WHAT IS ACE-083?
ACE-083 is a locally active agent being researched by Acceleron in their neuromuscular therapeutic program. Acceleron is a research organization focused on the researching of TGF-beta protein superfamily of substances looking to discover and develop medicines that will improve the lives of patients experiencing a wide range of rare or serious diseases. The potential medications researched by Acceleron go through many clinical research trials to see whether or not they could be used to treat patients with unmet medical needs.
ACE-083 is being studied for its potential to treat muscle loss such as that which occurs with muscular dystrophies. The belief is that ACE-083 would increase strength and overall functioning of specific muscles to improve the quality of life of patients experiencing muscle loss or neuromuscular disease symptoms.
HOW DOES ACE-083 WORK?
Acceleron is studying the effects of using multiple TGF-beta targets in clinical research to increase strength and muscle mass in the muscle where the substance is administered. Research has shown that ACE-083 achieves results by preventing select proteins in the TGF-beta protein superfamily from reducing the growth of muscle. For example, myostatin (GFF8) works to stop the production of muscle, so if ACE-083 can prevent myostatin from activating, then muscle will continue to grow. During research trials, it’s also been discovered that ACE-083 does not affect untreated muscles or any other organs beyond the site of administering the injection. This reduces the potential for systemic side effects.
ACE-083 PHASE 1 AND 2 CLINICAL TRIALS
Healthy volunteers work with Acceleron to test results of using ACE-083. In Phase 1 trials, research shows measurable increases in muscle volume as a result of administration of ACE-083. The amount of muscle volume was directly related to the dose of ACE-083.
Results from Phase 1 trials lead to a two-par Phase 2 trial in test subjects experiencing muscle weakness in their biceps or tibialis anterior and a separate trial of test subjects suffering from reduced mobility and foot drop as a result of muscle weakness.
FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY (FSHD)
Individuals with FSHD, the most common of muscular dystrophies, experience trouble walking, running, and using their upper arm muscles. The disease affects 20,000 in the United States and eventually limits their ability to perform daily tasks most people take for granted, such as getting dressed or preparing a meal. Roughly 20% of people with FSHD will eventually require a wheelchair. At this time, there are no FDA-approved treatments or therapies. Treatment currently involves surgical methods or physical therapy.
Acceleron is hoping ACE-083 will become a potential treatment for FSHD, and much of the research on ACE-083 is for this purpose.
CHARCOT-MARIE-TOOTH-DISEASE
Other clinical research is to determine whether or not ACE-083 can be used to successfully treat Charcot-Marie-Tooth-Disease, an inherited neurological disorder that affects over 100,000 individuals in the United States. Individuals with the disease experience frequent falls as their lower leg muscles lose strength. As the disease progresses, they may also experience muscle atrophy in the feet and hands. With no FDA-approved treatments beyond physical therapy, surgery, or orthopedic equipment, Acceleron is researching ACE-083 to determine if it can become a medication for the disease.