Dr. Steve Moore: University of Iowa Department of Pathology Central Site for Muscle Biopsy Evaluation in Sarepta Muscular Dystrophy Clinical Trials

September 22, 2016

‚ÄčDr. Steve Moore: University of Iowa Department of Pathology Central Site for Muscle Biopsy Evaluation in Sarepta Muscular Dystrophy Clinical Trials

Dr. Steve Moore recently appeared on TV9 in a story covering the accelerated approval for a Duchenne muscular dystrophy treatment. This approval has also received much wider publicity.

"It is very early in the game for this drug," Dr. Moore said. "Clearing the FDA hurdles to get accelerated approval was quite a long endeavor by the company, Sarepta Therapeutics."

The University of Iowa is involved in ongoing phase 2 and phase 3 clinical trials with the approved drug. Through Dr. Moore's leadership, the Department of Pathology at Iowa is the central site for the muscle biopsies to be sectioned and stored. UI is one of two sites in the USA doing the muscle biopsies, so we also have a critical role in getting the biopsies frozen appropriately. Histotechnologists Terese Nelson and Melissa Jans are key individuals working with the biopsies. Starting in 2017, the project will involve an extensive amount of sectioning for immunofluorescence evaluation and for PCR and western blotting studies. The department will function as a central resource for these studies.

A new phase 3 clinical trial is beginning next month with another exon skipping drug that will treat boys with DMD gene mutations slightly different from the boys being treated with eteplirsen, the drug just approved by FDA. The Department of Pathology will again be the central site for biopsy storage and processing. This trial will also have European centers participating. So, our department will be receiving frozen muscle biopsies shipped here from Europe.