25 June 2004

Holds The Key To Inherited Muscle Wasting

Since yesterday when the New England Journal of Medicine published a case report about a "a baby Superman, born in Berlin with bulging arm and leg muscles," who "has muscles twice the size of other children his age and half their body fat," the newswires (see Google accounts and this one) have been buzzing. The 4-year old boy can hold 7-pound weights with arms extended, something most adults are not capable of.

Courtesy of NEJM

The boy has an inborn defect in a muscle-controlling gene called myostatin. According to his doctors, his lack of myostatin does not appear to be affecting his health, but they are keeping watch in case it causes key organs, like the heart, to increase in size also. This gene has previously been examined in mice by Se-Jin Lee at Johns Hopkins University and blocking it can make mice twice as brawny as normal. No one knew its effects in humans.....until now.

What this discovery means: it means that successful therapies for degenerative muscle diseases in mice that target this gene might be also be effective in humans. Therefore. if scientists can find ways to interfere with myostatin function, perhaps there can be a way to slow down muscle loss in muscle wasting diseases like Duchenne's and Becker's muscular dystrophies and hopefully extend the lifespans of these patients.

Muscular dystrophies are genetic diseases that progressively damages skeletal muscle. No effective treatment exists, and most patients die in their 20s from a combination of heart and breathing problems.

Last thing I heard is that Wyeth Pharmaceuticals already has synthesized a protein that disables myostatin. Human safety trial of the substance, known as MYO-029, is ongoing. The company, which has a patent on all human therapeutic uses of myostatin, also is planning a study with muscular-dystrophy patients. [Seattle Times]

What's next: super athletes? super soldiers? superlarge chickens? superlarge beef and pork servings?

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