Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act

Amyotrophic Lateral Sclerosis, or ALS, is a progressive and fatal neurodegenerative disorder, affecting around 30,000 people in the United States. Although some cases of ALS are inherited, the majority are sporadic, meaning that they occur with no clear family history. ALS is characterized by the degeneration and death of motor neurons in the brain and spinal cord, which control voluntary muscle movement and breathing, resulting in difficulty chewing, swallowing, speaking, and breathing. There are currently no therapies that reverse damage to motor neurons or cure ALS. The goals of NINDS’s ALS research are to understand the cellular mechanisms involved in the development and progression of the disease, investigate the influence of genetics and other potential risk factors, identify biomarkers, and develop new treatments. Building on these efforts, the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS; P.L. 117-79), signed into law on December 23, 2021, directed the NIH to establish a research grant program that leverages data from the Food and Drug Administration (FDA)’s expanded access to experimental drugs pathway for individuals who are not eligible for ALS clinical trials. The ACT for ALS also called for the establishment of a public private partnership (PPP) to foster the development of treatments for ALS and other rare neurodegenerative diseases. People with lived experience of ALS (PWLE) are integral partners in implementing the ACT for ALS. Their perspectives, included throughout all outputs of the Act, help shape research priorities to ensure real-world needs and patient-centered outcomes are addressed. ACT for ALS is supplementary to NIH- and NINDS-funded investigator-initiated ALS research

Interstate, Intrastate, State (includes District of Columbia, public institutions of higher education and hospitals), Local (includes State-designated lndian Tribes, excludes institutions of higher education and hospitals, Sponsored organization, Public nonprofit institution/organization (includes institutions of higher education and hospitals), Other public institution/organization, U.S. Territories and possessions, U.S. Territories and possessions (includes institutions of higher education and hospitals), Individual/Family, Minority group, Specialized group (e.g. health professionals, students, veterans), Small business (less than 500 employees), Profit organization, Private nonprofit institution/organization (includes institutions of higher education and hospitals), Other private institutions/organizations, State Research Grants: Any public, private, nonprofit, or for-profit institution is eligible to apply. For-profit institutions are not eligible for Institutional National Research Service Awards but are eligible for Individual NRSAs. All proposals are reviewed for scientific merit, for evaluation of the qualifications of the investigators, for adequacy of the research environment, and for significance of the problem. Approved proposals compete for available funds. All Career Development Program awardees, with the exception of awardees of the Pathway to Independence Award (K99/R00), must be citizens or have been admitted to the United States for permanent residence. Candidates must be nominated for the program by a nonfederal public or private nonprofit institution located in the United States, its possessions or territories. To be eligible, postdoctoral NRSA trainees or fellows must have a professional or scientific degree (M.D., Ph.D., D.D.S., D.O., D.V.M., Sc.D., D. Eng., or equivalent domestic or foreign degree). SBIR grants can be awarded only to domestic small businesses (entities that are independently owned and operated for profit, are not dominant in the field in which research is proposed, and have no more than 500 employees). Primary employment (more than one-half time) of the principal investigator must be with the small business at the time of award and during the conduct of the proposed project. In both Phase I and Phase II, the entire research must be performed in the United States. To be eligible for funding, a grant application must be approved for scientific merit and program relevance by a scientific review group and a national advisory council. STTR grants can be awarded only to domestic small business concerns (entities that are independently owned and operated for profit, are not dominant in the field in which research is proposed and have no more than 500 employees) which "partner" with a research institution in cooperative research and development. At least 40 percent of the project is to be performed by the small business concern and at least 30 percent by the research institution. In both Phase I and Phase II, the research must be performed in the U.S. and its possessions. To be eligible for funding, a grant application must be approved for scientific merit and program relevance by a scientific review group and a national advisory council.

Research Grant, Cooperative Agreement, Training, Fellowship and SBIR/STTR applications are reviewed initially by technical panels, composed of scientific authorities, and by the National Advisory Neurological Disorders and Stroke Council composed of leaders in medical science, education, and public affairs. Approved applications will compete on a merit basis for available funds. Formal award notices are transmitted to the grantee or awardee.