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Myostatin Blockers: The Potential Benefits and Risks of These Forthcoming 'Super-Muscle' Drugs

In 2000, a boy was born in Charite Hospital in Berlin, Germany. Unlike the other babies, his muscles were not soft and undefined. They were bulging from his arms and legs. As the child grew, he developed muscles twice the size of his peers, while maintaining half the body fat, and before the age of 4 he was able to hold seven-pound weights with his arms extended.

super muscle drugs

A boy born in Germany in 2000 is the first known human to have a mutation in the myostatin protein, which results in enhanced muscle growth (he is 7 months old in this picture).

This little Hercules, who is now a healthy 7-year-old, is the first human with a proven mutation in myostatin, a protein that limits muscle growth.

By blocking this protein, for instance, researchers have grown muscular mice they call "mighty mice," and cattle breeders have developed the Belgian Blue breed -- the so-called "double muscle" cattle that are especially meaty and very lean.

The discovery of the myostatin mutation in a human, however, has sparked significant interest in myostatin-blocking drugs, which are surrounded by a growing sea of debate.

Myostatin Blockers for Muscular Dystrophy and Diabetes?

Myostatin-blocking drugs could theoretically help people with Duchenne muscular dystrophy and similar diseases that destroy muscle, such as cancer, AIDS, and even normal aging. Astronauts, who lose muscle mass after being in zero-gravity for prolonged periods, could also potentially benefit from such drugs.

Pharmaceutical company Wyeth has already gained the rights to develop a human myostatin blocker, and has begun testing a version with antibodies that block the protein in adults. Results are expected as early as March of this year.

"Just decreasing this protein by 20, 30, 50 percent can have a profound effect on muscle bulk," said Dr. Lou Kunkel, director of the genomics program at Boston Children's Hospital and professor of pediatrics and genetics at Harvard Medical School (and also a doctor participating in the Wyeth's research).

Further, according to Dr. Se-Jin Lee, a Johns Hopkins professor who helped create the "mighty mice" (and who, along with Johns Hopkins, would receive royalties for Wyeth's drug), a myostatin blocker may also be able to suppress fat accumulation and help stop the development of diabetes.

Potential Problems Down the Road?

While proponents say the drugs hold promise, others express concern at potential side effects of blocking the myostatin protein, which are completely unknown at this time.

super muscle drugs

While myostatin blockers hold promise for those with serious diseases like muscular dystrophy, they could also be abused by bodybuilders and athletes looking to gain a competitive edge.

One possible concern, according to Dr. Markus Schuelke, the pediatric neurologist at Charite University Medical Center in Berlin who discovered the myostatin mutation in the baby, is that blocking myostatin could interfere with satellite cells that help replace injured or dead muscle cells. It's thought that myostatin helps keep the satellite cells at rest until they're needed, and it's possible that without myostatin the satellite cells could become depleted.

Meanwhile, some say myostatin blockers may be too targeted to boost muscle growth, as there are a variety of proteins similar to myostatin that also limit muscle growth.

Another drug company, Acceleron Pharma, is planning to test a drug on humans that reportedly blocks not just myostatin but also many of the other similar proteins.

Myostatin-Blockers Could Become the New "Steroids"

Other companies looking to gain a piece of the myostatin fortune include food producers, who are looking to alter animals such as chickens so they contain more meat, and supplement companies, which are already promoting myostatin-blocking alternatives made from sea algae to body builders and other athletes.

"Myostatin blockade," said Dr. Elizabeth McNally of the University of Chicago, "will probably work its way into professional and amateur athletics, as well as into the ever-growing business of physical enhancement."

Experts say the knock-off supplements currently on the market don't work and could carry unknown health risks, as could the real myostatin-blockers that may one day be available not only to those with serious illnesses but also to those looking to simply "beef up" their physique.

Recommended Reading

Modafinil & Company: The Promises & Perils of Mind-Altering, World-Changing Drugs Like Modafinil

Drugs with Potentially Psychotic Side Effects: Which Ones are They (& Who Deserves to Know)?

Sources February 26, 2007

The New York Times

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