New Treatments & Clinical Trials for Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis is a progressive disease that kills motor neurons, the nerve cells that control voluntary muscles. About 5,000 people in the US are diagnosed each year. Current FDA-approved drugs — riluzole, edaravone, and the combination relyvrio — slow progression modestly but don't stop the disease.

What's actually going on in research

Trials are testing antisense therapies for genetic forms of ALS, stem cell treatments to support dying motor neurons, drugs that reduce inflammation in the brain and spinal cord, and treatments aimed at the protein clumps found in most ALS cases. Researchers are also studying whether targeting multiple disease pathways at once can produce larger benefits than single drugs.

What to know before you search

Eligibility typically depends on time since diagnosis, rate of progression, breathing function, and whether you have a known genetic mutation.

What types of trials are currently open

• Drug trials — Testing oral or intravenous drugs aimed at slowing motor neuron loss, often by reducing inflammation, protecting cells from stress, or clearing toxic proteins.
• Gene therapy trials — Testing antisense drugs or gene-silencing approaches for people with specific genetic mutations that cause familial ALS.
• Stem cell trials — Testing whether transplanted stem cells can protect remaining motor neurons and extend function.
• Device and support trials — Testing breathing devices, communication tools, or ways to manage symptoms like muscle cramps and excess saliva.
• Biomarker studies — Tracking markers in blood or spinal fluid to understand disease progression and predict who might respond to treatment.