FDA OKs clinical trial of DMD stem cell therapy MyoPAXon
Company to test treatment in adults with DMD who can’t walk
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The U.S. Food and Drug Administration (FDA) has given the green light for Myogenica to start a clinical trial testing its stem cell therapy MyoPAXon in people with Duchenne muscular dystrophy (DMD).
DMD, the most common type of muscular dystrophy, is caused by mutations in the gene that encodes the protein dystrophin. This protein normally acts to help prevent muscle damage; in DMD, the lack of functional dystrophin leads muscle cells to accumulate damage over time, ultimately driving disease symptoms like muscle weakness and wasting.
MyoPAXon is a line of healthy muscle stem cells generated from human umbilical cord cells. When muscle is injured, muscle stem cells are normally able to activate to help repair the damaged tissue, but this process is impaired in people with DMD. By supplying patients with healthy stem cells, MyoPAXon aims to allow their muscles to better heal and generate new muscle fibers.
Since the stem cells in MyoPAXon don’t carry a DMD-causing mutation, new muscle fibers generated from the transplanted cells would be expected to have functional dystrophin protein, so they’d be less susceptible to the type of damage that typically drives DMD.
“We believe stem cells have enormous potential as a tool for muscle regeneration in muscular dystrophy and the timing is perfect to move toward a human clinical trial of this type of therapy in Duchenne muscular dystrophy,” Peter Kang, MD, director of the University of Minnesota Medical School’s muscular dystrophy center and the trial’s leader, said in a press release.
Potential uses beyond DMD
The newly cleared clinical trial will test the safety and tolerability of MyoPAXon, as well as the therapy’s engraftment — that is, how well the cells are able to survive and make new muscle fibers after they are transplanted. In the trial, MyoPAXon cells will be injected into the muscles of DMD patients who aren’t able to walk.
If all goes well, researchers hope to then conduct a larger trial that would test the therapy’s effect on patients’ muscle function. The company noted that as MyoPAXon is not specific for any genetic mutation, it may one day be used as a treatment for other muscular dystrophies and degenerative conditions.
According to Myogenica, pilot studies of MyoPAXon’s safety have been successful, and studies in animals have shown efficacy and muscle regeneration.
Funding for the preclinical work that supported the trial application was provided in large part by Duchenne UK, which contributed more than $900,000 to support the research. Development of MyoPAXon has also been supported by organizations including the U.S. Department of Defense, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the care planning platform MyDirectives, and the Greg Marzolf Jr. Foundation, a philanthropic group focused on muscular dystrophy.
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