Clinical Trial for Duchenne Muscular Dystrophy Gene Therapy Restarts After Hold Lifted

According to a story from statnews.com, the drug developer Sarepta has recently announced the its clinical trial testing an experimental gene therapy has resumed after the FDA lifted the hold. This gene therapy is being tested as a treatment for Duchenne muscular dystrophy. The hold on the trial was announced by the FDA in July.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a neuromuscular disease, and it is one of the more severe types of muscular dystrophy. It is characterized by progressive muscle weakness that usually begins around age four and worsens quickly. As an X-linked genetic disease, boys are mostly affected, with girls only occasionally displaying mild symptoms. The disease is caused by mutations of the dystrophin gene. Symptoms of Duchenne muscular dystrophy include falling, abnormal walking posture, eventual loss of walking ability, muscle fiber deformities, intellectual disability (not in all cases), enlargement of the tongue and calf muscles, skeletal deformities, muscle atrophy, heart abnormalities, and difficulty with breathing. Treatment includes a variety of medications and therapies that can help alleviate symptoms and slow disease progression. Lifespan is usually into the thirties with good care. Better treatments for this disease are urgently needed. To learn more about Duchenne muscular dystrophy, click here.

Contaminated Samples?

The clinical hold was originally placed on the trial because of the small pieces of DNA that were turning up in samples of the therapy. Thankfully, these contaminated samples were not used on any patients. Sarepta announced in response that it would be using a higher quality type of plasmid for production in future batches. The gene therapy intends to introduce microdystrophin to the patients, which is essentially a smaller version of dystrophin gene which is nonfunctional in Duchenne muscular dystrophy patients.

Thankfully, the hold did not cause a huge delay in the timing for the trial. While the outcomes of this trial have yet to be determined, the next step for Sarepta will be running a clinical trial that is officially registered with the US Food and Drug Administration (FDA). They hope to begin this process before the end of the year.


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