Muscular Dystrophy Treatment

Nov 16, 2006:

In promising new research, stem cells worked remarkably well at easing symptoms of muscular dystrophy in dogs, an experiment that experts call a significant step toward treating people.

“It’s a great breakthrough for all of us working on stem cells for muscular dystrophy,” said researcher Johnny Huard of the University of Pittsburgh , who wasn’t involved in the work.

Sharon Hesterlee, vice president of translational research at the Muscular Dystrophy Association, called the result one of the most exciting she has seen in her eight years with the organization. Her group helped pay for the work. She stressed that it isn’t yet clear whether such a treatment would work in people but said she had “cautious optimism” about it.

Two dogs that were severely disabled by the disease were able to walk faster and even jump after the treatments.

The journal Nature published the study online. The study used stem cells taken from the affected dogs or other dogs, rather than from embryos. For human use, the idea of using such “adult” stem cells from humans would avoid the controversial method of destroying human embryos to obtain stem cells.

The Nature paper focuses on Duchenne muscular dystrophy, a muscle-wasting genetic disorder that occurs in about 1 in every 3,500 male births. It is the most severe and most common childhood form of muscular dystrophy and the best known. In theory, the stem-cell treatment might also help other muscle dystrophies or even age-related muscle wasting, Dr. Hesterlee said.

Children with the disorder have trouble walking as early as preschool, and almost all of them lose their ability to walk between ages 7 and 12. Typically, they die in their 20s because of weak heart and lung muscles. There is no known cure.

The dog study was done by Giulio Cossu, director of the stem-cell institute at the San Raffaele Scientific Institute in Milan , Italy , with colleagues there and elsewhere. He said he hopes to start a small experiment in children in the next year or two.

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