Utrophin is a protein very similar to dystrophin, which can perform similar functions to dystrophin to protect and repair muscle. During tests of SMT C1100, there was a two-fold increase in utrophin in human tissue from DMD patients. In the mdx mouse, the tests showed utrophin replaced dystrophin function, reducing damage from regeneration, fibrosis and inflammation for longer. The tests also showed an increase in the distance travelled by the exercised mdx mouse of around 50%. This study is comparable to the Six Minute Walk Distance test where an increase in distance walked is the current benchmark used to identify a successful treatment in human patient trials for Duchenne. The drug showed no toxicity issues.
A recent Phase I trial of a small number of healthy volunteers showed the drug in its current formulation was safe but not consistently present at beneficial levels in the blood. This is a common problem with Phase I trials and is often resolved through reformulation of the drug and it is intended to take a reformulation of SMT C1100 back into the clinic.
Professor Dame Kay Davies, Honorary Director, MRC Functional Genomics Unit., Head of Department. Co-Director, Oxford Centre of Gene Function commented, “These results show proof-of-principle for the development of small molecules able to increase levels of utrophin for the therapy of Duchenne Muscular Dystrophy. The great advantage of this approach is that it will be possible to treat all DMD and Becker patients, irrespective of their dystrophin mutation. The results provide strong support of retesting new formulations of SMT C1100 in new Phase I clinical trials with a view to progressing to DMD patient trials.”
Nick Catlin, CEO of Action Duchenne said, “This latest news from Summit is great for those living with Duchenne. We are now another step closer to further clinical trials for a therapy that could benefit all Duchenne patients and could be taken at home easily. However, we must not assume that the job is done, there is still a lot of work to do if these drugs are to provide an effective treatment for Duchenne. We need to continue our fundraising work to ensure that these drugs are brought to market as quickly, effectively and as safely as possible.”
A full version of the paper announcing these latest results is available from: www.plosone.org
Notes to Editors
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/
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/
For more information please visit: www.actionduchenne.org
CEO, Action Duchenne
Tel: 0208 556 9955
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