A report released by the U.S. Government Accountability Office (GAO) in February of 2026 titled “Rare Diseases: Funding for Rare Neurodegenerative Disease Research and Access to ALS Investigational Drugs” outlines funding spent on research on ALS and neurological diseases from 2022 to 2025.
The U.S. Government funded $276 million through grants from the National Institute of Health (NIH), the Food and Drug Administration (FDA), and public and private partnerships for research funding into experimental treatments for Amyotrophic Lateral Sclerosis (ALS) and neurological diseases.
ALS is a neurodegenerative disease that damages motor neurons, the nerve cells that control voluntary muscle movement. As these neurons die, muscles weaken and waste away, leading to progressive loss of mobility, speech, and breathing ability. ALS is fast-moving, and because of the length of time involved for diagnosis, many miss the opportunity for clinical trials. In response congress passed the Accelerating Access to Critical Therapies for ALS Act in 2021 to help expand experimental drug access for those who can not join drug trials.
Funding was split up into three main categories:
The largest portion went to grants managed by the NIH, $125.3 million. These grants support “expanded access” programs that allow patients to receive investigational drugs outside formal trials. Five major programs received funding, including therapies for CNM-Au8, pridopidine, rapamycin-based treatments, and ibudilast.
Public-Private Partnership for Rare Neurodegenerative Research (NIH + FDA), $124.4 million focused on rare neurological diseases, sharing research infrastructure, data platforms, and collaborative studies designed to speed up early-stage drug development.
Standalone grants from the FDA for rare neurological diseases totaling $26.5 million. These funds went to awarding contracts, biomarker development, regulatory science, and early therapeutic research for ALS and related disorders. Funding went towards basic scientific research, the development of new therapies, regulatory science tools, and groundwork to support longer-term clinical development for ALS and other rare neurodegenerative diseases.
The funding for these programs went toward investigational programs for 750 people with ALS, with 46 specialized clinics across 25 states, Washington, D.C., and Puerto Rico, with enrollment in these programs ranging from 32 to 203 participants, including remote enrollment that allowed participants from all 50 states. Giving people access to these programs without the financial burdens of traveling and temporary relocation.
“This represents a real shift in how ALS care and research intersect,” according to the report.
It also highlights early benefits and improvements from interviews with researchers, clinicians, advocacy groups, and caregivers, pointing to: more clinics able to offer experimental therapies, better coordination between research sites, increased participants from areas that are underserved and rural communities, and noting better data collection efforts thanks to improved access. However, the report also emphasizes that it is still too early to measure long-term impact, and most of these programs are still ongoing, with few definitive results reported so far.
There were several barriers with money arriving late in 2022, impacting how the NIH was able to award initial expanded access grants, posting future grant opportunities earlier, before funds were available. Also, the FDA didn’t receive money that was earmarked for its priorities on rare neurodegenerative disease research, which impacted how the agency could react and adapt to changes.
Many patients living with ALS are excluded from trials because of age, disease stage, breathing capacity, or location. These experimental therapies can offer hope and sometimes a last chance. The GAO report maintains a need for a balance between expanded access and rigorous data collection, noting that expanded access only works if the data received backs up the results, which directly impacts the approval process of many experimental therapies and drug trials.
While the GAO did not issue formal recommendations, it underlined the importance of ongoing monitoring. Notably, it indicates that the federal government has, for the first time, established a national system to support people with ALS who are not part of traditional research channels.
This report shows investment in ALS investigational drug access and rare neurodegenerative research, highlights the importance of expanded access as a foundation for future progress, and some of the efforts being made are having some traction. However, many will point out that while the funding is out there, a push for more funding is needed, arguing that even current high federal funding levels don’t meet the scale of ALS’s impact or research needs, much like funding for other diseases like Parkinson’s and Huntington’s Disease. Experts warn that unstable budgets, proposed cuts, and unmet research needs continue to slow progress toward effective therapies and a cure.
