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scientists react to potential stem cell therapy for muscular dystrophy as published in Nature

A joint Italian/French team of scientists have published a study using stem cells to treat a disease in dogs that is similar to muscular dystrophy in humans.


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.”


Professor Dominic Wells, Gene Targeting Group, Department of Cellular and Molecular Neuroscience, Imperial College Faculty of Medicine, said:

“This exciting study is a major step forward in demonstrating the potential of stem cell therapy to treat Duchenne muscular dystrophy (DMD), a fatal muscle wasting disease. The substantial clinical improvement in a natural mutant dog version of DMD will encourage development of human clinical trials of this form of therapy for a range of muscle diseases.

“This is yet another example of how the vital contribution animal research makes to the development of treatments of human disease.

“In summary, this is the first piece of research that has convinced me that stem cell therapy could play a role treatment for Duchenne muscular dystrophy.”


Kay Davies, Dr Lee’s Professor of Anatomy, MRC Functional Genetics Unit, University of Oxford, said:

“The use of stem cells to treat human disease holds great promise but the actual delivery of such therapy is thought to be many years away. The data presented in the paper from Giolio Cossu’s group, using a particular type of stem cell, a mesoangioblast stem cell, changes that view. They demonstrate that transplantation of these cells into a dog model of Duchenne muscular dystrophy can, in some animals, lead to recruitment of these cells into muscle and a dramatic amelioration of the progressive muscle weakness.

“These are adult stem cells which are easy to isolate (and could be harvested from patients themselves to prevent rejection). We need to know why not all dogs responded positively. Nevertheless, this is major step forward and suggests that this approach may be effective for use in DMD patients.”


Dr Peter Wigmore, Senior Lecturer, Queen’s Medical Centre, Nottingham University, said:

“Stem cell therapy is regularly touted as a cure for many human diseases but successful results have been thin on the ground. In this study, dogs suffering from Duchenne muscular dystrophy, a progressive muscle wasting disease which in humans is invariably fatal, were treated by injection of adult stem cells into the blood stream. The cells were carried around the body to the affected muscles and increased their strength which in turn improved the ability of the dogs to walk.

“The importance of this result is not only in providing a potential cure for a currently incurable condition but also in the use of relatively easy to isolate adult stem cells. The use of these cells avoids the controversial use of embryonic or fetal stem cells. In addition the relatively easy delivery of these cells via the blood stream makes this a viable method to treat human patients.”


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