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New funding for Duchenne muscular dystrophy research in
UK29 Mar 2004
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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