Stem cell treatment eases muscular dystrophy in dogs

Published 9:00 pm Wednesday, November 15, 2006

NEW YORK – Stem cell injections worked remarkably well at easing symptoms of muscular dystrophy in a group of golden retrievers, a result 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’s seen in her eight years with the organization. Her organization helped pay for the work.

She stressed that it’s not yet clear whether such a treatment would work in people, but she 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 study was published online Wednesday by the journal Nature. It used stem cells taken from the affected dogs or other dogs rather than from embryos. For humans, the use of such “adult” stem cells from people 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 affects only boys and occurs in about 1 in every 3,500 male births. It’s the most severe, most common and best-known childhood form of muscular dystrophy. In theory, the stem cell treatment might also help other muscle dystrophies or even age-related muscle wasting, Hesterlee said.

Boys with Duchenne dystrophy have trouble walking as early as preschool age, and nearly all of them lose their ability to walk between ages 7 and 12. Typically, they die in their 20s because of weakness in their heart and lung muscles. There is no known cure.

The researchers treated two dogs that were severely impaired by the disease. Both gained the ability to move much faster and to jump, and one was even able to run, although neither could use its hind legs normally.

One of these dogs rapidly lost walking ability when the anti-rejection treatment was stopped, but the other continued to walk well for five months until succumbing to pneumonia. That’s a common fate for dogs with the genetic condition because of weakness in breathing muscles.