Muscular Dystrophy: Gene and Stem Cell Therapy

Transcript

Hello, my name is Professor Allan Bretag, Adjunct Professor, School of Pharmacy and Medical Sciences at the University of South Australia and Director of Research of the Muscular Dystrophy Association. I joined the Editorial Advisory Board of the Virtual Neuro Centre two years ago, and today I would like to share with you my insights on gene and stem cell therapy for muscular dystrophy and other neuromuscular diseases.

Over the past few years, animal diseases that correspond to human muscular dystrophies and other neuromuscular diseases have been studied while looking for potential treatments for the human conditions. In particular, gene therapy and stem cell therapy have been widely promoted as being successful in animals and almost ready for clinical trials in humans.

Unfortunately however, exaggerated claims for these novel treatments have appeared throughout the media, with reports of major breakthroughs occurring increasingly, not only in newspapers and on TV but also in highly respected medical and scientific magazines. In most instances, these assertions are unjustified, leaving only the false hopes in patients, their families or carers and their doctors.

Reporting of research into possible treatments for neuromuscular diseases must be cautious and totally honest, while the research itself requires undeniable clinical and functional proof to deserve this kind of rave review. In recent months, there has been a research report from Genethon, the giant genetic research institute on the outskirts of Paris, that is funded by the French Muscular Dystrophy Association, that seems, on the face of it, quite promising. It concluded that a more recently developed virus, known as adeno-associated virus, could be used to carry the missing gene into the muscles of one leg of muscular dystrophy mice, producing full recovery from the muscle weakness in the muscles of that leg.

While this report suggests that considerable progress has been made, there are still many problems regarding the safety and injection of these viruses to be overcome, before it can be claimed that a cure has been achieved, even in the mice. Of more concern however, a number of recent research reports from stem cell research institutes associated with the University of Milan in Italy have promoted stem cell therapy using meso-angioblasts. For example one of these articles in the prominent scientific journal Nature, is very optimistic, claiming exceptional success in the treatment of dystrophic dogs. Disappointingly, the evidence that this treatment genuinely benefits the dogs is arguable. Further reports from the same research group on the use of meso-angioblast treatment in muscular dystrophy mice, have been no more reassuring, claiming complete repair of dystrophic muscle, and implying that so far as human muscular dystrophies are concerned, this work sets the stage for combined treatment, with perhaps the patient’s own genetically corrected stem cells.

Although it is hard to find solid evidence in the reports to support these assertions, it is now almost guaranteed that unauthorised therapy centres and premature human clinical trials will soon be established in third world countries. It is a great disappointment that many of the highly publicised gene and stem cell therapies proposed for neuromuscular diseases have not so far live up to the extensive media promotion that has surrounded them, but continue to dash the hopes they have aroused.

Thank you for watching.

More information

To learn more about the risk factors for Muscular Dystrophy, symptoms and treatment, see Muscular Dystrophy.