Myostatin blocking still under hot pursuit

Acceleron, a Cambridge, Mass.-based biotech firm, and other companies are still pursuing myostatin blocking, which can be four times more effective at building muscle versus high doses of steroids

Se-Jin Lee, the molecular biologist at Johns Hopkins University who discovered myostatin in mice in 1992, says it’s “disappointing” that MYO-029 is dead, but he still believes blocking myostatin holds promise. But what really disappoints Lee is that discussion of a promising treatment for a devastating disease becomes entangled in discussions of doping. The benefits go far beyond the Duchenne muscular dystrophy, a disease that is diagnosed in only 600 American boys a year, to diseases like cancer and AIDS. Such drugs could even have a big effect on the muscle weakening that comes with aging.

“Everybody gets old; everybody is going to lose muscle mass,” Lee says. “If you look at the benefit of buying people five more years of independent living, it seems a little out of whack to be worrying about sports records.”

Acceleron and some other companies are working on several different drugs that hit myostatin. And Affymax (AFFY), a Palo Alto biotech firm, is working on what may be a cheaper, easier to use version of EPO. These are baby steps, but also reminders that someday, performance-enhancing drugs will be able to really push the limits of what the human body can do–like it or not.

Other drugs and enhancements
PPAR delta drugs Status: Experimental.
Legitimate use: Would fight obesity, heart disease.
Athletic advantage: Mice with the PPAR delta receptor modified can run twice as long as their unmodified brothers and sisters.
Side effects: Unknown, but PPAR drugs to treat diabetes have had unpredictable side effects

Gene therapy
Status: Experimental.
Legitimate use: Treating genetically inherited diseases.
Athletic advantage: Extra EPO, myostatin or other hormones created by DNA implanted within the body. Would be undetected by drug tests.
Side effects: Unknown. Gene therapy treatments use viruses or other biotechnology to alter DNA; in most attempts, risks have outweighed the benefits.

Robotic Limbs and prosthetics
Status: Early versions in development now.
Legitimate use: Allowing amputees to walk and run.
Athletic advantage: Prosthetics are now good enough that amputee athlete Oscar Pistorius will run in Beijing games.
Side effects: For amputees, an easy decision. But it will be a long time before able-bodied athletes are replacing perfectly good limbs

Status: In development.
Legitimate use: Allowing workers to carry very heavy loads or walk great distances.
Side effects: None, so far.