Public Release: 

Cancer drug shows promise for treating Duchenne muscular dystrophy

University of Sheffield

A drug commonly used to treat leukaemia is showing potential as a treatment that could slow the progression of the muscle-wasting condition, Duchenne muscular dystrophy.

Duchenne muscular dystrophy most commonly affects boys, with around 2,400 people in the UK affected by the condition. There is currently no cure and most patients are not expected to live past the age of 30.

Researchers at the University of Sheffield investigated a drug called dasatinib, which works by blocking certain chemical signals that stimulate the growth of cancer cells. They found the same drug will also switch off similar signals in a protein implicated in Duchenne Muscular Dystrophy (DMD). This protein, called dystroglycan, has a part to play in maintaining healthy muscle tissue.

The team tested the drug in zebrafish bred to carry DMD and recorded a 40 % improvement in the condition of the fish. Those fish treated with dasatinib were able to swim further and for longer than those in a control group. It could be that by combining the drug with other treatments currently under development, their effectiveness could be improved even further. The results are published in the journal Human Molecular Genetics.

"Dasatinib clearly has promise as a treatment for DMD," says Professor Steve Winder, who led the research. "From our understanding of how the drug works we believe it could be effective in slowing muscle deterioration, prolonging patients' ability to walk and also protecting their heart and respiratory muscles. There is the potential that if the drug were taken immediately upon diagnosis, the disease progression could be dramatically reduced."

Because dasatinib is already cleared for clinical use, researchers hope that progress can be made more quickly towards trialling the drug in humans as a treatment for DMD. Experiments have already begun in mice, with promising results. Other drugs that work in a similar way to dasatinib are also under investigation by Professor Winder's team.

Dr Marita Pohlschmidt, Muscular Dystrophy UK's Director of Research, said:

"These are encouraging findings about a unique new avenue to treating Duchenne muscular dystrophy. It is a complex condition and we are of the view that it will take a combination of therapies to treat it effectively. Professor Winder's approach could complement potential therapies currently advancing through clinical trials, making them more effective.

"In time, we hope that its potential to treat other muscular dystrophies will also be investigated. For now, we are looking forward to seeing the results of further research into Duchenne muscular dystrophy to support these early promising results."

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The research was funded by Muscular Dystrophy UK, with additional funding from the Medical Research Council and the Duchenne Parent Project NL.

Notes to Editors

'Dasatinib as a treatment for Duchenne muscular dystrophy,' by Leanne Lipscomb, Robert W. Piggott, Tracy Emmerson and Steve J. Winder, is published in Human Molecular Genetics

The University of Sheffield

With almost 25,000 of the brightest students from around 120 countries, learning alongside over 1,200 of the best academics from across the globe, the University of Sheffield is one of the world's leading universities.

A member of the UK's prestigious Russell Group of leading research-led institutions, Sheffield offers world-class teaching and research excellence across a wide range of disciplines.

Unified by the power of discovery and understanding, staff and students at the university are committed to finding new ways to transform the world we live in.

In 2014 it was voted number one university in the UK for Student Satisfaction by Times Higher Education and in the last decade has won four Queen's Anniversary Prizes in recognition of the outstanding contribution to the United Kingdom's intellectual, economic, cultural and social life.

Sheffield has five Nobel Prize winners among former staff and students and its alumni go on to hold positions of great responsibility and influence all over the world, making significant contributions in their chosen fields.

Global research partners and clients include Boeing, Rolls-Royce, Unilever, AstraZeneca, Glaxo SmithKline, Siemens and Airbus, as well as many UK and overseas government agencies and charitable foundations.

For further information, please visit http://www.sheffield.ac.uk

Muscular Dystrophy UK

Muscular Dystrophy UK is the charity for 70,000 children and adults living with muscle-wasting conditions. We provide vital information, advice and support to help people live as independently as possible. We accelerate progress in research and drive the campaign for access to emerging treatments.

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