Ways to Optimize Trials for ALS Drug Development: Richard Bedlack, MD, PhD, MS

WATCH TIME: 5 minutes

"We’ve got to be smarter about how we do trials. If we’re going to use outcome measure like the ALS Functional Rating Scale, a measure of disability, we’ve got to select the right patients for the trial. They have to be people who are early in their disease course."

In the years that followed the FDA approval of riluzole (Rilutek) in 1995, over 60 molecules were investigated as possible treatment for amyotrophic lateral sclerosis (ALS), with an overwhelming majority of human trials failing to demonstrate clinical efficacy. The FDA’s first approval since riluzole came in 2017, with the addition of edaravone (Radicava; MT Pharma), one of the few agents to show a slowing of disease progression. More recently, in 2022, Amylyx Pharmaceuticals’ therapy AMX0035 (Relyvrio), a coformulation of sodium phenylbutyrate/taurursodiol, gained FDA greenlight as the third agent on the market to slow disease progression.

The expansion in treatment options over the past decade has spoken to the advances in translational research and increased interest from industry leaders to one day hopefully cure this disease. Earlier this year, the ALS community saw its first genetic-targeted therapy come to market with the approval of tofersen (Qalsody; Biogen), an agent designed for SOD1 mutation-mediated forms of the disease. While it’s encouraging to see drug development success, there are still ways in which the clinical community can more efficiently test agents, says Richard Bedlack, MD, PhD, MS.

Bedlack director of the Duke ALS Clinic at Duke Health, gave a presentation at the 2023 International Congress on the Future of Neurology (IFN), held September 22-23 in Jersey City, New Jersey, on the advances in ALS and where we currently stand. Following his presentation, Bedlack sat down with NeurologyLive® to discuss ways to improve efficiency in ALS trials. He spoke specifically about the need for patients early in their disease, the questions surrounding widely accepted measures like the ALS Functional Rating Scale, and whether neurofilament light outcomes will become more commonly used following the approval of tofersen.