STEM CELLS SUCCESSFULLY TREAT MD IN DOGS

TUCSON, Ariz., Nov. 15, 2006 – Researchers have successfully used stem cells to treat dogs with a form of muscular dystrophy similar to the Duchenne (DMD) type in humans, the Muscular Dystrophy Association (MDA) announced today.

The investigators, including Maurilio Sampaolesi and MDA-funded Guilio Cossu of the San Raffeale Scientific Institute in Milan, Italy, used a special kind of stem cell called a “mesoangioblasts” that can be isolated from muscle biopsies of living donors.

Thirteen dogs, divided into three groups, received either cells from healthy donors, their own cells that had been corrected to have the protein missing in DMD or no cells at all. The dogs that received cells from healthy donors responded the best, with one of that group walking well at 13 months (most dogs with the disease have difficulty walking by eight months of age and die at about one year).

“Many therapeutic approaches have been successful in mice with muscular dystrophy, but this is the first example of functional improvement in a large-animal model of the disease, and a major step toward testing these cells in the clinic,” said Sharon Hesterlee, MDA vice president of translational research.

The dogs in this project were injected from three to five times with 50 million cells at one-month intervals. The cells were injected into the arterial system of a limb. Some of the animals were also treated with drugs to suppress the immune system.

Results were documented biologically and through at least two measures of muscle function. There was no evidence of immune response in any of the dogs.

“It’s not clear how long it will take to move this promising work to the clinic, but we’re cautiously optimistic,” said Valerie Cwik, MDA medical director and vice president of research. “The fact that the protein was effectively delivered to several muscles, rather than just one muscle at a time, is very significant.”

MDA is currently funding Cossu to isolate mesoangioblasts from human biopsy tissue in anticipation of future studies.

MDA is a voluntary health agency that provides services, research, and professional and public health education. The Association’s programs are funded almost entirely by individual private donors.