Acamprosate for ALS

This trial tests the safety of acamprosate, a pill, in people with ALS—a disease that weakens muscles and affects physical function—who have a specific genetic mutation in the C9orf72 gene. The study will help researchers determine if this treatment is safe and how it might affect ALS symptoms. Participants must attend some in-person visits and complete others by phone over 32 weeks. This trial suits those with ALS and the C9orf72 gene mutation whose symptoms began less than two years ago. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

If you are taking riluzole, edaravone, or phenylbutyrate/TUDCA, you must be on a stable dose for at least 30 days before the screening visit or have stopped taking them at least 30 days before the baseline visit.

Previous studies have shown that acamprosate is safe. The FDA has approved it for other uses, indicating it is generally well-tolerated. These studies reported no major safety concerns. Participants took acamprosate without significant side effects. Although acamprosate is now being tested specifically for ALS, its past use in other conditions offers some reassurance about its safety.¹²³⁴⁵

Most treatments for ALS, like Riluzole and Edaravone, aim to slow disease progression by reducing nerve damage. But Acamprosate works differently, targeting neurotransmitter balance in the brain. Researchers are excited about this because it has a unique mechanism that might offer a new way to protect nerve cells. This could potentially enhance the quality of life for people with ALS by offering a fresh approach to managing symptoms.

Research suggests that acamprosate might help treat ALS, particularly for individuals with the C9orf72 gene mutation. Studies have found that acamprosate can protect motor neurons, the nerve cells responsible for muscle movement. This protective effect appeared in several cell samples from ALS patients. Although acamprosate typically treats alcohol dependence, its ability to protect nerve cells makes it a promising option for ALS treatment. However, further research is necessary to fully understand its impact on ALS symptoms. Participants in this trial will receive acamprosate to evaluate its safety profile throughout the study.¹²³⁵⁶