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Dystrophy Drug Passes 1st Test

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By DENISE GRADY, The New York Times

Published: December 30, 2007

Researchers in the Netherlands have developed a drug that eventually may be used to treat children with a severe and fatal type of muscular dystrophy.

In its first test in humans, a safety study in just four boys, the drug enabled patients to produce an essential muscle protein that is missing in Duchenne's muscular dystrophy, a genetic disease.

Not enough of the protein was produced to help the boys, but the presence of any at all was considered "proof of concept," meaning that the approach has the potential to work and is worth pursuing.

The experimental drug, called an "antisense" compound, works by canceling out the effects of certain genetic mutations. These types of drugs are being studied to treat cancer, heart disease, infections and other illnesses.

"I don't think you could ask for a better result from a preliminary study like this," said Sharon Hesterlee, the vice president for translational research at the Muscular Dystrophy Association, which was not involved in the study but helped pay for a phase of the work.

Though promising, the research still has a long way to go. The four boys, ages 10 to 13, each received just one injection into a leg muscle. There were no adverse effects.

But larger and longer trials with much higher doses, given systemically so that the drug reaches all muscles, are needed to test safety and efficacy. If the treatment works, it will have to be given regularly, for life.

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