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Gene therapy could bring hope to thousands


posted 24/05/2005   expires 24/06/2005

A new technique for treating muscle wasting diseases, which has been developed at the Royal Free and University College medical school, is ready to be tested on humans.

 

The gene therapy technique has been developed by Professor Geoff Goldspink and Dr Shi Yu Yang who discovered that injecting a gene which is naturally expressed into wasted muscles after exercise could increase strength by up to 40 per cent.

 

Professor Goldspink is hopeful that the therapy could help a wide range of patients; those with muscular dystrophy and motor neurone disease are obvious examples but he believes it could also benefit HIV and cancer patients.   “The gene activates the muscle stem cells. In dystrophic mice we have recorded an increase in strength of 35 to 40 per cent in just three weeks.  It makes this a very good candidate for therapy for muscular dystrophy, especially as we have found a way to introduce this growth factor (MGF) into the body systemically so that it affects all muscles, not just the one which has been injected.”

 

The hope is that the injected gene may also repair some of the damage associated with diseases such as motor neurone disease.  “The gene which we have cloned turns out to be more of a repair factor rather than just a growth factor,” explains Professor Goldspink.  “It is apparently involved in the maintenance of the nervous system and, in the case of motor neurone disease, it does improve the connection between the motor neurones and the muscle.”

 

Previous attempts to boost one type of cell via gene therapy have increased the risks of cancer but Professor Goldspink says his technique has no such side effects; in fact he hopes it will help cancer patients by counteracting the muscle loss associated with their disease, making chemotherapy more effective.  “There are many diseases where muscle loss is a problem and patients actually die from that loss rather than the illness.   My job is to make sure we can treat this muscle loss and I am very pleased that future research will benefit from this development.”

 

Professor Goldspink believes that it is “inevitable” that the technique will be abused by athletes looking to improve their performance but believes that it is too important to be ignored.  “It is very exciting even though it will be misused because it has such an incredible effect on muscle growth.  Every week I get emails from people who have read about our research and who have muscle wasting diseases for which there is no treatment at the moment.  It would be great to be able to do something to help save these people from what can be a dreadful, lingering death.”

 

NOTES

 

This research has yet to go through clinical trials.  The research team are in discussions with a pharmaceutical company with a view to starting trials, hopefully in the near future.

 

The work into this therapy is being carried out in conjunction with Dr Richard Orrell, a neurologist specialising in motor neurone disease who works at the Royal Free, and Linda Greensmith at the institute of neurology at UCL.

 

The injected material increases the production of a protein called mechano growth factor (MGF) which boosts muscle mass and improves the muscle’s ability to repair itself.