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STEM CELL RESEARCH
EuroStemCell one step closer to helping MD sufferers walk again
As stem cell research grabs headlines as a hot button issue, concrete scientific results can sometimes get lost in the fray. EuroStemCell, the European Consortium for Stem Cell Research, has been making significant advances in laboratories across Europe finding innovative solutions to a myriad of diseases. Most recently they have produced promising results for treating the debilitating, often fatal, genetic disorder muscular dystrophy. They published their findings in a recent issue of Nature.
Muscle deterioration caused by Duchenne MD significantly reduces a patient’s life expectancy.

EuroStemCell researchers have been able to temporarily restore a dog’s ability walk after the advanced stages of muscular dystrophy have set in. The prospects of such treatment have enormous implications for humans suffering from the disease, and researchers hope to move on to clinical trials for people as early as next year.

Muscular dystrophy is a general term referring to a range of genetic disorders that affect muscle cells. It is characterised by progressive muscle weakness and death of muscle cells and tissue. Symptoms can vary widely with some patients experiencing limited mild effects over a normal life span to others suffering from severe disabilities. In some cases it can lead to death. There is currently no cure, making EuroStemCell’s research all the more exciting.

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.

They decided to then test their findings on golden retrievers carrying a mutation of the dystrophin gene, which closely resembles the root of the disease in humans. The team 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. All four lived for more than a year after the experiment, and initially regained the ability to walk. As noted in the Nature article, dogs in such cases usually die around one year of age as a result of the failure of respiratory muscles. 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.