Vita Therapeutics’ cell-based therapy update

The company switches up its strategy and plans to develop muscle stem cells that evade the immune system

by June Kinoshita, FSHD Society

Hot off the press is this news that Vita Therapeutics shared at the Greater Philadelphia chapter meeting on March 2. The Baltimore-based company announced last year that it was developing muscle stem cell lines from individuals with FSHD. These cells would be treated in the lab to correct the genetic mechanism that causes FSHD. The treated cells would be transplanted into patients, where they could rebuild healthy muscles. Because the cells would originate from the patient, they would not trigger an immune response when transplanted back into the same individual.

A major challenge with this customized approach is that it would take a full year and $1.2 million to grow enough cells to treat a single patient. Now Vita has a better strategy, engineering muscle stem cells that don’t attract an immune reaction at all. The trick is to engineer the cells so that they do not express specific proteins on their surface that trigger an immune system attack. What this means is that anyone, not just people with FSHD, can donate the cells from which the muscle stem cells would be made. And anyone can receive the therapeutic cells. This approach promises to make this therapy available to many more people with muscle-damaging conditions while bringing down the cost.

Watch our video below to learn more from Drs. Steve Brooks, MD, senior vice-president of clinical affairs, and Mahendra Rao, MD PhD, chief scientific officer at Vita Therapeutics.