New Treatments & Clinical Trials for Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis destroys the nerve cells that control voluntary movement, leading to progressive weakness, loss of speech and swallowing, and eventually respiratory failure. Most people live two to five years after diagnosis. Two drugs slow disease progression modestly, and riluzole and edaravone remain the primary FDA-approved treatments since the 1990s and 2017.

What's actually going on in research

Trials are testing drugs that target specific genetic mutations like SOD1, slow motor neuron death through multiple mechanisms, reduce inflammation in the brain and spinal cord, and support breathing and nutrition. Researchers are also studying antisense oligonucleotides for familial ALS, stem cell approaches, and combination therapies that address multiple disease pathways at once.

What to know before you search

Eligibility typically depends on time since diagnosis, rate of progression, lung function, and whether the person has a known genetic mutation.

What types of trials are currently open

• Disease-modifying trials — Testing drugs that aim to slow the loss of motor function, usually measured by decline on the ALS Functional Rating Scale over six to twelve months.
• Genetic mutation trials — Testing antisense drugs or gene therapy for people with specific inherited forms of ALS, like SOD1 or C9orf72 mutations.
• Symptom management trials — Testing treatments for muscle cramps, excess saliva, difficulty speaking, or emotional lability that accompany the disease.
• Biomarker studies — Collecting blood, spinal fluid, and imaging data to find better ways to track disease progression and predict response to treatment.
• Respiratory support trials — Testing devices and interventions to support breathing as respiratory muscles weaken, the leading cause of death in ALS.