New funding for Duchenne muscular dystrophy research in UK

Main Category: Muscular Dystrophy / ALS
Article Date: 29 Mar 2004 - 0:00 PDT

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Cutting edge research into the fatal muscle wasting condition Duchenne muscular dystrophy, which could lead to the first UK safety trial in humans, has received £1.6 million Government funding it was announced today.

The new technique aims to insert a 'molecular patch' over the genetic error which causes Duchenne muscular dystrophy, to restore production of an essential muscle protein. This will initially be achieved by directly injecting the 'molecular patch' (known as antisense oligonucleotides) into the muscles.

The research project has been put together by a Consortium of leading UK scientists co-ordinated by the Head of Research at the Muscular Dystrophy Campaign, Jenny Versnel. "This injection of funding by the Department of Health to develop the technique for use in humans is extremely encouraging. It is hoped that the severity of Duchenne muscular dystrophy could be reduced to a much milder form, significantly improving quality and length of life." said Versnel.

Professor Francesco Muntoni, the Principal Investigator for this project, from Imperial College London said "Our consortium is delighted with the news of funding for this research leading to a clinical safety trial. Although this is only the first step towards developing an effective treatment for this condition, the consortium has the necessary expertise not only to test the safety of this approach, but also to try and improve its efficiency so that its impact could be even more significant in the future".

Christine Cryne, Chief Executive of the Muscular Dystrophy Campaign said "It is fantastic that this research can now start. There is still a long, long way to go, but a treatment which could preserve the muscles of affected children would represent an amazing step forward. At present there is no cure or treatment to prevent the muscle breakdown associated with Duchenne muscular dystrophy."

Duchenne muscular dystrophy is first noticed in early childhood and leads to progressive disability and reduced life expectancy of around 20 years of age. It is caused by genetic errors in the dystrophin gene, which leads to the absence of a protein essential for the functioning of muscle cells. Without this dystrophin protein muscle cells break down and die.

Laboratory studies have shown that the injection of 'molecular patches' directly into small muscles can modify the faulty genetic code leading to the production of dystrophin.

The Government funding of £1.6 million towards the research will come from money made available through last year's Government White Paper on Genetics and follows concerted campaigning by the Muscular Dystrophy Campaign, Parent Project UK and the Duchenne Family Support Group.

Hundreds of parents of boys with Duchenne muscular dystrophy have been lobbying their MPs and the Department of Health, with active support from the All Party Parliamentary Group on muscular dystrophy, chaired by Kevin Brennan MP. The campaigners have been calling on the Government to invest £20 million over five years in order to speed up research into Duchenne muscular dystrophy.

"This investment from the Department of Health is vital. Now we have to seek further funding of around £1 million to take research beyond initial safety trials in humans. And we will need an even bigger cash injection to take the research further if the 1800 boys in the UK and the thousands around the world who have Duchenne muscular dystrophy are to benefit" added Christine Cryne.

The Consortium is made up of scientists from the MRC Clinical Science Centre, Hammersmith Hospital; Institute of Human Genetics, University of Newcastle upon Tyne; MRC Clinical Sciences Centre, Royal Postgraduate Medical School; Division of Neuroscience and Psychological Medicine, Imperial College London; School of Biological Sciences, Royal Holloway College, University of London, and the Department of Human Anatomy and Genetics, Oxford University.

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