementation plan requires the hiring only link to the outside world; their only of TIS’s to support multiple chapter service way to stay in touch with family and friends . areas . This vastly improves their quality of life, as The TIS’s primary goal is to build up a it provides them with the ability to gather chapter’s capacity to support technological information for decision-making, and offers advancements, including augmentative opportunities for a variety of interactions . technology, for people living with ALS in THE ALS ASSOCIATION’S ROLE their geographic region . The TIS will assist in developing strategies focused on engaging Often, ALS patients have difficulty in using staff, constituents, and people living with their devices and may have limited access ALS on utilizing technological opportunities to their clinic speech-language pathologist that benefit the ALS community as a whole . between clinic visits . One of the challenges This specialist will also work in conjunction The ALS Association faces is that, too often, with Speech-Language Pathologist (SLP) in Care Services chapter staff are neither ALS certified centers and clinics, vendors, trained in how to use these devices nor distributors, and other organizations able to instruct patients on how to best use providing communication devices throughout

2–26 their service area . This will be critical to These programs are imperative for people ensuring the creation of a culture in which living with ALS to remain as independent as appropriate resources and solutions are possible . Through your sponsorship, you will identified, planned for, sustained, and used have a role in helping to improve the quality safely and effectively . of life for people living with ALS .

The ALS Association is seeking $100,000 to YOUR BENEFITS: hire each TIS. The number of specialists will y Promotion of your own brand’s assistive be based on geographical distribution. technology on The ALS Association’s many YOUR ROLE platforms . y Branding on The ALS Association’s website The ALS Association is asking for your help or at Walks to Defeat ALS . in helping people with ALS to live a fuller, less isolated, life . By choosing to fund this y Personalized social media campaigns initiative, you will have a direct positive highlighting your support . impact on so many in the ALS community .

2–27 CHILDREN’S PROGRAMS AND RESOURCES

THE NEED caregiving population . In many instances, the isolation experienced by young caregivers oping with ALS can be especially restricts them from reaching out for difficult for the children of a parent assistance . Cor other close family member living with ALS, as children do not have the same The ALS Association continues to develop intellect and emotional maturity as adults . resources and programs that support efforts And, in most cases, they are not given to educate and assist children who have a the same age-appropriate educational loved one with ALS . Children handle trying resources to help them understand . This is a times differently than adults and The ALS widespread concern, as a significant number Association aims to ensure that they receive of people living with ALS report having a age and developmentally appropriate child who is directly impacted by the disease . services .

Children often don’t understand what is happening to a loved one with ALS, leaving them feeling scared of the unknown and restricted in how they may be able to spend their time, such as feeling responsible for helping to care for the diagnosed loved one, rather than socializing with friends . The need for them to provide direct caregiving (sometimes in intimate situations, such as bathing a parent), combined with the need for them to provide emotional support to adults and siblings, can lead to feelings of isolation from peers, depression, and decreases in self-worth and self-esteem .

THE ALS ASSOCIATION’S ROLE The ALS Association understands these young caregivers receive little of the attention given to their adult counterparts, leaving them a fragmented and relatively unknown

2–28 YOUR ROLE where children can interact and connect with other children impacted by ALS while The ALS Association is asking for your help participating in organized group encounters . in achieving its mission priority of helping Youths attending such camp programs all families live with ALS . By helping children have something in common (the significant directly impacted, you can enable positive impact of ALS on their lives) . They can build experiences and increased knowledge, new friendships and establish contacts, which will not only provide guidance during participate in sharing sessions, feel less alone their journey with ALS, but will also have a in their challenges, and have an ongoing lasting impact on their lives as they mature to communication plan . adulthood . Each multi-day camp requires a budget of YOUR BENEFITS approximately $40,000, which includes Through our abundance of resources, there expenses associated with venue, food, are a number of opportunities from which lodging, transportation for youths, activities, to choose a program that will positively and professional staff . No child is required to impact your brand . Some opportunities for pay for attending . partnership include: y Content and co-branding on one or more of our family-oriented resource guides y Build public goodwill by supporting a much loved, but underserved, demographic group y Demonstrate to your employees that you care about the causes that matter most to them 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 the US that provide specialized programs

2–29 The ALS Association is seeking $100,000 shock, sadness, and grief as their adult to support five camps, which will be held in counterparts . strategically identified locations across the The Families and ALS guide was created United States. Enrollment at each camp is primarily for families living with ALS, expected to be approximately 30 youths, 10 including parents, grandparents, siblings, chaperones, and 10 professional staff. and other family members . The goal of FAMILIES AND ALS RESOURCE the Families and ALS resource guide is to facilitate discussions surrounding ALS, GUIDE so family members can begin answering With no known cure and minimal known many of the questions that will be asked . treatments, ALS can be a difficult subject This resource also offers guidelines for to explain to children . The resource guide professionals assisting with families affected is the result of many years of clinical social by ALS . A key theme throughout the guide is work practice and formal research with communication — how to do it, maintain it, families, children, and youths affected and include it in everyday life . by neurological illness . While much attention is given to the person living The ALS Association is seeking $10,000 with ALS and their adult family member/ to fund the printing of 2,000 of this guide caregivers, children are often voiceless, for distribution to children, families, and despite experiencing much of the same professionals around the country. No family is charged for these resources.

