In April, the FSH Society awarded Julie Dumonceaux PhD, or University College London, Institute of Child Health. a grant of $9,659.43 for one year for a project aimed at better understanding patients’ response to a class of drugs called myostatin inhibitors.
These drugs, such as Acceleron’s ACE-083 which is currently in a clinical trial in FSH muscular dystrophy patients, target myostatin, a molecule that the body produces naturally to inhibit muscle growth. The rationale for blocking myostatin is to enable muscle to achieve greater mass than it otherwise would. In conditions such as FSHD, where muscle mass is lost, a myostatin inhibitor would in theory enable muscle to re-grow without having to fight an uphill battle against the growth-blocking effects of myostatin.
“Muscle wasting is one of the biggest challenges in neuromuscular disorders. Myostatin being a negative regulator of muscle mass, its down-regulation has been seen as a promising tool to counterbalance this muscle wasting and at least 6 anti-myostatin molecules have been developed by pharmaceutical companies. However, so far, the clinical trials have been very disappointing and clinical endpoints have been barely reached. These results are surprising since during the phase 1 trials on healthy volunteers, an improvement of muscle mass was observed. ” writes Dr. Dumonceaux in the summary of her FSH Society grant proposal.
Dr. Dumonceaux states further that: “Several hypotheses have been proposed among them the poor efficacy of the anti-myostatin molecules or the specificity of the drugs themselves. In our study we are investigating another possibility based on the expression levels of several effectors of the myostatin pathway. Our experiments indicate that patient’ stratification (based on the expression of these effectors) might be useful to determine patient eligibility?
The funding provided by the FSH Society will help us to finish performing the experiments which may be of importance for neuromuscular patients, and FSHD patients in particular, and may deeply impact future and current clinical trials using myostatin inhibitors.”
Read more about grants funded by the FSH Society here.