EuroStemCell, the European Consortium for Stem Cell Research, most recently have produced promising results for treating the debilitating, often fatal, genetic disorder muscular dystrophy. They published their findings in a recent issue of Nature.
EuroStemCell researchers from the San Raffaele Scientific Institute in Milan, Italy, led by Dr Giulio Cossu, have been studying the most common form of the disease, known as Duchenne muscular dystrophy. They discovered that the mesoangioblast muscle stem cell, present in the walls of blood vessels, produced encouraging results when injected in mice.
Researchers took mesoangioblast stem cells from the blood vessels of dogs with the mutation, corrected it using gene therapy, and re-injected the modified stem cells. They also repeated the procedure with cells from healthy dogs, using drugs to prevent immune rejection.
Both treatment procedures resulted in the increased production of dystrophin, though the injection of the donor stem cells yielded the most encouraging results. Repeated doses of cells from the healthy dogs restored muscle function in four of five dystrophic dogs.
In their experiment, researchers found that stem cells successfully established themselves in the host tissue allowing for the production of dystrophin, off-setting the effects of muscular dystrophy. The injected stem cells not only produced dystrophin in the effected leg, but in other areas of the body as well, including respiratory muscles leading researchers to believe that they are on the right track to finding an effective treatment for MD sufferers.