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‘Heat shock’ protein halts muscle disease

U. MELBOURNE (AUS) — Researchers have found that increasing a specific protein in muscles could help treat Duchenne muscular dystrophy.

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


Results from the studies 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 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 percent.

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,” says Lynch.

“We hope that these exciting findings will serve as the basis for future clinical trials within the next five to 10 years,” he says.

Researchers from University of Oxford, UK and Deakin University, Australia also contributed to the research, which was supported by the National Health and Medical Research Council, The Heart Foundation, Association Française contre les Myopathies, and the Muscular Dystrophy Association. The pharmaceutical company N-Gene provided BGP-15.

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