Focus on Amyotrophic Lateral Sclerosis

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.

bullhorn
Join Our Listserv
For the most current updates on NIH activities related to ALS, including upcoming activities, research opportunities, and NIH-funded science advances, sign up for our listserv.

To submit written feedback or ask questions about the Critical Path Institute- Rare Neurodegenerative Disease (CP-RND) and Food and Drug Administration (FDA) Initial Public Discussion on ALS Clinical Outcome Assessments, please email the Critical Path Institute- Rare Neurodegenerative Diseases, use "COA Feedback" in the Subject line. Submitted questions will be used to help guide the discussion for future public sessions.

News and Upcoming Events

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-specific Funding Opportunities

Featured Initiatives and Programs

Related Federal Programs

Proceedings and Outcomes

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.


NINDS Interdisciplinary Team Science RM1

The NINDS Interdisciplinary Team Science RM1 encourages interdisciplinary teams of experts that seek to cross technical and conceptual boundaries through collaboration to achieve ambitious goals for basic, translational, and clinical research questions within the mission of NINDS.


NINDS Division of Translational Research

The NINDS Division of Translational Research (DTR) provides funding and through grants, cooperative agreements, and contracts to academic and industry researchers to advance early-stage neurological technologies, devices, and therapeutic programs to industry adoption (i.e., investor funding and corporate partnerships). 


Ultra-rare Gene-based Therapy (URGenT) Program

The Ultra-rare Gene-based Therapy (URGenT) program will support the development of state-of-the-art gene-based therapies for ultra-rare neurological diseases, including familial forms of ALS.


NINDS Biomarker Program

The NINDS Biomarker Program 1) promotes rigorous biomarker identification and validation through milestone-driven funding opportunities, 2) maintains an updated website containing information about existing NINDS and NIH biomarker sample and data repository resources and 3) facilitates the development of future resources focused on bridging the gaps in the biomarker development pipeline.  In addition, the Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases program of NINDS DON supports studies to develop clinically validated biomarkers and clinical outcome assessment measures appropriate for use in upcoming clinical trials.


NINDS Small Business Program

The NINDS Small Business Program is committed to helping small business concerns commercialize their technologies through its grant funding, technical assistance program participation, and outreach at meetings. 


NINDS Office of Neural Exposome and Toxicology Research (ONETOX)

The NINDS Office of Neural Exposome and Toxicology Research (ONETOX) supports research and provides resources to advance knowledge of internal and external exposures that affect brain and nervous system health (Neural Exposome), leads research related to chemical threats, and provides resources that promote chemical safety.


NINDS Division of Clinical Research

The NINDS Division of Clinical Research (DCR) provides oversight for clinical trials to test the safety and efficacy of innovative treatments of neurological disorders and stroke, epidemiological studies of natural history, biomarker studies, and studies designed to elucidate the causes of neurological disorders. The Division of Clinical Research also develops new clinical science initiatives, ensures the proper level of patient safety monitoring, maintains the scientific integrity of clinical trials, and provides expertise in statistics and clinical trial design to the Institute and to clinical investigators. 


NIH Brain Research Through Advancing Innovative Neurotechnologies® (BRAIN) Initiative

The NIH BRAIN Initiative is accelerating the development and application of innovative technologies to enable researchers to produce a revolutionary new dynamic picture of the brain that, for the first time, shows how individual cells and complex neural circuits interact in both time and space. Long desired by researchers seeking new ways to treat, cure, and even prevent brain disorders, this picture will fill major gaps in our current knowledge and provide unprecedented opportunities for exploring exactly how the brain enables the human body to record, process, utilize, store, and retrieve vast quantities of information, all at the speed of thought. 


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
 

Image
3D illustration of neurons.

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.

     

    Image
    3D illustration of cells with DNAL helix on the background

    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.

     

    Image
    NeuroBioBank logo

    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.

    Image
    CP-RND logo

    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.

    Image
    Wheel infographic with icons representing 5 ALS strategic plan working group topics. Images are of four hands, hands holding a heart, medicine, brain and spinal cord, and a neuron

    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. 

     

    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

    Contacts
    Name Role Areas of Interest
    Amelie Gubitz, PhD Lead Program Director for ALS ALS basic, translational, and clinical research; ALS Public-Private Partnership
    Carol Taylor-Burds, PhD Program Director Biomarkers
    Christine Swanson-Fischer, PhD Program Director ALS Public-Private Partnership
    Emily Caporello, PhD Program Director Small business innovative research (SBIR) & small business technology transfer (STTR)
    Frank Shewmaker, PhD Program Director Frontotemporal dementia (FTD)
    Srikanth Ranganathan, PhD Program Director ALS Public-Private Partnership
    Lumy Sawaki-Adams, MD, PhD Program Director Clinical research, including natural history & clinical trials
    Amy Y. Tsou, M.D., M.Sc. Program Director Clinical research and outcomes
    Dina M. Lyon, M.S., R.N. Clinical Research Project Manager Neurodegenerative disease, neuromuscular disorders
    Rebekah Corlew, PhD Lived Experience Engagement Strategist

    Incorporating perspectives of people with lived experiences throughout the research process

    Elio Peraza, M.Sc. ALS Program Coordinator Planning, coordination, and management of internal/external ALS-related efforts