New Treatments & Clinical Trials for ALS

ALS (amyotrophic lateral sclerosis) is a disease where nerve cells controlling voluntary movement gradually die. About 5,000 people are diagnosed with ALS in the US each year. Until recently, treatment focused on riluzole and edaravone, which modestly slow progression, plus extensive supportive care for breathing, swallowing, and mobility.

What's actually going on in research

Trials are testing drugs that target specific genetic mutations (like SOD1), approaches to reduce inflammation in the brain and spinal cord, antisense therapies that silence disease-causing genes, and combinations of existing drugs. Researchers are also studying stem cells, gene therapy, and ways to protect motor neurons from dying. Much attention is on catching the disease earlier and identifying biomarkers that predict progression.

What to know before you search

Eligibility typically depends on time since diagnosis (often within 18-24 months), breathing capacity measured by forced vital capacity, and for genetic trials, confirmed presence of a specific mutation.

What types of trials are currently open

• Treatment trials — Testing new drugs to slow disease progression, often comparing them to riluzole or placebo in people with recent ALS diagnosis.
• Gene therapy trials — Studies of therapies targeting specific genetic mutations, available only to people with that mutation confirmed by genetic testing.
• Stem cell trials — Testing whether stem cells injected into the spinal cord or given intravenously can protect motor neurons or replace damaged ones.
• Device and care trials — Studies of breathing support devices, communication tools, feeding methods, and physical therapy approaches.
• Observational studies — Following people with ALS over time to understand how the disease progresses and what factors affect survival and quality of life.