Duchenne muscular dystrophy is a progressive genetic disorder causing severe disability and early death. Promising animal studies and earlier investigations led to a trial of intravenous stem cell therapy in an 11-year-old boy, described in the winter issue of the Journal of American Physicians and Surgeons by Darah Mohammed Pourakbar, M.S., Kipp Van Camp, D.O., and Cameron D. Knackstedt, D.O.
The patient underwent a 90-day treatment protocol involving three intravenous infusions of mesenchymal stem cells and exosomes derived from placenta and umbilical cord, mixed with platelet-rich plasma. The patient showed improvement in several measures on a musculoskeletal assessment test and stability on cognitive testing. Both the patient and his parents reported positive improvement in his overall balance and dexterity from their daily observations, and they elected to continue treatments after the protocol was completed.
The treatment appeared to be effective and safe. Future studies should involve blinded evaluations of treatment in a large number of patients over a prolonged period, with special attention to comparing the available delivery methods for the stem cells.
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