2–30 ASSISTING WITH ACTIVITIES S . Kavanaugh, PhD, from the University of OF DAILY LIVING: DURABLE Wisconsin, who has conducted significant research on young caregivers and children MEDICAL EQUIPMENT who are impacted by a loved one’s ALS TRAINING diagnosis . This project will result in the Coping with ALS can be difficult for the many creation of the manuals that will provide children of a family member living with ALS, guidance, skill development, and support for given differences in emotional maturity and children and youth caregivers who provide lack of age-appropriate educational and care to their loved ones living with ALS . This supportive resources . This is a widespread project specifically addresses our mission concern, as a significant number of people priority to provide persons with ALS and living with ALS report having a child who is their family caregivers access to high quality impacted by the disease . Moreover, children consistent and compassionate support are tasked with the physical care of a person services . By working across Association living with ALS, yet had little to no training chapters, this project strengthens the ability or support, leading to anxiety and concern of the chapters to provide targeted, evidence- in their lack of skill . In a study of young based, and rigorously tested caregiver caregivers for ALS, 68 percent had no training educational programs, which will be made or education in providing care, despite having available to the ALS community nationwide . to handle an average of 11 tasks, including Some opportunities for partnership include: complicated assistive devices . These children expressed fear of harming their family y Content and co-branding on all manuals member with ALS, yet had no one to talk that are created by this project . with, underscoring their anxiety and concern y Building public goodwill through media in their lack of skill . campaigns that demonstrate how your company supports an important cause . THE ALS ASSOCIATION’S ROLE y Demonstrating to your employees that In order to assist with the education and your company cares about the public training of youths in caregiving including the causes that matter to them; the social correct use of durable medical equipment, piece of business . the ALS Association will create a specific program in conjunction with Dr . Melinda

2–31 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 T of 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 .

This scholarship will help me achieve my goals of becoming a registered nurse, and a healthcare provider with the skills to help others with illnesses like ALS. It will also help eliminate the financial burden, allowing me to focus more on my studies. The financial support lifts a weight off of my family and is a true blessing as our lives are progressively more affected by ALS.

— Emma Lauer, Nursing Student, Hibbing Community College 45 SCHOLARSHIPS WERE AWARDED IN 2019 Relationship to ALS Scholarship Recipients by College Type Scholarship Recipients by Year in School 31% 40 7% 29% 20% 20% 93%

4 1 Undergraduate Undergraduate Undergraduate Undergraduate Parent Grandparent Self 4 Year College/Grad School Junior/Community College 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 or to make a donation, please visit www.als.org/get-involved/jane-calmes-als-scholarship-fund.

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

2–32 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 https://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 .

2–33 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 living Health Plan in Boston, Massachusetts . with ALS had access to the highest standard of Prior to joining The ALS Association, Maginnis care in the state . She served on several National worked at Inova Health System in Falls ALS Association committees and task forces Church, VA for more than 20 years as senior including the Care Services Committee and the director of Corporate Health Services, Urgent Clinic Certification/Recertification Sub-team . Care Centers and Employee Occupational Lori joined the National ALS Association in 2019 Health . Some of her key accomplishments in where she brings her experience to her new this role include her developing successful, role . In managing the National Certified Center collaborative relationships with her colleagues Program, she works directly with both Chapters in other departments; achieving income and ALS multidisciplinary clinics to obtaining targets while managing risk and achieving and successfully maintaining designation as strong operational controls; developing a Certified Treatment Center of Excellence strategic business plans that yielded defined or Recognized Treatment Center . She works financial and operational successes . closely with all mission areas including Research and Advocacy . Lori received her BA in Maginnis holds a B .S . in Hospital and Health Psychology and Adult Education from Alverno Services Administration, Ithaca College, College . She has been a Licensed Practical Ithaca, New York and an M .B .A . in Business Nurse for over 30 years serving in many areas of Administration/Finance from American health care . University, Washington, D .C .

2–34 CYNTHIA to joining the national office, Leslie worked KNOCHE, RRT, at The ALS Association Rocky Mountain Chapter as the Care Services Director for 15 BBA, DIRECTOR, years . She received her Bachelor of Science CHAPTER CARE in Health Studies and a Master of Science in SERVICES Physical Therapy from Boston University, and Cynthia has served practiced clinical care in a variety of settings Association stakeholders since 2007 by before transitioning to the nonprofit sector . providing consultation, guidance, and support to the Association’s nationwide network MIRIAM BRODKIN of chapters with respect to the wide range MANAGER, of care and support programs they deliver . RESOURCE Her clinical background as a registered CONNECTION respiratory therapist, and management Miriam joined the ALS experience in the healthcare business Association in 1997 after arena support successful assessment and her mother passed away from ALS . It was development of program services . Cynthia important to her to help others with going works closely with our advocacy mission through the same experience . In her role, she team to support meaningful legislative and communicates with people living with ALS, regulatory benefits for those living with ALS . their families, healthcare professionals, and In addition, she leads professional outreach the general public through email and phone efforts as an invited guest speaker, presenting inquiries . Miriam helps connect people to allied practice healthcare and service to appropriate ALS Association chapter organizations on a state and nationwide level . and community services and programs to LESLIE RYAN, help them address important-day-to-day decisions and to find solutions and options MSPT, DIRECTOR, for managing the symptoms and journey of EDUCATION AND ALS . Miriam also helps people from other PROFESSIONAL countries identify resources in their own DEVELOPMENT communities . PROGRAMS Leslie joined the ALS Association in 2019 . In her position, she oversees the development and implementation of educational materials and programs for people living with ALS, the community, and healthcare providers . Prior

2–35 ALISA BROWNLEE, in History from East Stroudsburg University . CLINICAL She is a Certified Assistive Technology Professional (ATP) through the Rehabilitation MANAGER, Engineering and Assistive Technology Society ASSISTIVE of North America (RESNA) and a Certified TECHNOLOGY Aging in Place Specialist (CAPS) through the SERVICES National Association of Home Builders . Alisa is a 24 year staff member of the ALS Association working for both the Greater MARKEYA MARTIN Philadelphia Chapter and the National Office . COORDINATOR, Alisa speaks, blogs, writes articles and uses MISSION AREAS social media to increase awareness of ALS Markeya joined the ALS and the use of assistive technology . Her Association in 2018 . In specialty areas are communication, computer this role she assists with access, electronic aids for daily living and coordinating and planning home modifications . She is a strong advocate for the three mission areas; Care Services, for patient rights with elected officials, Advocacy/Public Policy, and Research . She insurance companies, and other healthcare also assists with organizing and overseeing providers . major grants and programs provided . Prior to Alisa is 2000 graduate of the California joining the national office, Markeya worked at State University Northridge with a Graduate LifeSpan Network in Columbia, Maryland as a Certificate in Assistive Technology . She has grant coordinator . She received her Bachelor a Bachelor of Science Degree in Hospitality of Science in Community Health from Hofstra Management and a Bachelor of Arts Degree University .

