MyoPAXon, a new stem cell therapy, will be tested in patients with Duchenne muscular dystrophy (DMD) after showing encouraging safety and benefit in animal studies, according to a study published recently in Molecular Therapy.
MyoPAXon is designed to replace damaged muscle cells with healthy ones. The therapy uses lab-grown human stem cells (called human induced pluripotent stem cells, or iPSCs) engineered to express a key factor called PAX7, which helps muscle stem cells develop and renew.
“MyoPAXon will be the first iPSC-derived skeletal muscle product to be tested in a clinical trial, and the first product designed to contribute to the tissue stem cell compartment,” noted the study’s authors.
In mouse models of DMD and another muscle disease, transplantation of these cells led to new muscle fibers, stronger muscle force and lasting engraftment for up to 13 months. Similar results were observed in primates, where the treatment integrated safely into muscle tissue without signs of inflammation.
Before moving to human testing, researchers confirmed the safety of MyoPAXon in rigorous laboratory studies. No tumors or harmful cell overgrowth were found.
The U.S. Food and Drug Administration has now approved a first-in-human trial led by the University of Minnesota to test local injections of MyoPAXon in nonambulatory adults with DMD. Participants will also receive short-term immune-suppressing medicine to help the transplanted cells survive.
Read more about therapies for DMD
Patients and families should know that MyoPAXon is still experimental. The current trial is focused on safety, not yet on improving movement or strength. Treating the many muscles affected by DMD will require very large numbers of cells, and it is not yet clear how well the therapy will work in humans. Long-term immune suppression or other strategies may also be needed.
Even so, experts view the launch of this study as a milestone. Restoring even limited muscle function could mean greater independence and better quality of life for patients living with DMD. For now, MyoPAXon offers a promising step toward the possibility of meaningful treatment.
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