Genetic Variants Impact on ALS Clinical Trials: James Berry, MD, MPH

The director of the Massachusetts General Hospital ALS Care Center detailed the role genetic backgrounds can have on outcomes within ALS clinical trials.

"We don’t want to create this altar of homogenous population that we sort of bow to because there’s some statistical benefit to it, while ignoring the critical importance of getting potential new therapies to people now, and allowing them to help the community, help themselves, help other people, by participating in trials. That balance is critical.”

There are a lot of unknowns surrounding the role that genetic variants play for patients with amyotrophic lateral sclerosis (ALS), but many researchers feel as though this is an area that needs further examination. To date, there has not been conclusive evidence that indicates which genetic mutations of the disease may progress earlier rather than later, or the degrees of severity to which they progress.

Similar to other basic sciences, the statistical power can be boosted by having a more homogenous group coming into the trial, according to James Berry, MD, MPH. Berry, director of the Massachusetts General Hospital ALS Care Center, feels as though there needs to be the right balance of homogenous populations while not forgetting some of the other more sporadic and unpredictive genetic backgrounds.

In an interview with NeurologyLive, Berry detailed some of the complexities within ALS clinical trials, adjusting for genetic backgrounds, and the pros and cons to focusing on a more homogenous gene population.