Amyotrophic lateral sclerosis (ALS) is a devastating, complex, and heterogeneous neurodegenerative disease that affects the neurons that control voluntary muscle movement. People with the disease become progressively weaker and lose their ability to walk, talk, move, swallow, and eventually breathe on their own, with most people dying within 3-5 years of symptom onset. But there is hope: scientific discoveries have resulted in the identification of multiple therapeutic targets, and three disease-modifying, plus one symptom-managing, ALS therapies have been approved by the Food and Drug Administration (FDA). Yet, to develop truly effective ALS treatments, we must address numerous challenges.
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ALS Lived Experience Community
NINDS seeks to include people with lived experiences of neurological disorders (PWLE) in the planning, execution, and communication of research.
Who are people with lived experience (PWLE)?
In the context of NINDS’ research, the lived experience community is comprised of individuals directly affected by neurological disorders. This includes those diagnosed and genetically at risk, as well as caregivers, loved ones, and those who have lost a family member to a neurological disease.
Opportunities for PWLE
To better represent and understand PWLE, NINDS invites the community to be active in the design and decision-making of research by joining working groups, planning committees, advisory groups, and other projects. Join our lived experience expert list to be contacted as opportunities arise.
Participating in Clinical Research
For more information and opportunities to participate in research studies, visit the NIH Clinical Trials page.
Current ALS-Relevant Funding Opportunities
- NINDS Division of Clinical Research
- NINDS Division of Translational Research
- Brain Research Through Advancing Innovative Neurotechnologies® (BRAIN) Initiative
Featured Initiatives and Programs
- National Academies of Sciences, Engineering, and Medicine (NASEM) study on ALS: Accelerating Treatments and Improving Quality of Life
- Accelerating Access to Critical Therapies for ALS Act (ACT for ALS)
Related Federal Programs
- Alzheimer’s Disease and Related Dementias
- Amyotrophic Lateral Sclerosis (ALS) Strategic Priorities
- Centers for Disease Control and Prevention/Agency for Toxic Substances and Disease Registry: National ALS Registry
- Food and Drug Administration: ACT for ALS implementation
- Department of Defense: Congressionally Directed Medical Research Program for ALS
Proceedings and Outcomes
- October 6, 2022: ALS Strategic Priorities Workshop
- November 6, 2023: ACT for ALS & ALS Strategic Priorities Community Update
- January 9, 2024: Technical Assistance Webinar recording for RFA-NS-24-029 Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)
- March 8, 2024: Recording: Rare Neurodegenerative Disease Efforts Under the ACT for ALS
- April 23, 2024: AMP ALS PWLE Forum: ALS Clinical Outcome Assessment- Initial Public Discussion
- June 18, 2024: National Academies of Sciences, Engineering, and Medicine- "Living with ALS" Consensus Study Report
- June 18, 2024: AMP ALS PWLE Forum: ALL ALS- Protocol Update and Outreach Efforts
- October 29, 2024: Webinar #1- Technical Assistance Webinar recording for RFA-NS-25-024: Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)
- December 3, 2024: Webinar #2-Technical Assistance Webinar recording for RFA-NS-25-024: Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)
Related Topics
NINDS Division of Neuroscience
The NINDS Division of Neuroscience (DON) supports research aimed at understanding fundamental mechanisms of development, structure and function of the nervous system in health and disease. DON utilizes a variety of grant mechanisms to support this research. A complete list of grant mechanisms can be found on the Types of Research Support page.
Rare Diseases Clinical Research Network (RDCRN)
The RDCRN program is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. Through the RDCRN consortia, physician scientists and their multidisciplinary teams work together with patient advocacy groups to study more than 200 rare diseases at sites across the nation. The Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) Consortium.
NINDS Division of Intramural Research
Investigators in the NINDS intramural program conduct research in the basic, translational, and clinical neurosciences at the NINDS Bethesda, Maryland laboratories.
Featured NINDS ALS Research Resources and Initiatives
In February 2023, the NINDS ALS Strategic Priorities(pdf, 1818 KB) were approved by the NINDS Advisory Council. These priorities were developed with input from a public Request for Information (RFI responses(pdf, 180 KB)) and working groups of scientists, clinicians, advocates, and people living with ALS.
The Accelerating Medicines Partnership® for Amyotrophic Lateral Sclerosis (AMP® ALS) is a public-private partnership aiming to build the largest data source for amyotrophic lateral sclerosis (ALS) and expedite the identification of biomarkers and clinical outcome assessments that would allow earlier diagnosis and accelerated drug development for people living with the disease. This collaboration with the Foundation for NIH (FNIH), Food and Drug Administration (FDA), and pharmaceutical, biotechnology, and nonprofit organizations will address the challenges of diagnosing, understanding, and treating the disease.
Critical Path (C-Path) for Rare Neurodegenerative Diseases (CP-RND) is a public-private collaboration focused on advancing the understanding of neurodegenerative diseases and fostering the development of treatments for ALS and other rare neurodegenerative diseases.
The NINDS Human Cell and Data Repository (NHCDR) includes fibroblasts and/or induced pluripotent stem cells (iPSC) for ALS, FTD, Alzheimer’s Disease, Ataxia-telangiectasia, Huntington’s Disease, Parkinson’s Disease, and healthy controls. Cell sources, including isogenic cell lines for current and new diseases covered by the NINDS are continuing to be added.
The iPSC Neurodegenerative Disease Initiative (iNDI) of the NIH Intramural Center for Alzheimer's and Related Dementias (CARD) is building a repository of genetically engineered cellular models of Alzheimer’s and related dementias, including FTD/ALS.
The NIH NeuroBioBank, supported by the NIMH, NINDS, and NICHD is a national resource for investigators utilizing human post-mortem brain tissue and related biospecimens for their research to understand conditions of the nervous system.
Estimates of Funding for Various Research, Condition, and Disease Categories
| Research/Disease Areas* | FY 2020 (Actual) | FY 2021 (Actual) | FY 2022 (Actual) | FY 2023 (Actual) |
|---|---|---|---|---|
| ALS | $107 | $120 | $151 | $219 |
*Dollars in millions and rounded
To learn more about this NIH Investment, please visit the Categorical Spending site and enter "ALS".
Contact Us
| Name | Role | Areas of Interest |
|---|---|---|
| Amelie Gubitz, Ph.D. | Lead Program Director for ALS | ALS basic, translational, and clinical research; ALS Public-Private Partnership |
| Carol Taylor-Burds, Ph.D. | Program Director | Biomarkers |
| Christine Swanson-Fischer, Ph.D. | Program Director | ALS Public-Private Partnership |
| Emily Caporello, Ph.D. | Program Director | Small business innovative research (SBIR) & small business technology transfer (STTR) |
| Frank Shewmaker, Ph.D. | Program Director | Frontotemporal dementia (FTD) |
| Srikanth Ranganathan, Ph.D. | Program Director | ALS Public-Private Partnership |
| Amy Y. Tsou, M.D., M.Sc. | Program Director | Clinical research and outcomes |
| Dina M. Lyon, M.Sc., R.N. | Clinical Research Project Manager | Neurodegenerative disease, neuromuscular disorders |
| Elio Peraza, M.Sc. | ALS Program Coordinator | Planning, coordination, and management of internal/external ALS-related efforts; engagement of people with lived experience |