Jeffrey S. Chamberlain, PhD, on Regulating DMD Gene Therapy Clinical Trials


The professor and director of the Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center at University of Washington School of Medicine discussed working with the FDA toward a first disease-modifying therapy approval in Duchenne.

“It’s an exciting time, there's a lot of breakthroughs happening and new approaches to therapy, and the FDA is pretty receptive to getting as much input as they can to help them evaluate and grant approvals. They’re trying to speed things up and be able to grant early approval based on surrogate endpoints so that we can have an impact on these very serious disorders that have enormous impact on kids and other patients worldwide.”

Microdystrophin is used by clinical trials in the accelerated approval pathway assessing therapies for Duchenne muscular dystrophy (DMD) as a surrogate endpoint. A recent paper published in Human Gene Therapy reviewed its use as a surrogate endpoint in gene therapy trials in DMD. The authors, including first author Jeffrey S. Chamberlain, PhD, Professor and McCaw Chair, Muscular Dystrophy, and director, Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, concluded that it is a valid endpoint that correlates with clinical benefits in preclinical mouse studies.

CGTLive spoke with Chamberlain to learn more about challenges in regulating gene therapies for diseases like DMD. He discussed the importance of staying in communication with the FDA when working toward the approval of novel therapies.

Chamberlain J, Robb M, Braun S, et al. Micro-dystrophin expression as a surrogate endpoint for Duchenne muscular dystrophy clinical trials. Human Gene Therapy. Published online January 24, 2023.
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