Myostatin inhibitor trials on humans

As noted by the facioscapulohumeral muscular dystrophy (FSHD) society, myostatin inhibitor clinical trials have begun on humans. The first trials on humans actually started in Feb, 2005.

UPDATE: MYO-029 development has been dropped as it is safe but not effective. Boosting follistatin seems to be the better pathway to proceed for more effective results ith myostatin inhibition.

This is related to my article which indicated that testing on mice indicated that myostatin drugs had four times the muscle growth effect as high doses of steroids but with less side effects.

these are pictures of a German boy that naturally has the genes for inhibiting myostatin

There was also the discovery of a superstrong american boy who also has the natural genes for hypermuscles.

“He could do the iron cross when he was 5 months old,” said his adoptive mother, Dana Hoekstra of Roosevelt Park. She was referring to a difficult gymnastics move in which a male athlete suspends himself by his arms between two hanging rings, forming the shape of a cross.

Liam has the kind of physical attributes that bodybuilders and other athletes dream about: 40 percent more muscle mass than normal, jaw-dropping strength, breathtaking quickness, a speedy metabolism and almost no body fat.

Liam can run like the wind, has the agility of a cat, lifts pieces of furniture that most children his age couldn’t push across a slick floor and eats like there is no tomorrow — without gaining weight.

Liam Hoekstra was hanging upside down by his feet when he performed an inverted sit-up, his shirt falling away to expose rippled abdominal muscles. It was a display of raw power one might expect to see from an Olympic gymnast. Liam is 19 months old.

The so-called myostatin blockade has generated tremendous interest in the bodybuilding community. Some nutritional supplements claim to block myostatin, but researchers have said the claims are not scientifically valid.

“If the myostatin protein is knocked out, muscles grow and rejuvenate much more quickly,” Dr. Larson said. “It has potential for great abuse in the future as the new steroid.”

[Despite being born to a troubled mother who gave him up for adoption at birth and Liam being born with a suite of medical problems not related to the muscle genes.] Liam being born four weeks early and had a small hole in his heart. He also had eczema, enlarged kidneys, was lactose intolerant and had severe stomach reflux that made him vomit several times each day, his mother said. Two days after he was born, Liam could stand up and support his weight if someone held his hands to provide balance.

His is one of roughly 100 known cases in the world, according to experts and medical literature.

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The clinical study details

Each site will enroll 12 patients. 4 FSHD, 4 LGMD, 4 Becker MD.

Initial toxicity studies are done. There some side effects that are consistent with monoclonal antibody therapy.

This next stage of the trial is to determine dosing at three different levels and clinical efficacy.

Patients must be genetically confirmed.

The MSTN-inhibitor drug is administered via intravenous infusion.

Initial patient visit/transfusion may or may nor require an overnight stay. New infusion every other week for six months and three more months follow-up. Two muscle biopsies are optional.

Patients should have average muscle strength grade of 3. As general rule patient should be able to walk 30 feet unaided except for use of orthotic braces e.g. AFOs.

Phase I and phase II clinical trials are under way.

In Phase I clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.

In Phase II clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.

In Phase III studies, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.

One of the pathways to successful weight control will be increasing lean muscle mass myostatin inhibition by boosting follistatin. It could boost lean muscle mass a lot (30-50%). It makes exercise far more effective. Each pound of lean body mass, which includes skeletal muscle, burns a bit over 13 calories a day at rest. 910 calories from body fat in a week for an extra 10 pounds of muscle. It will take about 27 days to lose a pound of body fat (12 pounds per year, IF the person does not increase food/calorie intake). So with some cardio exercise it would be a substantial help for weight control.

It would definitely make the TNT diet more effective. Any health risks like possible increase in tendon injuries needs to be offset against health gains from improved weight control.

Other gene therapy and drugs can enhance endurance and longevity

Darpa is working on a variety of ways to augment soldiers.

A common enhancement that is performed now is the improvement of vision

Gene therapy has been used to enhance resistance to radiation.

The article on the muscle bound boy who naturally has the genes that inhibit myostatin.

Wikipedia on myostatin

MD Sports looks at different ways to enhance sports performance legally. They examine myostatin inhibitors.

A discussion of gene therapy for performance enhancement in sport.

“You can deliver the [gene] to neonatal animals or in utero,” says Dr. Jeffrey Medin, a biochemist who runs a gene-therapy lab at the Ontario Cancer Institute in Toronto. “As the animal ages, that [gene] gets distributed very nicely. If you wanted to provide long-term gene doping, that would be the time to start.”

But despite setbacks, gene therapy has produced far more successes than failures. “Gene therapy really hasn’t had that many negative effects,” notes Medin. “You look at bone-marrow- transplant patients in the 1970s, and until they figured it out everybody died. Here, we’ve had two or three patients out of 1,100 that have had severe consequences. If you’re looking at a new cancer drug, that’s an acceptable risk for a lot of people.”


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