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Treatment hope for muscular dystrophy

Date:
April 4, 2012
Source:
University of Melbourne
Summary:
Medical researchers have found that increasing a specific protein in muscles could help treat Duchenne muscular dystrophy, a severe and progressive muscle wasting disease that affects young boys.

An international team led by the University of Melbourne Australia, has found that increasing a specific protein in muscles could help treat Duchenne muscular dystrophy (DMD), a severe and progressive muscle wasting disease that affects young boys.

Approximately one in every 3,500 boys worldwide is afflicted with DMD. There is no cure for the disease which causes muscle fragility, spinal curvature and premature death.

Results from the studies recently published in Nature showed that by increasing levels of 'heat shock protein 72' (HSP72) in the muscles of animal models of DMD, muscle strength improved, the disease progression slowed and lifespan increased.

The research led by Professor Gordon Lynch, Head of the Department of Physiology at the University of Melbourne and conducted by Dr Stefan Gehrig for his PhD, investigated several scientific approaches of increasing the levels of the protein. The study was also performed in collaboration with Professor Mark Febbraio and his team at Baker IDI Heart and Diabetes Institute.

One approach revealed that administering the drug BGP-15 (which increases HSP72) improved overall muscle function in limbs and the diaphragm, a muscle critical for breathing. In addition, the lifespan increased by 20 per cent.

The researchers also discovered that increasing HSP72 in muscles improved the function of a pump responsible for controlling calcium levels confirming it as a target for future therapeutic drugs for the disease.

"Our studies show that by increasing HSP72, we can improve calcium pump function which could be a way to help reduce the muscle breakdown in boys suffering the condition," Professor Lynch said.

"We hope that these exciting findings will serve as the basis for future clinical trials within the next five to 10 years," Professor Lynch said.

Researchers from The University of Oxford, UK and Deakin University, Australia also collaborated in the research. The project was supported by the National Health and Medical Research Council, The Heart Foundation, Association Franηaise contre les Myopathies and the Muscular Dystrophy Association (USA). BGP-15 was provided by the pharmaceutical company N-Gene.


Story Source:

The above story is based on materials provided by University of Melbourne. Note: Materials may be edited for content and length.


Journal Reference:

  1. Stefan M. Gehrig, Chris van der Poel, Timothy A. Sayer, Jonathan D. Schertzer, Darren C. Henstridge, Jarrod E. Church, Severine Lamon, Aaron P. Russell, Kay E. Davies, Mark A. Febbraio, Gordon S. Lynch. Hsp72 preserves muscle function and slows progression of severe muscular dystrophy. Nature, 2012; DOI: 10.1038/nature10980

Cite This Page:

University of Melbourne. "Treatment hope for muscular dystrophy." ScienceDaily. ScienceDaily, 4 April 2012. <www.sciencedaily.com/releases/2012/04/120404133757.htm>.
University of Melbourne. (2012, April 4). Treatment hope for muscular dystrophy. ScienceDaily. Retrieved July 31, 2014 from www.sciencedaily.com/releases/2012/04/120404133757.htm
University of Melbourne. "Treatment hope for muscular dystrophy." ScienceDaily. www.sciencedaily.com/releases/2012/04/120404133757.htm (accessed July 31, 2014).

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