Stem cells used to treat muscular dystrophy in mice

Muscular dystrophy is a particularly tragic—and lethal—disease that currently has no effective therapy. But researchers at the University of Minnesota have made some progress in using stem cells to treat a form of muscular dystrophy in mice.

Muscular dystrophy can occur in adults but is most common in children and causes the person’s muscles to atrophy. When the muscles break down, the stem cells within the muscle aren’t able to replace the lost tissue. It’s a devastating condition

Stem cell researchers have, in the past, thought that one approach to treating the disease could involve maturing embryonic stem cells or reprogrammed iPS cells into muscle-specific stem cells (called satellite cells) and transplanting those into the muscles of people with the disease. The problem has been that, until now, nobody had been able to prod those embryonic or iPS cells to form the sought-after satellite cells.

The group from Minnesota finally worked out the right combination of signals that would push the cells to form the muscle precursors in a lab dish

Using their technique, the group generated large numbers of satellite cells from either human embryonic stem cells or iPS cells. When they transplanted those cells into mice with a form of muscular dystrophy those mice regained some strength. A story in Medical News Today quotes the lead researcher Rita Perlingeiro:

“Seeing long-term maintenance of these cells without major adverse side effects is exciting. Our research proves that these differentiated stem cells have real staying power in the fight against muscular dystrophy.”

In the paper, published May 4 in Cell Stem Cell, the group wrote that they think they can make enough of the appropriate type of cell to be able to treat a person with muscular dystrophy. However, they do have several obstacles to overcome. The way they generated the muscle stem cells would not be a safe approach for use in humans. Despite that hurdle, the scientific director of clinical research at the University's Stem Cell Institute and renowned blood and marrow transplant expert John Wagner said the work has promise:

"This research is a phenomenal breakthrough. Dr. Perlingeiro and her collaborators have overcome one of the most significant obstacles to moving stem cell therapies into the treatment of children with devastating and life threatening muscular dystrophies."

CIRM also funds researchers investigating stem cell therapies for muscular dystrophy. A complete list of those awards with descriptions of the research is available on our website.

A.A.