Super Strength Substance (Myostatin) Closer to Human Trials

Get muscles now, ask me how. As published in the journal Science Translational Medicine, researchers at the National Children’s Hospital (NCH) and Ohio State University have proven that blocking myostatin in monkeys will lead to skeletal muscle growth with few or no discernible negative side effects. Myostatin is the protein that helps mammals regulate muscle building, acting as a signal for muscles to stop consuming resources and stop growing. Blocking myostatin leads to enhanced muscle strength and continuous muscle growth. You may remember Liam Hoekstra, the baby apparently born without the myostatin gene, and similarly enabled animals that have absurd strength. Using gene therapy, NCH scientists were able to get follistatin (a myostatin blocker) to promote phenomenal muscle growth in the quadriceps of macaque monkeys. NCH is now working with the FDA to perform the preliminary steps necessary for a human clinical trial. We could see a superman gene therapy available in the next decade.

The National Children’s Hospital interest in myostatin is not to create super strong children, but to help those children whose muscles have already atrophied. Muscular Dystrophy (MD) affects thousands of children in the US who slowly lose muscle and rarely survive into adulthood. Follistatin gene therapy could serve as a method to extend their lives or perhaps even reverse the symptoms of their conditions. Likewise, the eldery are susceptible to several diseases that lead to a loss of muscle strength and coordination. By blocking myostatin, we may all be able to live with the strength of our youth even as we age into our 80s.

My concerns about myostatin have largely focused on potential organ damage, possible unknown dangerous effects on smooth muscle tissue, and ligament/tendon stresses. The NCH work addresses these concerns rather well. Macaques were observed for 15 months after receiving a gene therapy that promoted follistatin (and blocked myostatin) in their quadriceps. There was no observed damage to internal organs, the treatment only seemed to affect skeletal muscle, the reproductive cycles and cells functioned normally, and there was no reported damaged to tendons or ligaments (though this last issue wasn’t expressly pursued by the research).

As with all animals we’ve seen with myostatin inhibition, the monkeys at NCH enjoyed some serious boosts in strength. The macaques exhibited enhanced muscle growth for 12 weeks after treatment, beyond which muscle mass stabilized. The average circumference of the animals quadriceps increased by 15%. Using electric stimulation (you can’t order a monkey to lift weights) scientists were able to observe profound increases in leg strength. One specimen demonstrated a 78% increase over control results.

The next step for NCH is toxicology and biodistribution tests as outlined by the FDA. After that, further rounds of testing will be necessary before human clinical trials can begin. Still, considering the lack of negative side effects, and the profound need from the Muscular Dystrophy community, one hopes that those trials will begin in the next few years. If ultimately successful, follistatin gene therapy could treat the symptoms of MD while different genetic therapies could affect the causes of some forms of MD. And let’s not forget the weight-lifting 800 pound gorilla in the room. Along with MD treatments, and therapies for muscle loss in old age, the defeat of myostatin could lead to an effective way for all of us to get fit fast. No workouts necessary, eat almost all you want, and have a body like Adonis. This research is taking us one step closer to that goal…but it still sounds too good to be true. We can’t all be Superman, can we?

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