A Clinical Trial for Gene Therapy is Underway for Muscular Dystrophy!

Limb-Girdle Muscular Dystrophy or LGMD, is a form of Muscular Dystrophy caused by a mutation which disrupts production of the protein beta-sarcoglycan. There are currently no treatment options for LGMD and patients normally don’t live past age 30.

However, there’s renewed hope for this community thanks to positive results in pre-clinical examination of a new vector called MYO-101 for gene therapy.

The initial study of this vector used mouse models and after just one injection, beta-sarcoglycan was present in 95% of their muscle cells. In the treated mice, there was no immune response to the vector. Additionally, their activity was improved and their scarring was reduced. Strength and cardiac output were normalized after that one single dose. The results for both the safety and efficacy of this vector were highly promising.

It’s time for a clinical trial using real patients. If the clinical trial brings positive results, this could be life-changing for the muscular dystrophy community.

The Trial

Myonexus Therapeutics is the organization which came up with the vector MYO-101. They utilize the AAVrh.74 vector system to deliver it to patients. In collaboration with Nationwide’s Children Hospital, they have been studying this vector system for over a decade. But finally, after positive pre-clinical results with the mice, Myonexus was ready to start a clinical trial.

It officially began in October of this year and all of the participants will receive a dose of the vector before the end of 2018. That means we should have data on this study by early 2019.

Also involved in the project is Sarepta Therapeutics. Their VP of Gene Therapy, Dr. Louis Rodino-Klapac says-

“If successful in our clinical trial, this could become the first corrective treatment for LGMD2E, a merciless disease that has invariably robbed patients of strength and then life.”

You can read more about this study here, and stay tuned for updates on this trial’s results!


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