Muscular Dystrophy: Stem Cell Help?

Stem Cell Treatment Shows Potential in Lab Tests on Dogs

Medically Reviewed by Louise Chang, MD on November 15, 2006
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Nov. 15, 2006 -- Lab tests on dogs show that adult stem cells may help treat a certain type of muscular dystrophy, say Italian researchers in the journal Nature.

They included Giulio Cossu, MD, of the Stem Cell Research Institute in Milan, Italy.

The strategy needs more work. But Cossu's team says their study "sets the logical premise for the start of clinical experimentation" for Duchenne muscular dystrophy.

Duchenne muscular dystrophy is one of the nine major types of muscular dystrophy. It's the most common form of muscular dystrophy in children, and it only affects males.

In human Duchenne muscular dystrophy, muscles weaken over time. Many patients eventually need wheelchairs; most die in their late teens or early 20s.

Stem Cell Study

Cossu and colleagues studied 13 male golden retrievers that had a canine form of Duchenne muscular dystrophy.

The researchers took adult stem cells from the dogs with muscular dystrophy and from a dog without muscular dystrophy.

Cossu's team split the dogs with muscular dystrophy into three groups.

The first group included three dogs that got no stem cell shots. Their muscular dystrophy worsened.

The second group included six dogs that got five shots of donated stem cells from the healthy dog, along with drugs to help their immune systems accept those cells.

The last group included four dogs that got up to five shots of their own stem cells that had been tweaked to make more dystrophin, a protein lacking in Duchenne muscular dystrophy.

Mixed Results

The results varied, but the dogs that got the donated stem cells generally faired best.

Two of the six dogs that got donated stem cells were able to walk, one of which was able to run at the end of treatment.

A third dog in the same group died suddenly, and a fourth gradually lost walking ability after drugs for the immune system were stopped.

The fifth dog that got donated stem cells showed no improvements or setbacks, and the sixth showed "modest" worsening of its condition.

Of the four dogs that got shots of their own genetically tweaked stem cells, one was able to walk stiffly after treatment. Three worsened, two of which died of pneumonia.

Muscular dystrophy worsened in all of the dogs that didn't get stem cell shots.

Donated Cells?

The donated stem cells "seemed to be more efficient" than the genetically tweaked stem cells, the researchers write.

But transplanting stem cells can have drawbacks, notes editorialist Jeffery Chamberlain, PhD, of the University of Washington's neurology department.

"As with any transplant, unless the donor and recipient are immunologically matched, the recipient must be placed on lifelong immune suppression; this is not always effective and can have nasty side effects," Chamberlain writes in Nature.

He adds that it would be "preferable" to give each patient their own genetically corrected stem cells, if the technique can be made more effective.

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SOURCES: Sampaolesi, M. Nature, Nov. 15, 2006; online edition. WebMD Medical Reference: "Understanding Muscular Dystrophy -- the Basics." Chamberlain, J. Nature, Nov. 15, 2006; online edition. News release, Nature.

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