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.
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.
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.