2–36 ADVOCACY INTRODUCTION TO ALS ADVOCACY INTRODUCTION

he ALS Association empowers advocates to talk about issues important to people living with ALS . Our goal is to educate policymakers and the public and to drive smart decisions Trelated to research, treatment, and access to care that benefit people with ALS . We are the largest and most influential national advocacy organization in the United States focused solely on ALS . We are a nonpartisan organization, and we are 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 including:

y development and promotion of legislation; y development/expansion of both Republican and Democratic Congressional champions; y direct lobbying of key members of Congress to improve services and increase research funding; y outreach to ALS chapters to lobby their congressional delegations; y mobilization of a vibrant grassroots network; y engagement of a variety of other ALS organizations; y partnerships with patient advocacy, physician and health professional organizations on cross-cutting issues such as pre-existing conditions; y use of social media to advocate to reach more people in more places .

3–1 HISTORY OF ALS ADVOCACY

ver the past two decades, The (CMS), the Food and Drug Administration ALS Association has been at the (FDA), Department of Veterans Affairs (VA), Oforefront of public policy efforts to and the Social Security Administration (SSA) . better the lives of people with ALS . The ALS Our chapters and advocates are essential Association is the only national nonprofit to our success . The Association empowers a organization fighting ALS on every front . national grassroots network of approximately As the preeminent ALS organization, the 20,000 advocates with the information, tools, Association leads the way in research, care and expertise needed to speak out and play services, public education, and public policy an active role in the fight for treatments — giving help and hope to those affected and a cure . Between February and July of by ALS . We bring our community together 2019 alone, ALS chapters and advocates to speak with one voice and advocate on accomplished over 690 meetings with behalf of people living with ALS and for ALS members of Congress in Washington, DC . research funding at the national level . We Many more meetings took place throughout advocate before Congress, the White House, the year in states and congressional districts . and with key federal agencies — including From January through October of 2019, the National Institutes of Health (NIH), the National Office sent ten action alerts Department of Defense (DOD), the Centers to chapters and advocates who in turn for Disease Control and Prevention (CDC), the generated more than 51,000 messages to Centers for Medicare and Medicaid Services Congress .

3–2 HISTORY OF ALS ADVOCACY Timeline

2001 2003 2006

The ALS Association The ALS Association successfully prompted the Social Security The ALS Association supported passage of successfully lobbied Congress Administration to publish new rules that made it easier for people the Lifespan Respite Care Act, which led to to waive the 24-month waiting living with ALS to qualify for Social Security Disability Insurance and $15 million in funding per year for the kinds period for Medicare coverage added ALS to the list of conditions that automatically qualify for of respite care programs that are urgently of people diagnosed with ALS . presumptive disability payments under Social Security Insurance . needed by people with ALS .

2008 2007

The ALS Association helped to implement historic regulations at the Department of Veterans The ALS Association spearheaded efforts to establish Affairs that designate ALS as a service-connected disease, ensuring that veterans with ALS a more focused, coordinated, and better-funded and their survivors have access to VA benefits, including health care and disability benefits approach for studying ALS, which led to the creation The ALS Association led the fight to establish the National ALS Registry, which secured of the ALS Research Program (ALSRP) as part of a federal funding to design, build, and implement the National ALS Registry at the Agency for $5 million appropriation in the FY2007 Defense Toxic Substances and Disease Registry, the single largest ALS research project ever created . Appropriations Bill .

2012 2013

The ALS Association helped enact the FDA Safety and Innovation Act (FDASIA), The ALS Association partnered with FDA to convene the which strengthened the Fast Track and Accelerated Approval processes, required first-ever ALS specific public hearing and urged the FDA FDA to partner more closely with patient organizations representing those to partner with the ALS community to help expedite the with rare diseases, and provided additional flexibility to FDA to approve new drug development and approval process and bring new treatments quicker through the use of biomarkers and other surrogate endpoints . treatments to patients as soon as possible .

2017 2016

The ALS Association brought the ALS community together to create the first patient-focused The ALS Association helped push Congress to pass guidance for ALS drug development ever submitted to the FDA . It has the potential to speed the 21st Century Cures Act, which included provisions access, reduce costs, ensure more effective use of resources, and incentivize the pharma/ that will improve the process of developing therapies biotech industry to enter the ALS market and develop new treatments for ALS . targeting rare diseases and authorized $4 .8 billion The ALS Association worked with Congress to increase funding for the ALS Research over 10 years for the NIH . Program at the Department of Defense from $7 .5 to $10 million, bringing the total funding for the ALSRP to nearly $80 million since the program’s inception in 2007 . The ALS Association played a key role in securing a bill to protect access to customized wheelchairs and accessories that caused CMS to reverse its decision to add these complex devices to the Competitive Bidding Program .

