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2020-ALS-Mission-Toolkit Web.Pdf 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 .
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