Millions Allocated for ALS Research and Treatment Access, GAO Report Analyzes

A recent report from the U.S. Government Accountability Office (GAO) details significant federal investment in the research and accessibility of treatments for Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease. The GAO’s findings indicate that between fiscal years 2022 and 2025, the National Institutes of Health (NIH) and the Food and Drug Administration (FDA) collectively awarded approximately $276 million to implement the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS Act). This funding aims to advance scientific understanding of ALS and other rare neurodegenerative diseases, as well as to improve access to experimental treatments for individuals diagnosed with ALS.

The bulk of the allocated funds, specifically 45 percent, were channeled through NIH grants. These grants are designed to support two key areas: providing individuals with ALS access to investigational drugs—treatments not yet approved for general marketing—and furthering related research. Another substantial portion, also 45 percent of the total, was dedicated to a public-private partnership focused on rare neurodegenerative disease research, with contributions from both the NIH and FDA. The remaining 10 percent of the funding was awarded by the FDA through grants and contracts to expand scientific knowledge concerning ALS and other rare neurodegenerative conditions, and to aid in the clinical development of potential therapies. It is anticipated that approximately 750 individuals with ALS will benefit from access to investigational drugs through the NIH-funded grant programs.

The GAO report also identified challenges encountered by the NIH and FDA in the implementation of the ACT for ALS Act, particularly during the initial fiscal year of 2022. For instance, limited timeframes for grant applications in FY2022 were attributed to appropriations becoming available midway through the fiscal year. In response, NIH has since adopted a strategy of issuing requests for grant applications prior to the enactment of appropriations in subsequent years. However, some obstacles, such as the absence of direct appropriations to the FDA for its specific priorities within the public-private partnership concerning other rare neurodegenerative diseases, were outside of the agencies’ direct control.

Despite these implementation hurdles, the report highlights positive outcomes stemming from the federal funding. Stakeholder interviews, existing research literature, and data from the NIH suggest benefits such as an increase in both the number and geographical distribution of clinic sites offering access to investigational ALS drugs. This expansion is expected to make experimental treatments more accessible to patients across the nation, including those in more remote locations. Furthermore, the funding has contributed to an increase in researcher collaboration, fostering a more interconnected and efficient research environment. While the full impact of this federally funded ALS research is still unfolding, as much of the work is ongoing, anticipated benefits include the generation of valuable data on ALS, which is expected to meet high quality standards and be made available to other researchers. This data is seen as crucial for addressing existing research gaps and advancing the understanding and treatment of ALS and other rare neurodegenerative diseases.

The GAO conducted its review in response to a provision within the ACT for ALS Act that mandates reporting on the funding allocated by NIH and FDA. The study involved an examination of relevant laws, congressional reports, agency documentation, and grant applications and progress reports. Additionally, the GAO reviewed data from NIH databases on funded research studies and analyzed relevant published research from January 2019 through September 2025. Interviews with agency officials and a diverse group of 21 stakeholders, including patient advocacy groups, researchers, drug sponsors, and individuals with ALS and their caregivers, provided varied perspectives on the research efforts.

Article by Mel Anara, based upon information from the U.S. Government Accountability Office.

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