2018 2019

The ALS Association led the charge that secured $10 million in ALS Association initiated and led the successful campaign to increase funding funding for the Department of Defense (DOD) ALS Research for the ALS Research Program at the Department of Defense (DOD) to $20 Program and $10 million for the Centers for Disease Control (CDC) million - a $10 million increase - through direct lobbying, chapter advocacy, National ALS Registry . grassroots mobilization and engaging other ALS organizations . The ALS Association secured the first-ever Senate “Dear Colleague” The ALS Association spearheaded and co-led coalition efforts to protect letter circulated for signature by senators and sent to the access to noninvasive ventilators (NIV) in collaboration with physicians Appropriations Committee supporting funding for the DOD ALS and clinicians . This included meetings with Medicare program and the Research Program and the CDC National ALS Registry . introduction of the Safeguarding Medicare Access to Respiratory Therapy (SMART) Act of 2019 .

3–3 ADVOCACY ACTION ADVOCACY GUIDING PRINCIPLES

s legislative, regulatory, and other 2. Preserve and increase support for ALS policy proposals are considered, research. Funding and support must be Athey must be measured against the provided for research in all phases to mission and values of The ALS Association: discover new life-extending therapies and To discover treatments and a cure for ALS, technologies for people with ALS . The and to serve, advocate for, and empower ALS Association is dedicated to ensuring people affected by ALS to live their lives to laws, regulations, and incentives relating the fullest . to the therapy development process effectively and expeditiously promote The ALS Association’s public policy priorities new interventions and, ultimately, a cure are grounded in our advocacy guiding for ALS . principles which maintain that policy proposals must preserve and/or enhance 3. Fight for people living with ALS on a wide health care, benefits, and services for those range of issues. People with ALS should who have it, provide coverage to those who be protected against discrimination in all do not, and lower costs and improve quality settings, including in the workplace, when for all . This must be achieved at the federal purchasing health insurance, or receiving and state level and in both public and private veterans’ benefits . The ALS Association settings . Research funding must be preserved monitors legislation and regulation and increased, people with ALS must not regarding health care, disability rights, be discriminated against, and resources and taxation, and more to ensure the ALS support for caregivers must be increased . community is represented .

PRINCIPLES 4. Increase resources and support for families and caregivers of people with 1. Protect and expand access to health care, ALS. Families and caregivers are the People with ALS benefits, and services. lifeline for people with ALS and must and their families must have health care be recognized and supported . The that is accessible, affordable, adequate ALS Association believes federal, state, and understandable . The ALS Association and private programs must do more is committed to fighting for people with for families and caregivers in terms of ALS on every front to break down barriers programs, supports, and financial aid . to health care, benefits, and support services .

3–5 HOW WE ADVOCATE

he Association works closely with and form the backbone of our long-term volunteer leaders, ALS advocates, grassroots strategy . researchers, and partners on the T While our grassroots advocacy grew as a federal and state level to advocate result of the ALS Ice Bucket Challenge, it is for policies that are supportive of and through our chapters that we expand our responsive to the needs of people living with grassroots through their local connections ALS and their families . and the work that they do on the ground, Our network of over 20,000 grassroots every day . This helps us continue to build advocates is mobilized on a regular basis our roster of grassroots advocates, who through action alerts and comes together for help amplify our message and make our conferences and signature events throughout story personal . Leaders at the chapter the year to discuss and learn about public level also serve as our grasstops advocates policy priorities, and advocate directly with — building the Association’s long-term lawmakers . Once someone becomes an relationships with members of Congress advocate, they can be activated to contact and their staff, and bringing the local voice their Senators and Representatives on to Capitol Hill . important issues . The ALS Association employs several tools as The Association values a collaborative part of our grassroots advocacy strategy . approach to advocacy and belongs to several formal and informal coalitions, which allow us ACTION ALERTS AND to benefit from and leverage the combined CONTACTING CONGRESS expertise or the patient advocacy community . The Association maintains an Advocacy Lawmakers have limited resources and time, Action Center that allows us to reach out and they rely on coalitions to provide policy to thousands of advocates with updates solutions developed through consensus by on our public policy priorities and the diverse groups . latest developments from Capitol Hill, and to encourage them to contact their THE ROLE OF THE CHAPTER members of Congress and participate in the NETWORK IN ADVOCACY legislative process . In a matter of minutes, our Our chapter network plays a vital role in advocates can send a personalized email to our public policy efforts . Chapters serve as their members of Congress . a resource for what people with ALS need

3–6 ANNUAL FLY-IN THE NATIONAL ALS ADVOCACY Every year, The ALS Association hosts a CONFERENCE meeting in Washington, DC, where our Every summer, the Association holds its chapter executives and other leaders largest annual conference in Washington, DC get a chance to participate in a day of providing advocates from across the country meetings and training at the height of the an opportunity to come together to network, appropriations process . Chapter leaders share their stories, and meet with members engage in discussion with national office of Congress . People with ALS come with their staff and policy experts on the Association’s family members and chapter staff to learn public policy priorities and travel to Congress about legislation and regulations and what for meetings with lawmakers and their Congress needs to do . The conference also staff to discuss our priorities, including provides program sessions on Care Services, continuing appropriations for ALS research Research, and Communication . More than at the Department of Defense and National 600 people attend this meeting where they Institutes of Health, as well as the ALS receive materials and trainings on issues Registry at the CDC . and tips on how to meet with members of Congress . The most important participants

3–7 are people living with ALS and their families The National ALS Registry is the single largest — and their advocacy produces tremendous ALS research project ever undertaken . As the results . world’s largest population-based registry for people living with ALS, the Registry connects OPPORTUNITIES FOR patients directly with clinical trials, fuels ADVOCACY AT SIGNATURE research as a repository of data for scientists, EVENTS and empowers patients to make invaluable contributions to a future freed from ALS . The Walk to Defeat ALS provides an In January 2017, the ATSDR launched the opportunity for chapters to expand their National ALS Biorepository as an added roster of advocates and to participate in active component of the Registry, enabling advocacy with a large audience of people researchers to request samples from both who already have an interest in fighting ALS . living and deceased (post-mortem) persons Advocacy tents or tables serve as gathering with ALS . The ALS Association has led efforts spaces where chapters can sign up new in Congress that have secured more than $80 advocates or encourage existing advocates million in funding for the Registry since its to sign letters to lawmakers . Chapters also creation have the opportunity to invite members of Congress to participate in the walk, which gives the member an opportunity to address the audience and to interact directly with people with ALS and their supporters and see how this disease impacts their community .

