University of Minnesota researchers have effectively treated muscular dystrophy in mice using human stem cells. This is the first time human stem cells have been shown to be effective in the treatment of muscular dystrophy.
Researchers used a new process that makes the production of human muscle cells from stem cells more efficient and effective.
The research was published May 3 in the journal Cell Stem Cell, outlining the strategy for developing a rapidly dividing population of muscle-forming cells.
The U of M says this research is significant because there has been a notable lag in translating studies using stem cells from mice into therapeutically-relevant studies involving human stem cells. This development should speed-up the creation of cell therapies or clinical trials for humans
"One of the biggest barriers to the development of cell-based therapies for neuromuscular disorders like muscular dystrophy has been obtaining sufficient muscle progenitor cells to produce a therapeutically effective response," said principal investigator Rita Perlingeiro, Ph.D. "Our results demonstrate that it is indeed possible and sets the stage for the development of a clinically meaningful treatment approach."
"This research is a phenomenal breakthrough," said Dr. John Wagner, scientific director of clinical research at the University of Minnesota's Stem Cell Institute. "Dr. Perlingeiro and her collaborators have overcome one of the most significant obstacles to moving stem cell therapies into the treatment of children with life threatening muscular dystrophy"
The study was funded through grants from the National Institute for Arthritis and Musculoskeletal and Skin Diseases, National Institute for Child Health and Development and the Dr. Bob and Jean Smith Foundation.