A new study conducted by researchers from the UK and US has revealed that Myostatin, a protein that blocks muscle growth, seems to be an effective potential therapeutic target for safe muscular dystrophy treatment.
The protein has shown promising results as a potential therapeutic target for treating muscular dystrophy in animal studies, and its inhibition resulted in increased muscle mass and strength.
The new study, which is the first to evaluate a myostatin inhibitor in patients, assessed its safety in adults with muscular dystrophy and found that it was well-tolerated.
The researchers conducted a double blind, randomized study of 116 patients with muscular dystrophy.
They divided patients with multiple different types of muscular dystrophy into four groups given sequentially higher doses of a myostatin inhibitor called MYO-029.
The researchers randomized each group to receive the test drug or a placebo in a 3:1 ratio.
The drug or placebo was given intravenously every two weeks for six months, after which the patients were followed for three months.
While the aim of the study was to test for safety, muscle strength and mass were also assessed.
The researchers found that safety assessments, including vital signs, laboratory tests and physical examination showed no significant differences between treatment and placebo groups.
They also found that there were no side effects to skeletal, smooth or cardiac muscle, and the most significant side effects related to the treatment were hypersensitivity skin reactions.
There was no increase in muscle strength or improvement in function during the nine months of the study, although muscle mass did increase in some of the patients.
Since the sample sizes in the different dosage groups were small, differences between the groups did not reach statistical significance.
"However, the consistency of the response to treatment in the various measures of effects on muscle tissue suggests that MYO-029 reached its intended target, producing a modest degree of muscle fiber hypertrophy and increased muscle mass in some treated subjects," said Kathryn R. Wagner of The Johns Hopkins University School of Medicine and her co-authors.
The researchers further said that larger studies over longer periods of time would be necessary to properly evaluate the efficacy of this new treatment.
"This trial supports the hypothesis that systemic administration of myostatin inhibitors provides an adequate safety margin for clinical studies, and these inhibitors should be evaluated for stimulating muscle growth in muscular dystrophy," the authors said.
The study was published in Annals of Neurology.