Jeffrey S. Chamberlain, PhD, on Reviewing Micro-Dystrophin as a Surrogate Endpoint in DMD

“The goal really was just to do a critical evaluation of the existing data from animal models and from the extensive clinical trials that are going on to make the argument that we have an update on predicting clinical benefit. We'd like the FDA to hopefully evaluate all that data, and we think they will come to the same conclusion.”

A recent paper published in Human Gene Therapy reviewed the use micro-dystrophin as a surrogate endpoint for an accelerated approval pathway of Duchenne muscular dystrophy gene therapy trials. 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, and colleagues, concluded that it is a valid endpoint that correlates with clinical benefits in preclinical mouse studies.

CGTLive spoke with Chamberlain to learn more about the rationale for the paper. Hediscussed the precedent of approving exon-skipping therapies for DMD using micro-dystrophin as a surrogate endpoint and how the same should apply for gene therapies.

REFERENCE

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. http://doi.org/10.1089/hum.2022.190