Brian Wainger, MD, PhD: Momentum Within the ALS Treatment Landscape

"It’s a very exciting time for understanding sporadic ALS and advancing treatments for sporadic ALS. That includes better mechanistic understanding, better development of biomarkers, and innovation in clinical trial design.”

For patients with amyotrophic lateral sclerosis (ALS), the options to treat their symptoms have been few and far between. To date, the FDA has approved the drugs riluzole and edavarone (Radicava; MTP Pharma), which have shown to prolong survival and slow the decline in clinical assessment of daily functioning. Non-pharmacologic treatment options include psychical and speech therapy, nutritional support, and breathing support.

Despite the limited number of options, there has been a growing momentum within the community over the previous years, according to Brain Wainger, MD, PhD. Wainger, assistant professor, neurology and anesthesiology, Harvard Medical School, and physician scientist and principal investigator, Sean M. Healey and AMG Center for ALS, Massachusetts General Hospital, feels this growing hope can be said for both patients with familial and sporadic ALS.

He recently led a study evaluating ezogabine, an FDA-approved drug for epilepsy, in patients with ALS, and found the agent to decrease cortical and spinal motor neuron excitability, a variable commonly found among this patient population. Despite the positive results, the drug did not show efficacy on measures that included survival or disease progression, leading Wainger to believe it may not be a good potential option for patients with ALS.

He sat down with NeurologyLive to outline the increased momentum from within the space, and why there is reason to have hope going forward.