making muscle wasting history

Funding for pioneering research into gene therapy by Action Duchenne has led to further funds for research into this life limiting disease

A recent £2.5 million award made by the Health Innovation Challenge Fund (HICF) to Dr Matthew Wood (Department of Physiology, Anatomy and Genetics and Somerville College) to further work into developing treatments for Duchenne Muscular Dystrophy has been welcomed by Action Duchenne, the UK charity for DMD. The award will fund more research exclusively for Duchenne, a life limiting disease that is caused by damage to the dystrophin gene and affects 1 in 3,500 male births in the UK. It will be used to further develop treatments that have been successfully trialled by Dr Wood in collaboration with Mike Gait at Cambridge Universities, and colleagues at the MDEX Consortium, a network of research scientists.

The initial project was funded by Action Duchenne.
Dr. Matthew Wood said, “This successful Wellcome funding award is a fantastic boost for DMD research which we would have been unlikely to win without the foresight and earlier funding commitment by Action Duchenne.”

The development of drugs that can act on gene splicing has long been a key research aim for Action Duchenne. Nick Catlin, CEO of Action Duchenne commented, “Initial clinical trials have shown these drugs to be safe and do produce dystrophin in humans. A second trial conducted by the MDEX consortium has now been completed and shows the potential for systemic delivery.”

One of three grants totaling £4.4m for the development of innovative medical technologies and treatments, the award is among the first to be made by the HICF initiative, established in 2009 to address gaps in funding for innovative medical research and help turn scientific breakthroughs into potential medical treatments.

Dr Wood said: ‘Being selected as one of the first recipients of an HICF award is a fantastic opportunity to expand our world-leading science and to develop and test a ground-breaking new treatment for Duchenne muscular dystrophy, a devastating disease where new treatments are desperately needed.’
Action Duchenne has funded a key research project with Matthew Wood’s team at Oxford and Mike Gait at Cambridge Universities. The aim of the project for £175,000 is to develop new and improved ways of delivering the antisense oligonucleotides (AOs) needed for exon skipping. The Wood team have now shown in animal models that these new conjugated AO’s can improve the efficiency of delivery in animal models even in the heart.

Led by Dr Wood, the group is now developing a new generation of AO drugs which will dramatically improve the delivery and restoration of dystrophin by all muscle. The treatment will be tested in a clinical trial on nine DMD patients, starting in 2013.

CEO of Action Duchenne Nick Catlin said; “Action Duchenne is delighted by the news of this award. It will enable the pioneering work that Dr Wood and his colleagues have achieved to date to continue. It demonstrates the recognition of the value and success that they have had in developing a treatment for this life limiting disease, that gives hope to all our families and boys living with Duchenne.”