Muscular dystrophy 'countered' by cancer drugs
A cancer drug 'could help' treat muscular dystrophy
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Monday, 18, Sep 2006 08:20
Scientists looking for cures to muscular dystrophy believe a class of drugs currently undergoing investigation as a potential cancer treatment could help counter the disease.
A group of Italian researchers from the Burnham Institute, publishing their findings online yesterday evening, identified the Trichostatin A drug class as effectively restoring deteriorated skeletal muscle mass.
They also found that the drug class improves resistance to contraction-coupled degeneration, the primary means through which the most prevalent form of the disease, Duchenne muscular dystrophy, breaks down muscle function.
The scientists drew their conclusions from studies conducted using mice over a period of three months. Those mice receiving treatment were able to run on a treadmill like healthy mice, while dystrophic mice were not.
"We have identified a new rationale for treating muscular dystrophy, aimed at correcting the devastating effects of a single flawed gene," said Professor Lorenzo Puri, lead author of the study, referring to the recessive X chromosome which causes the Duchenne type of the disease.
"This is a significant advance over the use of steroids - currently the only treatment available - which offers palliative relief, often with severe side effects."
Muscular dystrophy affects around 30,000 people in the UK, according to the Muscular Dystrophy Campaign charity. Most children who develop the disease die in their late teens or early 20s.
"These exciting results, while encouraging, will require extensive investigation to determine whether the effectiveness of these drugs in dystrophic mice will translate into an effective treatment for individuals suffering this disease," Professor Puri warned.
"It is difficult to predict how long it will take before these studies will be translated into therapies for human patients," he added.