DMD Treatment: A Promising Alternative to Glucocorticoids

Duchenne muscular dystrophy (DMD) is a rapidly progressive genetic disorder primarily affecting young boys, with symptoms appearing before age 6. According to a study published in JAMA Network Open, it is the most common form of childhood muscular dystrophy, affecting many male newborns worldwide.

Glucocorticoids (GCs), such as prednisone and deflazacort, are the only medications proven effective in clinical trials for treating DMD. However, these drugs come with various adverse effects, including weight gain, growth problems, behavior changes, and bone-related issues, which negatively impact the quality of life for individuals with DMD. Researchers emphasized finding alternative treatments with fewer side effects is critical in DMD research.

Vamorolone is a unique steroidal anti-inflammatory drug that differs from conventional GCs in its chemical structure and mechanism of action. Unlike GCs, vamorolone does not interact with certain receptors linked to side effects and is less likely to cause growth problems or insulin resistance.

Several clinical trials have were conducted to evaluate vamorolone's safety and effectiveness in boys with DMD. These trials showed that vamorolone treatment did not significantly change certain motor function measurements, such as standing time, over a 30-month period. However, it demonstrated similar efficacy to GCs in maintaining muscle function, and its safety profile appeared more favorable, with fewer adverse effects on growth and metabolic parameters. Vamorolone did not seem to negatively impact bone health but may lead to adrenal suppression with prolonged use.

Researchers noted the study's limitations, including its open-label design and the use of historical data for comparison. "A randomized double-blind vamorolone study (VBP15-004; NCT03439670) is near completion; results from this study will provide class I evidence for vamorolone efficacy and safety," said researchers.

Vamorolone shows promise as a potential treatment for DMD, offering a favorable safety profile compared to traditional GCs while maintaining muscle function. "Vamorolone appeared to be well-tolerated, with fewer safety concerns that are typically seen with standard-of-care GC treatment for ambulatory patients with DMD," they said.

Reference

Mah JK, Clemens PR, Guglieri M, et al. Efficacy and safety of Vamorolone in Duchenne muscular dystrophy: a 30-month nonrandomized controlled open-label extension trial. JAMA Network Open. 2022;5(1):e2144178-e2144178. doi:10.1001/jamanetworkopen.2021.44178