THE NATIONAL ALS REGISTRY The ALS Association led the fight to establish the National ALS Registry at the CDC by working with Congress to enact the ALS Registry Act in 2008 . The legislation provided the Agency for Toxic Substances and Disease Registry (ATSDR), a branch of the CDC, with the authorization and guidance necessary to create a National ALS Registry . The Association partnered with the CDC as it implemented the Act, and for the first time began identifying cases of ALS on a nationwide basis and collecting information urgently needed for ALS research .

3–8 PRIORITIES, IMPACT, AND COALITIONS ALS GUIDANCE

he goal of a community-developed The ALS Association is actively engaged in ALS Guidance document is to serve as advocating for the most useful FDA Guidance Ta roadmap to help industry navigate and other regulatory improvements . In the drug development process and provide response to the issued FDA Draft Guidance, the FDA with an ALS community-centered the Association hosted a FDA Open view of how the Agency should approach Forum called, ALS Community Workshop: therapies for ALS . Developing Drugs for Treatment, Guidance for Industry . The forum took place in July 2018 Specifically, the ALS Guidance Document has in Washington, D .C . where over 90 members the potential to: of the pharmaceutical industry, clinical y Speed access, reduce costs, help ensure research experts, representatives from the resources are most effectively utilized, and FDA, and people with ALS and caregivers incentivize industry to enter the ALS market came together to provide targeted feedback and develop new treatments for ALS . and information to further inform the FDA’s y Provide input to FDA on how to make Draft Guidance on ALS Drug Development . the drug development process, including An FDA Workshop Report summarizing the clinical trials, more efficient, predictable, recommendations that from the day were and effective at assessing drug safety and delivered to the FDA and are available to the efficacy . public . The ALS Association submitted the first In August 2019, The ALS Association also patient-focused community guidance for released Principles for Urgent, Patient- Cen- ALS drug development to the Food and tered ALS Clinical Trials, a series of recom- Drug Administration in 2017 . It included mendations to clinical trial sponsors and reg- feedback of over 100 participants, including ulatory bodies worldwide . The principles set industry, clinicians and researchers, the out a clear framework for ALS clinical trials National Institutes of Health, the Centers that are efficient, impactful, and respectful for Disease Control and Prevention, and to people with ALS . most importantly, people with ALS and In September 2019, the FDA issued the ALS their families . The FDA was also engaged Drug Development Guidance for Industry . This throughout the process of developing the was one important step in comprehensive Association’s ALS Guidance . efforts to bring therapies to people with ALS more quickly . Five years ago, there was no In February 2018, the FDA released its Draft clarity around what the FDA expected from Guidance on ALS Drug Development .

3–10 companies pursuing ALS treatments . The of the final Guidance on the development of ALS Association recognized this problem, drugs and treatments for ALS . In our latest galvanized the broader community, and comments, the Association and our partners engaged the FDA to provide a clear roadmap recognize the Agency’s efforts to address that also can respond to new science as it community concerns raised after the Draft emerges . The FDA Guidance is one part of a Guidance was published in 2018, as reflected commitment that will not be complete until in changes made to the final version of the we have a cure for all people with ALS . Guidance .

In November 2019, The Association and its Read more here: https://www.als.org/stories- partners submitted comments to the FDA to news/als-community-responds-fda-guidance- further inform the agency’s implementation document-call-more-urgency-flexibility

3–11 ADVOCACY IMPACT With the support of over 20,000 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!

Enrolled bipartisan congressional Spearheaded the effort to pass the champions to introduce the SMART bipartisan ALS Disability Insurance (Safeguarding Medicare Access to Access Act to waive the 5-month Respiratory Therapy) Act to ensure wait for people living with ALS . As of Medicare beneficiaries with ALS can December 2019, 220 Representatives access noninvasive ventilators (NIV) and 50 Senators support the bill . by removing NIV from competitive bidding . Initiated and led the campaign to increase funding for the ALS Continued to play a key role in $20 Research Program at the Department ensuring people with ALS have access of Defense (DOD) to $20 million to wheelchairs, speech-generating MILLION in fiscal year 2020 — a $10 million devices, and other complex increase over the funding levels in technology through legislative and previous years. regulatory pressure . Fought for $10 million in funding Joined with other leading patient for fiscal year 2020 for the National organizations in the fight to $10M ALS Registry at the Centers for preserve protections for pre-existing NATIONAL Disease Control and Prevention to conditions for people with ALS ALS identify genetic and environmental under all insurance plans, including REGISTRY factors for ALS, provide support to Medicare, Medicaid, and private researchers to find treatments and a insurance . cure, and promote access to clinical trials . Supported over 690 meetings with 690+ Members of Congress at the 2019 Joined with other leading patient Advocacy Fly-In and National ALS $41.7B organizations to secure for $41 .7 MEETINGS billion in fiscal year 2020 funding ACTION Advocacy Conference . Empowered more than 20,000 ALS Advocates for the National Institutes of Health 11ALERTS (NIH) . In fiscal year 2018, NIH with eleven action alerts ­­—­­ resulting NIH in nearly 73,000 advocacy messages invested $83 million on ALS research . to Congress .

