An independent analysis by researchers at The University of Western Australia has found that the synthetic steroidal drug vamorolone offers a potentially promising treatment for Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD), which occurs mainly in boys, is the most common childhood form of muscle wasting and is caused by a genetic error that prevents the body from producing dystrophin, a protein essential for maintaining muscle fibre strength and stability.
Vamorolone is particularly topical following the US Food and Drug Authority (FDA) approving its clinical use in the US a fortnight ago under the name AGAMREE®.
The Journal of Neuromuscular Diseases invited UWA's Professor Miranda Grounds to write an independent commentary on vamorolone in the context of DMD, regarding its merits, outcomes, and related aspects, compared with other glucocorticoids routinely used for treating DMD.
Emeritus Professor and Honorary Senior Research Fellow in UWA's School of Human Sciences, Professor Grounds has more than 40 years' experience investigating skeletal muscle cell biology with a strong focus on pre-clinical studies using animal models for muscular dystrophies including DMD, with the overall aim of clinical translation to treat such neuromuscular disorders.
UWA School of Human Sciences postdoctoral researcher and lecturer Dr Erin Lloyd contributed her expertise in physiology, muscular dystrophies, statistics and data analytics.
Dr Lloyd examined the clinical and pre-clinical data, tackling the challenge of synthesising the data to help visualise the major published findings from the ongoing vamorolone research and clinical trials.
Professor Grounds said their analysis found that vamorolone offered a promising steroid treatment for DMD, with similar efficacy and some reduced adverse side effects, in comparison with the traditional glucocorticoid steroid treatments for DMD, such as prednisone and deflazacort.
"Our findings showed some of the beneficial effects of vamorolone included reduced inflammation and improved muscle function, with less adverse effects on growth and bone quality, which are well-documented adverse effects of glucocorticoids," she said. "However, some adverse effects, such as adrenal suppression, were still evident."
Professor Grounds said an independent assessment of the research into vamorolone for DMD treatment was important for the worldwide DMD community, which includes young patients with DMD and their families, and many associated clinician/scientists, researchers and other experts.
"There is an urgent need for therapies and superior drugs to treat this lethal childhood disease, while vamorolone is also of potential interest for other muscular dystrophies," she said.
