MDA Awards $1.5 Million to Acceleron for DMD Drug Testing

MDA has awarded $1.5 million to Acceleron Pharma for continued testing of ACE-031, an inhibitor of myostatin and related proteins, in children with Duchenne MD

Article Highlights:

  • Acceleron Pharma has been testing its experimental compound ACE-031 in boys with Duchenne muscular dystrophy (DMD) in Canada.

  • ACE-031 is designed to act as a decoy to trap myostatin and related muscle-limiting proteins, thereby allowing for more muscle growth.

  • An ACE-031-like decoy has improved muscle growth and strength in DMD mice compared to untreated mice, and ACE-031 has increased muscle volume in healthy human volunteers.

  • The new MDA funding will aid development and testing of ACE-031 for DMD.

by Margaret Wahl on January 14, 2011 - 9:52am

MDA has begun funding tests of the experimental drug ACE-031 in children with Duchenne muscular dystrophy (DMD). The drug is being developed by Acceleron Pharma, a Cambridge, Mass., biotechnology company in collaboration with Shire, a global specialty biopharmaceutical company that focuses on developing, manufacturing and commercializing therapies for rare genetic diseases.

The Association announced Jan. 6, 2011, that it has awarded $1.5 million to Acceleron through its Venture Philanthropy drug development program.

About ACE-031

ACE-031 is designed to interfere with the actions of myostatin, a naturally occurring protein that limits muscle growth, and with other, related proteins that regulate muscle mass. It's designed to act as a "decoy receptor" that sticks to these proteins and inhibits their usual activities.

About Clinical Trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur. MDA has no ability to influence who is chosen to participate in a clinical trial. To learn more, see Understanding Clinical Trials and Being a Co-Adventurer, which is about neuromuscular disease clinical trials.

Mice with a DMD-like disease that were treated with a decoy receptor like ACE-031 showed larger and stronger muscles than their untreated counterparts; and healthy human volunteers treated with ACE-031 experienced modest increases in thigh-muscle volume (see Luring Away Myostatin Can Boost Muscle Size).

Acceleron is now testing ACE-031 in boys with DMD in a multicenter clinical trial in Canada. (See Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy for details and contact information.) MDA's support will help Acceleron complete the ongoing trial and a six-month extension study.

Meaning for people with DMD

The additional funding from MDA will help speed development and testing of ACE-031 as a possible treatment for DMD. Unlike strategies that target specific genetic mutations, ACE-031 is designed to preserve or perhaps even enhance muscle size and strength regardless of the specific DMD-causing genetic mutation. If it proves safe and effective, it may have widespread application to DMD and perhaps even other muscle diseases.


Editor's note 5/3/11: Trials of ACE-031 have been stopped pending resolution of safety issues. See ACE-031 Clinical Trials in Duchenne MD Stopped for Now.


Your rating: None Average: 4.3 (6 votes)
MDA cannot respond to questions asked in the comments field. For help with questions, contact your local MDA office or clinic or email See comment policy