15 November 2006

Research into a potential future treatment for Duchenne muscular dystrophy using stem cells has been published this week in the leading science journal, Nature.

Researchers, from Italy and France, extracted stem cells from both normal dogs and from dystrophic dogs. The dystrophic stem cells were then genetically modified to mirror the normal cells and were then delivered directly into the bloodstream of dystrophic dogs. Over a period of five months one group of animals received the modified stem cells while the other group received the cells taken from healthy dogs.

Significantly, improvements in muscle function and strength were seen in all cases, but those receiving healthy cells showed a generally greater improvement than those injected with the corrected versions.

The research demonstrates, for the first time, that stem cells can be delivered systemically directly into the bloodstream via an artery, restoring muscle function and strength throughout the entire body. Director of Research at the Muscular Dystrophy Campaign, Dr. Marita Pohlschmidt, said:

“The Muscular Dystrophy Campaign recognises the potential of stem cell therapy for the repair of damaged and degenerated muscle tissue. We feel encouraged by the work published by Cossu and his colleagues because the results provide initial evidence that we might be one step closer to a stem cell treatment for Duchenne muscular dystrophy. The research is still at an early stage and there are a number of issues to be resolved before this technology can be tested on humans. Importantly though, if it does prove to be successful in humans, this technology has the potential to develop into an efficient and groundbreaking treatment not only for Duchenne, but also other muscular dystrophies.”

The full paper is published on Nature’s website at 18.00 on 15 November at www.nature.com

For more information call the Muscular Dystrophy Campaign on 020 7720 8055 or email info@muscular-dystrophy.org

You can also refer to the questions and answers page at Stem cell research - questions & answers