3–12 ADVOCACY STAFF KATHLEEN SHEEHAN, VICE and Mental Health Services Administration . PRESIDENT, PUBLIC POLICY Kathleen also possesses expertise in public policy issues related to the development and Kathleen brings over approval of pharmaceuticals at the Food and 20 years of government Drug Administration . relations experience on health care issues to ABRAM BIELIAUSKAS, The ALS Association . As ASSOCIATE DIRECTOR, an advocate and policy expert, she has extensive GOVERNMENT AFFAIRS knowledge of patient concerns as well as the Abram joined The ALS complexities of Medicare, Medicaid, private Association in 2017 and insurance, and pharmaceuticals . Throughout works with Kathleen her career, Kathleen has championed access Sheehan to lobby Capitol to care for patients, as well as increased Hill on the Association’s appropriation for health science research various public policy at NIH and other federal agencies . Kathleen priorities . Mr . Bieliauskas is a respected health care policy strategist previously served as Government Affairs known for her bipartisan approach and and Advocacy Specialist at the Pancreatic ability to create effective relationships Cancer Action Network, where he managed across the political spectrum . Hallmarks of logistics for that organization’s Advocacy Day her career include vibrant engagement with conference, oversaw a national grassroots chapters and volunteer leaders, as well as network of advocates strategically located the development of effective collaborations in key congressional districts, and advocated with other national organizations . Kathleen through influencing Congress to support has held leadership positions with the increased cancer research funding . Abram American College of Cardiology, the Visiting also brings more than a year of Capitol Nurse Associations of America, the Maryland Hill experience, having worked in both the Homecare Association, as well as national US House and Senate . He is leveraging his organizations focusing on substance abuse appropriations experience to support CDC and mental health . Her leadership has and ALSRP . Originally from Cincinnati, Ohio, resulted in numerous legislative victories, Abram moved to Washington, DC, after including securing a key provision in the graduating cum laude with a bachelor’s Affordable Care Act and crafting key sections degree in Political Science Pre-Law from Ohio of reauthorization for the Substance Abuse University .

3–13 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 Concentration, from Johns Hopkins University .

3–14 HOW WE COLLABORATE The Value of Coalitions

hen organizations join forces enable The ALS Association to join with and work together to impact others to examine complex issues and cross- Wpolicy, they maximize access to cutting issues that impact patients . policymakers and increase the likelihood of advocacy success . Members of Congress and COLLABORATIONS WITH administration officials, who have limited OTHER ALS ORGANIZATIONS resources and time, rely on coalitions to The ALS Association works in collaboration provide policy solutions developed through with a number of organizations, including consensus by diverse groups . the Muscular Dystrophy Association, I AM Belonging to a coalition in no way limits ALS, Team Gleason, and Les Turner ALS the ability of the ALS Association to act Foundation . independently . It does amplify access to policymakers and the influence of The ALS MEMBERSHIP ORGANIZATIONS Association in representing the concerns of 1. 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 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 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 . action . As new issues emerge, coalition efforts

3–15 2. Consortium for Citizens with Disabilities the repeal/replacement efforts on the (CCD): CCD is the largest coalition of Affordable Care Act (ACA) and favors a national organizations working together bipartisan approach to reforming the to advocate for federal public policy healthcare system . that ensures the self-determination, 6. Research!America: Research! America is independence, empowerment, integration the nation’s largest not-for-profit public and inclusion of children and adults with education and advocacy alliance working disabilities in all aspects of society . to make research to improve health a 3. Defense Health Research Consortium higher national priority . Relevant efforts (DHRC): Comprised of more than two focus on increasing funding for NIH, CDC, dozen diverse organizations, DHRC and the FDA . focuses on continued Congressional 7. Alliance for a Stronger FDA: The Alliance support for dozens of medical research for a Stronger FDA works to ensure annual programs at the Department of Defense . appropriations that will adequately fund These programs, funded annually in the the FDA’s essential missions . A science- Defense Appropriations Act, make up based and effective FDA supports the “Congressionally Directed Medical biomedical innovations as well as Research Programs” (CDMRPs), which approves safe and effective drugs and include the vital ALS Research Program medical devices for people living with ALS (ALSRP) . and all Americans . The Alliance unites a 4. National Organization for Rare Disorders broad and diverse spectrum of patient (NORD): A patient advocacy organization groups, consumer advocates, biomedical dedicated to individuals with rare researcher, health professionals and diseases and the organizations that companies . serve them . NORD, along with its more than 260 patient organization members INFORMAL COALITIONS IN WHICH THE ALS is committed to the identification, ASSOCIATION PARTICIPATES: treatment, and cure of rare disorders 1. Independence Through Enhancement of through education, advocacy, research, Medicare and Medicaid (ITEM) Coalition: and patient services . The ITEM Coalition is devoted to raising 5. Partnership to Protect Coverage: A awareness and building support for policies coalition of the nation’s leading patient that will enhance access to assistive advocacy organizations which have devices, technologies, and related services collectively opposed health care reform for people with disabilities and chronic legislation that would negatively impact conditions . The coalition is consumer-led patients . The coalition leadership on and includes a diverse set of disability

