Stem cells reverse muscular dystrophy in dogs

20 November 2006

Stem cells from blood vessels have helped dogs with muscular dystrophy to walk again - opening up new possibilities for treating this disease in humans.

Muscular dystrophies are a group of genetic and hereditary muscle diseases where the muscles gradually break down. The most common form, Duchenne muscular dystrophy, is caused by mutations in a gene called dystrophin. It affects around one in every 3500 children (usually boys), and survival is rare beyond the early 30s.

EuroStemCell researchers from the San Raffaele Scientific Institute in Milan, Italy, led by Giulio Cossu, had previously shown that the mesoangioblast muscle stem cell, which lives in the walls of blood vessels, could help mice with a form of muscular dystrophy.

Now, in research published online last week in Nature, they've applied their findings to golden retriever dogs with a mutation in the dystrophin gene - a disease much closer to human muscular dystrophy.

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 regimes resulted in the production of dystrophin, but injection of the donor cells yielded the most dramatic 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. The cells colonised muscle and produced dystrophin, not just in the injected leg but elsewhere too, including the diaphragm.

With further testing, the team hopes that these promising results could lead to clinical trials in a few patients with Duchenne muscular dystrophy by late 2007.

Read the full paper (subscription required)

Selected media coverage
New Scientist (subscription)
The Seattle Times
BBC News
Telegraph online
The Australian

Relevant patient organizations
Parent Project
Muscular Dystrophy Association
Association Francais contre les Myopathies

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