Masitinib is an experimental oral therapy being investigated in clinical trials as a potential treatment for amyotrophic lateral sclerosis (ALS). The medication is designed to reduce the activity of immune cells thought to be involved in ALS progression by blocking specific proteins called tyrosine kinases. In clinical trials, the therapy helped to slow functional decline and significantly extended survival among patients.
AB Science, the company developing masitinib, is sponsoring a Phase 3 clinical trial to explore the efficacy of masitinib as an add-on therapy for amyotrophic lateral sclerosis (ALS). At present, it is too early to determine if or when the therapy might be up for approval by the U.S. Food and Drug Administration. The therapy now is being considered for conditional approval in Europe and in Canada.
The most common side effects reported in amyotrophic lateral sclerosis clinical trials of masitinib included rash, tissue swelling, respiratory failure, nausea, diarrhea, and anxiety. Yet, because the drug is still in clinical testing, its full safety profile may not be fully known. Patients taking masitinib should discuss the potential side effects of the medication with their healthcare provider.
The interaction between masitinib and alcohol has not been studied in-depth. It’s recommended patients talk to their healthcare team about whether and how much is safe for them to drink while taking any medication.
In a Phase 2/3 clinical trial in people with amyotrophic lateral sclerosis (ALS), a significant difference in disease progression rates was evident between masitinib and a placebo after about a year of treatment. However, patients given the therapy still experienced disease progression — just more slowly than what was seen with the placebo.