New drugs that mimic mutations found in buff animals could treat muscle-wasting diseases
Scattered throughout the mammalian menagerie are a few supermuscular freaks: double-muscled cows more ripped than any bodybuilder; racing dogs too burly to run; sheep praised for their massively muscled buttocks; and even one small German boy, born in 2000 with muscles twice the size of those of a normal newborn. All these Herculean creatures share one thing: naturally occurring mutations in a gene that produces myostatin, a protein that blocks growth of skeletal muscle. Disable that gene, and viola--spectacular muscle growth results.
Over the past few years, pharmaceutical companies have been racing to develop ways to mimic myostatin gene mutations in the hope of treating everything from the muscle loss that accompanies muscular dystrophy, cancer, and aging to obesity and other metabolic disorders.
Pharmaceutical giants Wyeth and Amgen are expected to release clinical-trial results of myostatin inhibitors for muscle-wasting diseases within the next few months. A smaller company, Acceleron Pharma, based in Cambridge, MA, says that its more broadly acting drug could bring more brawn than can drugs targeting myostatin alone.