3–16 organizations, aging organizations, other MAPRx members represent every consumer groups, voluntary health segment of Medicare beneficiaries and associations, and non-profit provider join together to advocate on their behalf associations . The ALS Association is a and collaborate with national and state member of ITEM’s steering committee . policymakers to ensure all beneficiaries have access to needed medications . 2. Safeguarding Medicare Access to Respiratory Therapy (SMART) Coalition: 5. Alliance for Connected Care: Alliance The coalition was formed to protect for Connected Care was formed to access to non-invasive ventilators with create a statutory and regulatory support from respiratory therapists in environment in which every provider in the Medicare program . A majority of America is permitted to deliver and be people living with ALS require access to adequately compensated for providing ventilators at some point to maintain safe, high quality care using connected their quality of life . Working with national care (e .g . telemedicine and telehealth) associations of respiratory physicians at their discretion, regardless of care and therapists, the Association drafted delivery location or technological and lobbying for immediate passage modality . Members include leading of Safeguarding Medicare Access to companies from across the health care Respiratory Therapy (SMART) Act (H .R . and technology spectrum, representing 4945) . insurers, retail pharmacies, technology and telecommunications companies, and 3. Ad Hoc Group for Medical Research: Ad health care entrepreneurs . The Alliance Hoc Group for Medical Research is a is advised by a distinguished group of coalition of patient and voluntary health patient and provider advocacy groups, medical and scientific societies, academic and research organizations, 6. Partnership to Improve Patient Care and industry that support enhancing the (PIPC): PIPC is at the forefront of applying federal investment in the biomedical, principles of patient-centeredness to behavioral, and population-based research the nation’s health care system – from conducted and supported by the NIH . the generation of comparative clinical effectiveness research at the Patient- 4. Medicare Access for Patients Rx (MAPRx): Centered Outcomes Research Institute MAPRx is a coalition of patient, family (PCORI), to the translation of evidence caregiver and health professional into patient care in a manner that achieves organizations committed to safeguarding value to the patient . Having driven the the well-being of patients with chronic concepts of patient-centeredness and diseases and disabilities who rely on patient engagement in the conduct of Medicare Prescription Drug Coverage . research, PIPC looks forward to bringing

3–17 the voices of patients and people with pursuit of that goal, NCART works with disabilities to the discussion of how to consumers, clinicians, and physicians advance patient-centered principles along with federal, state and private throughout an evolving health care system . policy makers to establish and protect appropriate coverage, coding, supplier 7. National Coalition for Assistive & standards, and funding policies . Rehab Technology (NCART): NCART is a national organization of suppliers 8. Medical Expense Deduction (MedEx) and manufacturers of Complex Rehab Coalition: MedEX is an AARP-convened Technology (CRT) products and services coalition of more than 60 organizations used by individuals with significant that supports retention of the medical disabilities and chronic medical tax deduction that allows taxpayers to conditions . NCART seeks to ensure these claim an itemized deduction for high out- individuals have adequate access to CRT of-pocket medical expenses . products and supporting services . In

3–18 APPENDIX ABBREVIATED GLOSSARY OF SCIENTIFIC TERMS A chewing, and swallowing . Agonist: A drug that increases neurotransmitter activity by directly stimulating the nerve cell C receptors . Central Nervous System (CNS): The brain and spinal cord combined . ALS Functional Rating Scale – Revised (ALSFRS-R): A survey of questions that assesses Cerebrospinal Fluid (CSF): A clear fluid that the impact of als on activities of daily living . It is covers and protects the brain and spinal cord . often used as a primary outcome measure of als Chromosome: A visible carrier of the genetic clinical trials . information . One of the 20 building blocks of Amino Acid: Corticospinal Tract: The bundle of nerves that protein . reach from the motor area of the brain (see Antibody: A defense protein that binds to foreign cortex) to the spinal cord, connecting to the molecules to allow their elimination . nerves that go out to control the muscles . Antigen: A substance that is capable of causing CRISPR/Cas9: Genome editing technology that the production of antibodies . Antigens may or allows the permanent modification of genes may not lead to an allergic reaction . within an organism . By delivering the Cas9 nuclease bound to a synthetic guide RNA into a Antioxidant: A chemical compound or substance cell, the cell’s genome can be cut at the designed/ that inhibits oxidation . desired location . This allows existing genes to Assay: An investigative procedure (i .e ., experiment) either be removed or added in . CRISPR stands in the laboratory . for Clustered Regulatory Interspaced Short Ataxia: Loss of balance . Palindromic Repeats . Atrophy: The progressive loss of muscle mass, or wasting, caused by reduction in the size or D number of muscle cells . It is one of the later DNA: Deoxyribonucleic acid . Hereditary material symptoms of als . that encodes genetic information . Axon: The long, hairlike extension of a nerve cell Dysarthria: Impaired speech and language due that carries a message to the next nerve cell . to weakness or stiffness in the muscles used for speaking . B Dyskinesia: Abnormality or impairment of Blood-Brain Barrier (BBB): A protective barrier voluntary movement . formed by the blood vessels and glia of the brain . Dysregulation: Dysregulate: An impairment of a It prevents some substances in the blood from physiological regulatory mechanism; to cause a entering brain tissue . dysfunctional level of an activity or chemical in an Bradykinesia: Slowness of movement . organism by disrupting normal function . Bulbar Muscles: The muscles that control speech, Dysphagia: Difficulty in swallowing .

