Anyone heard of ACE-031?

[shelly2612]

Was wondering if anyone has heard anything good or bad about this study?


ACE-031 (Muscle Growth)

 

[GlenBrittle]

Never heard of it.

I have to ask: Why bother when its the connection to the muscle that is failing from ALS. We lose muscle mass once its not being used anymore.

Maybe , some of the other MND's might benefit from it.

Glen

 

[shelly2612]

CAMBRIDGE, Mass. – September 10, 2009 - Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including red blood cells, bone, and muscle, today announced preliminary results from the ACE-031 Phase 1 single dose clinical trial demonstrating that ACE-031 increased lean body mass and muscle volume. The results from this randomized, placebo-controlled study were presented at the 14th International Congress of the World Muscle Society in Geneva, Switzerland

"We are thrilled that this ACE-031 Phase 1 clinical trial generated a substantial amount of encouraging safety data as well as clear signs of biological activity on muscle, bone and fat all following just a single dose of ACE-031,” said Matthew Sherman, M.D., Chief Medical Officer at Acceleron. “ACE-031 produced rapid, sustained and dose-dependent increases in lean body mass and muscle volume, increased biomarkers of bone formation and positively altered biomarkers of fat mass. Based on these results, we’ve initiated a Phase 1 multiple dose study and look forward to further development of ACE-031 for the treatment of patients suffering from neuromuscular diseases.”

Summary Results

* ACE-031 was well tolerated at all dose levels and demonstrated a linear pharmacokinetic profile with an average half-life ranging from 10-15 days
* Single doses of ACE-031 at 1 mg/kg and 3 mg/kg produced dose-dependent increases in lean body mass measured by dual energy X-ray absorptiometry (DXA) as early as day 15 that were sustained through day 57
* Subjects given single doses of placebo had a 0.2% decrease in lean body mass at day 57 compared to a 2.4% increase in subjects receiving 1 mg/kg ACE-031 and a 2.6% increase in subjects receiving 3 mg/kg ACE-031
* Subjects given single doses of placebo had a 0.2% decrease in muscle volume assessed by MRI at day 29 compared to a 3.5% increase in subjects receiving 1 mg/kg ACE-031 and 5% increase in subjects receiving 3 mg/kg ACE-031
* ACE-031 favorably affected biomarkers of fat mass (increased adiponectin and decreased leptin) and bone formation and resorption (increased bone-specific alkaline phosphatase (BSAP) and decreased C-terminal type 1 collagen telopeptide (CTX) at doses of 1 and 3 mg/kg

“ACE-031 holds great potential as a treatment for patients suffering from neuromuscular diseases such as muscular dystrophy and amyotrophic lateral sclerosis (ALS),” said John Knopf, Ph.D., Chief Executive Officer of Acceleron. “We are moving forward with great confidence and excitement with the ACE-031 program as well as our overall muscle franchise.”

Muscle is increasingly recognized as central to many biological processes and plays a major role in human health. The loss of muscle mass and strength is ultimately directly related to the cause of death in neuromuscular diseases such as muscular dystrophy and amyotrophic lateral sclerosis. Severe muscle loss in cancer leads to serious complications and a poor prognosis. Muscle loss is a natural consequence of aging, similar to bone loss, resulting in decreased muscle strength (frailty), reduced mobility and an increased risk of a fall and broken bones. In metabolic diseases, an imbalance of diet, energy utilization and skeletal muscle leads to poor metabolic function. By increasing muscle mass there is a corresponding decrease in fat mass and improvements in metabolic function.

About ACE-031

ACE-031, a soluble fusion protein based on the activin receptor type IIB (ActRIIB), is a biologic therapeutic that inhibits signaling through the ActRIIB receptor. By preventing signaling though ActRIIB, ACE-031 increases muscle mass and strength. In numerous and varied animal models of disease, ACE-031 significantly increased muscle mass and muscle strength. ACE-031, and a related molecule ACE-435, have shown encouraging preclinical results in animal models of age-related muscle loss, neuromuscular disease, cancer treatment-related muscle loss and metabolic diseases. ACE-031 is currently being studied in a Phase 1 multiple dose clinical trial in healthy volunteers. ACE-435 is expected to enter clinical trials in 2010.

 

[shelly2612]

Thats a good point Glen, just thought you all might be interested in something that, according to the company is supposedly going to be helpful in treating ALS

 

[BethU]

Sounds very exciting. Let's hope this works. Surely being able to build healthy muscle again in ALS would be significent. It might accomplish putting the patient on a plateau where fresh, healthy muscle fibers keep replacing damaged and dying muscle, and thus extending function.

I'm not a scientist and don't know how these processes really work, but anything that says "We are moving forward with great confidence and excitement with the program" and "It shows great potential for those with ALS" sounds good to me !

 

[Al]

Yes but being the skeptic that I am, didn't the guys in China, Mexico and many other 3rd world countries tell us the same things about stem cells? Show me results with REAL ALS patients and I'll jump on the bandwagon.

AL.

 

[Big Mike]

Even though it doesn't treat the underlying problem, as Glenn pointed out, this substance might have some potential as it could slow down muscle loss, thereby giving PALS functionality for a longer period of time. Maybe it targets the neuromuscular connection, which seems to be involved in the disease process.