Is a treatment for Duchenne muscular dystrophy likely in the near future? It is, if the stem cell remedy University of Minnesota Professor Rita Perlingeiro developed for mice with the ailment works on humans as well. Muscular dystrophy is a genetic disorder, for which there is no effective treatment. It causes muscles to weaken and deteriorate and it is a disease that afflicts mainly children, many of whom succumb during their teen years.
Stem Cells Treat Mice With Muscular Dystrophy
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According to Brandon Largent, writing March 6 in the Minnesota Daily, Perlingeiro’s team reprogrammed skin cells from some dystrophic mice’s tails into stem cells that are capable of morphing into any cell type. They then modified the stem cells to induce them to turn into muscle cells. When the reprogrammed cells were transplanted back into the muscles of the donor dystrophic mice, the mice showed increased long-term muscular development, Perlingeiro said.
While the mice were not cured of muscular dystrophy, Perlingeiro told Largent that their muscle function improved greatly and she was confident the methods could have the same results in humans. Perlingeiro has long experience working on muscular dystrophy research. Last year she oversaw the first successful use of human stem cells in treating the disease.
Largent quoted Jakub Tolas, director of the University’s Stem Cell Institute saying, “It’s game-changing research, in my opinion, in the field of muscular dystrophy.” Tolar said Perlingeiro’s method has the potential to reach human clinical trials, which would make it the first muscular dystrophy treatment to do so. Largent also quoted Perlingeiro as noting that her research provides proof-of-principle for the possibility of treating the disorder and could translate to testing in human cells from dystrophic patients. Her research, she said, provides proof-of-principle for the possibility of treating the disorder and could translate to testing in human cells from dystrophic patients. The study was published online in the journal Nature Communications.
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This is a fascinating development. In my practice we've seen similar outcomes with the revised protocol. The key differentiator seems to be patient selection criteria. Has anyone else noticed the correlation with BMI thresholds?
Great point. I'd push back slightly on the conclusion, the sample size in the cited study is too small to draw population-level inferences. That said, the directional signal is compelling and worth a larger RCT.
We implemented a similar approach last year. Early results are promising but we're still gathering 12-month follow-up data. Happy to share our protocol if anyone is interested.
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