This ALS Awareness Month, lawmakers and regulators face a pivotal moment in the fight against ALS. For the first time in history - following decades of stagnancy - breakthroughs in ALS science, policy, and coordination are beginning to align. But we need swift action from Congress and the FDA to continue pushing urgently so that progress can benefit those who need it most. I AM ALS - the largest national movement to end ALS - has been encouraged by action from our champions in Congress. We are hopeful that leaders in Washington will pass the Accelerating Access to Critical Therapies for ALS Reauthorization Act of 2026 (ACT for ALS) and protect the FDA’s evolving efforts to accelerate ALS drug development and treatment access.
Nearly 35,000 people in the United States are living with ALS today and most survive only three to five years after diagnosis. Treatment options remain limited, leaving patients with few options beyond symptom management and supportive care. Over the past few decades, diseases with significant unmet need - such as cancer and HIV - have seen a steady stream of approvals. ALS, by contrast, still has fewer than a handful of approved treatment options, despite the fast progression and terminal nature of the condition. ALS can take a person’s life in less time than it takes most drugs to complete the path to approval. Patients and families cannot afford for the system to keep moving at this pace.
Congress took an important step five years ago by passing ACT for ALS, which authorized $100 million annually through 2026 to accelerate drug development, expand access to promising experimental therapies, and strengthen public-private partnerships aimed at improving patient outcomes. That funding helped build an ALS research and clinical infrastructure that enabled new treatments to be tested more efficiently and reach a broader group of patients.
But that progress is now at risk. If Congress does not renew ACT for ALS before September 30, 2026, the funding will expire, possibly disrupting ongoing work or halting the treatments people living with ALS are counting on. Allowing the law to lapse would undermine clinical progress and jeopardize patient access to emerging therapies.
While significant unmet need remains, hope is still on the horizon. In just the past few years, the FDA approved a treatment for patients living with a rare genetic form of ALS, giving some families more time and better function than was thought possible even a decade ago. Experimental therapies are advancing toward potential accelerated FDA review based on promising biomarker data. Together, these developments signal that new treatments could reach patients faster than ever before.
Scientific progress, regulatory innovation, and bipartisan leadership do not often move in step. In ALS, they finally are, creating a rare and important opportunity to change the trajectory of this disease. Now is the time to build on that momentum. Congress should pass the ACT for ALS Reauthorization Act before the September 30 deadline, and the FDA should continue advancing its framework for novel surrogate endpoints in neurodegenerative disease. Together, these actions can help sustain the unprecedented progress underway in ALS research and drug development, and bring new hope to patients and families waiting for effective treatments.
