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.”
Notes to Editors
The Health Innovation Challenge Fund (HICF) is a parallel funding partnership between the Wellcome Trust and the Department of Health. The funders are collaborating to stimulate the creation of innovative healthcare products, technologies and interventions, and facilitate their development for the benefit of patients in the NHS and beyond. The HICF operates a succession of thematic calls for proposals, each selected to focus on unmet needs in healthcare relevant to the NHS, and will support innovative developments that are within three to five years of launch or adoption. www.hicfund.org.uk
The Wellcome Trust is a global charitable foundation dedicated to achieving extraordinary improvements in human and animal health. It supports the brightest minds in biomedical research and the medical humanities. The Trust’s breadth of support includes public engagement, education and the application of research to improve health. It is independent of both political and commercial interests.www.wellcome.ac.uk
The Department of Health (DH) works to improve the health and well-being of people in England. The Department sets overall policy on all health issues and is responsible for the provision of health services through the National Health Service. www.dh.gov.uk
The MDEX Consortium
The MDEX Consortium is a group of research scientists that has received over £2m funding from the UK Government and Medical Research Council (MRC) to conduct clinical trials on DMD patients.
The MDEX Consortium is funded by the department of Health and groups together nine researchers as well as the charities Muscular Dystrophy Campaign, Action Duchenne and Duchenne Parents Support Group.
For more information visit www.mdex.org.uk
About Action Duchenne
Established in 2001 Action Duchenne aims to support and promote innovative research into a cure and effective medicines for Duchenne/Becker Muscular Dystrophy. The charity, which is led by Duchenne families, aims to promote awareness of the condition, to improve care services, and provide access to a range of educational and support/development programmes for people living with Duchenne at every stage of the condition. This is achieved by working in partnership with government agencies, NHS and care organisations, other charities, academic, scientific and research groups, and biotech companies worldwide.
Action Duchenne is actively campaigning for best practice in care for Duchenne based on the publication of an international consensus document in Lancet Neurology in Jan/Feb 2010. The published article was the product of an extensive review by 84 international experts in Duchenne Muscular Dystrophy diagnosis and care, and was supported by the Centers for Disease Control (CDC) in the US. It provides an expert guide to recommendations on the multidisciplinary care that should be available to all individuals with Duchenne. Several Duchenne patient organisations including Action Duchenne and the TREAT-NMD network have helped to produce a ‘Guide for Families’, based on the original academic article, which is written in language accessible to all without a medical background.
Since 2003 Action Duchenne has provided £4m for research projects and partnerships. The charity has worked with the MDEX consortium, Department of Health, and the Medical Research Council to deliver new clinical trials for Duchenne drugs. In addition, it has been instrumental in developing projects with biotech companies both in the UK and US including key projects with AVI Biopharma and Summit.
In 2005 Action Duchenne launched the DMD Patient Registry, a national database that holds gene information of people living with Duchenne that is used to speed up the recruitment of patients for clinical trials. For more information please visit: www.dmdregistry.org
In 2008 Action Duchenne launched the Include Duchenne project in partnership with Decipha. This programme addresses the learning and behaviour needs of those living with Duchenne. For more information please visit: www.decipha.org
Action Duchenne promotes social inclusion within the community for young men living with Duchenne through its Genius programme. For more information please visit: www.actionduchenne.org/geniusproject
In 2010 the National Advocacy Council was launched to spearhead the lobby for government funding for research into Duchenne and to improve access to Standards of Care. www.actionduchenne.org/NAC
For more information please visit: www.actionduchenne.org
CEO, Action Duchenne
Tel: 0208 556 9955
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