Beginning a study of ACE031 in DMD

Tests show the myostatin inhibitor ACE031 can safely increase muscle size.ACE031, a laboratory-modified necessary protein developed by Acceleron Pharma of Cambridge, Mass., has proven promise as a therapy to increase muscle mass, based on results of a trial in wholesome volunteers. The company will now test it in Duchenne muscular dystrophy (DMD).

Kenneth Attie, vice president for medical research at Acceleron, and colleagues, introduced the findings April 14, 2010, at the annual getting together with of the American Academy of Neurology (AAN), held in Toronto.

About ACE031

The investigational necessary protein ACE031 is developed to build muscle and increase strength by stopping the interaction of naturally happening muscle-inhibiting proteins, such as myostatin, with their docking websites (receptors) on muscle fibers.

Myostatin and other muscle-growth limiters normally dock on these muscle-fiber receptors and send signals through them.

ACE031 lures myostatin aside from its organic receptor and renders it powerless to limit the progress of muscles.

About the brand new findings

The investigators measured safety, tolerability, lean muscle mass, muscle amount and markers of bone and fat metabolism in 48 healthy, postmenopausal women who received a single subcutaneous injection of ACE031 or even a placebo.

ACE031 was provided at six diverse dosage levels. In each dosage group, six women received the drug and two others received a placebo injection.

In general, the drug was safe and nicely tolerated. There were no drug-related serious adverse events, and no 1 developed antibodies to ACE031 (which would have indicated rejection of the laboratory-engineered necessary protein through the immune system).

The long half-life of ACE031 in the system may permit dosing of only 1 or two injections every month, the researchers said.

In the team that received 1 milligram every kilogram of system weight of ACE031, there was an typical increase of 3.5 % in thigh-muscle volume. in the team that received 3 milligrams every kilogram of system weight of ACE031, there was an increase of thigh-muscle amount of as a lot as 5.1 percent.

There were increases in calf-muscle volume, but they were not as great, the investigators reported.

They also noted increases in biological markers of bone creation and decreases in biological markers of bone destruction in the ACE031-treated women in comparison for the placebo groups.

The investigators are now beginning a research of ACE031 in DMD. For details, see research of ACE-031 in Subjects With Duchenne Muscular Dystrophy.

Meaning for people with neuromuscular diseases

The drug has the potential to support individuals having a variety of neuromuscular diseases, simply because almost all these disorders include loss of muscle mass and strength.

Inhibitors of myostatin have received a lot interest from your neuromuscular disease research community, ever considering that it was discovered numerous years ago that people and animals having a genetic deficiency of myostatin seem to have large muscles and good strength with no apparent ill effects.

A few years ago, an experimental drug called MYO029, an antibody that blocks myostatin, was developed and tested by Wyeth Pharmaceuticals (now part of Pfizer). In 2008, the company announced the drug was safe and nicely tolerated but that it do not enhance muscle strength or function and do not increase muscle progress in trial participants.

ACE031 is identified as a diverse type of molecule from MYO029 and may prove more effective. Acceleron describes ACE031 as an ActRIIB signaling inhibitor, a molecule that blocks myostatin and related proteins from singaling through the ActRIIB receptor. (For details, see regularly inquired issues on Acceleron’s site.)

If tests in DMD are promising, it’s possible ACE031 could be developed for remedy for this and other neuromuscular diseases.

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