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

APP–3 study in which exposure to a potential therapy insulates the axon of some nerve cells to help or drug is assigned and being tested . It is used speed nerve transmission . It is important for to determine the effectiveness and safety of a proper function of the nervous system . potential treatment . Investigator: A person who carries out a scientific N study . A researcher . Nerves: Bundles of fibers that use electrical and chemical signals to transmit sensory and motor In Vitro: In an artificial environment outside the information from one body part to another . living organism, such as in a dish or test tube in the laboratory . Nervous System: The system of cells, tissues, and organs that regulates the body’s responses In Vivo: In a living organism, such as a mouse or to internal and external stimuli . In vertebrae it human . consists of the brain, spinal cord, nerves, ganglia, and parts of the receptor and effector organs . L Neuron: Neurons are the nerve cells which make Lower Motor Neurons: Nerve cells (motor neurons) up the central nervous system . They consist of a originating in the spinal cord that connect nucleus, a single axon which conveys electrical to muscles, conduct signals to allow muscle signals to other neurons and a host of dendrites movement . which deliver incoming signals . M Neurodegenerative: The progressive loss of the structure and function of the nervous system, Molecule: The smallest unit of a substance that especially neurons . can exist alone and retain the character of that substance . Neuroprotective: If an agent provides protection to any part of the body’s nervous system, it is said Motor Neuron: A neuron that conveys impulses to provide neuroprotection . initiating muscle contraction or glandular secretion . Neurotransmitters: Chemical substances that carry impulses from one nerve cell to another; Motor Neuron Disease (MND): A group of found in the space (synapse) that separates the disorders in which motor nerve cells (neurons) transmitting neuron’s terminal (axon) from the in the spinal cord and brain stem deteriorate receiving neuron’s terminal (dendrite) . and die . ALS is the most common motor neuron disease . O Muscle Atrophy: Loss of muscle fiber volume Observational Study: Type of trial in which characterized by a visible decrease in muscle size . enrolled participants are observed . Outcome This occurs because muscles no longer receive measures (i .e . measures of strength or function) impulses or signals from nerve cells . may be part of the observation . No treatment/ Mutation: A permanent change, a structural drug is given . It is often used to learn about trends alteration, in the DNA or RNA . Mutations can be of symptoms, the course of disease, and can caused by many factors, including environmental include biomarker studies . insults such as radiation and mutagenic chemicals . Oxidative Stress: Accumulation of destructive Mutations are sometimes attributed to random molecules called free radicals can lead to motor chance events . neuron death . Free radicals damage components Myelin: A fatty substance that surrounds and of the cells’ membranes, proteins or genetic

APP–4 material by “oxidizing” them – the same chemical components of muscles, skin, bones, and the body reaction that causes iron to rust . as a whole . Protein is also one of the three types of nutrients used as energy sources by the body . P Proteomics: The study and identification of the Phenotype: The observable characteristics of an proteins produced by the genetic instructions individual resulting from the expression of genes . carried by a cell . This may be directly observable (eye color) or Protocol: A precise and detailed plan for the study apparent only with specific tests (blood type) . of a biomedical problem or for a regimen of an Some phenotypes, such as the blood groups, are experimental therapy . completely determined by heredity, while others are readily altered by environmental agents . Q Pluripotent Stem Cells: Human pluripotent stem Qualitative: Relating to measuring or cells are a unique scientific and medical resource . measurement of the quality of something, such as They can develop into most of the specialized its size, appearance, etc . cells and tissues of the body, such as muscle cells, nerve cells, liver cells, and blood cells . They are Quantitative: Relating to measuring or self-renewing, making them readily available for measurement of the quantity (amount) of research and, potentially, for treatment purposes . something . Scientists derive these unique cells from human embryos, from fetal tissue, or from adult tissue R (in the case of induced pluripotent stem cells RNA: Ribonucleic acid . The primary function of (iPSCs)) . RNA is to act as a messenger carrying instructions Positron Emission Tomography (PET) Scan: A from DNA for controlling protein synthesis within computer-based imaging technique that provides a cell . a picture of the brain’s activity rather than its structure . The technique detects levels of injected S glucose labeled with a radioactive tracer . Sclerosis: A hardening within the nervous system, Potassium Channel: A type of ion channel that especially of the brain and spinal cord, resulting forms potassium-selective pores that span the cell from degeneration of nervous elements such as membrane, thereby helping transport potassium the myelin sheath . across the cell membrane . They are found in most Sialorrhea: Drooling . cell types and control a variety of cell functions . Spinal Cord: Part of the central nervous system Precision Medicine: A tailoring of medical extending from the base of the skull from the treatment to the individual characteristics of brain stem through the vertebrae of the spinal each person, while taking into account individual column . It carries information from the body’s variability in genes, environment, and lifestyle nerves to the brain and signals from the brain to for each person . In precision medicine programs, the body . researchers aim to learn as much as possible from Stem Cells: Cells that can differentiate into many each unique person living with ALS . different cell types when subjected to the right Protein: Proteins are large molecules required biochemical signals . Stem cells are a promising for the structure, function, and regulation of the new therapeutic approach to treating central body’s cells, tissues, and organs . Each protein nervous system disorders . The most versatile stem has unique functions . Proteins are essential

APP–5 cells, called pluripotent stem cells, are present in neurons) originating in the brain’s motor cortex the first days after an egg is fertilized by sperm . and running through the spinal cord . Researchers believe they can coax stem cells to become whatever tissues patients need . Stem V cells come from embryos, bone marrow, and Vector: The agent used (by researchers) to carry umbilical cords . View the stem cell glossary to new genes into cells . Plasmids currently are learn more . the vectors of choice, though viruses and other Stratify: To arrange or classify . bacteria are increasingly being used for this purpose . Superoxide Dismutase: An enzyme that destroys superoxide, which is a highly reactive form For more ALS vocabulary, visit The ALS Association of oxygen . With ALS, 20 percent of the total online, found at: http://www.alsa.org/research/ population of patients have mutations in the gene our-approach/glossary.html for copper/zinc superoxide dismutase type SOD1 . SOD1 normally breaks down free radicals, but mutant SOD1 is unable to perform this function . Synapse: A tiny gap between the ends of nerve fibers across which nerve impulses pass from one neuron to another; at the synapse, an impulse causes the release of a neurotransmitter, which diffuses across the gap and triggers an electrical impulse in the next neuron . Synergistic: Interaction or cooperation between two or more substances or organizations to produce a greater combined effect .

T Toxicity: The extent, quality or degree of being poisonous . Transgenic: An organism whose sperm or egg contains genetic material originally derived from an organism other than the parents or in addition to the parental genetic material . Translational Research: Studies that apply findings from basic science discovered in the lab to relevant disease therapies that enhance patient well-being . Trophic Factor: One of a class of proteins that help keep cells healthy .

U Upper Motor Neurons: Nerve cells (motor

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