MDA Puts Muscle Behind Research

Pharmaceutical company receives $600K grant to support muscular dystrophy drug research

/ Author:  / Reviewed by: Joseph V. Madia, MD

(RxWiki News) Acceleron Pharma, Inc. has announced receipt of a $1.5 million grant awarded from the Muscular Dystrophy Association (MDA) to support clinical studies of the therapy ACE-031.

The biopharmaceutical company is developing ACE-031 to build muscle mass and increase strength by blocking proteins that impede muscle growth and strength.

Muscular dystrophy (MD) is a set of progressive, neuromuscular disorders that contribute to a loss of muscle tissue and strength.

ACE-031 is an investigational protein that impedes signaling through a cell surface receptor called activin receptor type IIB (ActRIIB). Blocking this signal may result in increased muscle mass and improved muscle strength and function, according to the research. In Duchenne muscular dystrophy, proteins such as GDF-8 (myostatin) bind to and activate ActRIIBm and thus limit the growth and regeneration of muscle.

MDA is looking to support ongoing clinical studies of ACE-031 in boys with Duchenne muscular dystrophy, an inherited form of the disease that results in rapid muscle weakness in young boys, usually below the age of six.

The clinical trials will help establish safe dosing and pharmcodynamic activity imperative to future studies.

The grant was awarded through the Association's Venture Philanthrophy Program (MVP), continuing MDA's $164 million in efforts to combat Duchenne muscular dystrophy.

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Review Date: 
January 24, 2011
Last Updated:
January 25, 2011