New research insights hold potential for kids with DMD

Experimental drug shows promise for simultaneously treating inflammation and heart disease in trial models. The drug in question called Vamorolone, does so by simultaneously targeting two nuclear receptors important in regulating inflammation and cardiomyopathy

Prednisone, the current standard of care used to treat kids with Duchenne muscular dystrophy (DMD), reduces chronic inflammation but has harsh side effects. Eplerenone, a heart failure drug, is used in older patients to treat cardiomyopathy, a leading cause of mortality for people with DMD.

The new medicine under development appears to combine the beneficial effects of Prednisone and Eplerenone, these drugs for the heart and muscle while also showing improved safety in experimental models.

DMD is a progressive X-linked disease that occurs mostly in males. It is characterized by muscle weakness that worsens over time, and most kids with DMD will use wheelchairs by the time they’re teenagers.

DMD is caused by mutations in the DMD gene, which provides instructions for making dystrophin, a protein found mostly in skeletal, respiratory and heart muscles.

Cardiomyopathy is used as an umbrella term for diseases that weaken the heart, it’s a leading cause of death for young adults with DMD, causing up to 50 per cent of deaths in patients who lack dystrophin.

Experts have found genetic dystrophin loss provides “a second hit” for a specific pathway that worsens cardiomyopathy in experimental models of DMD.

“Some drugs can interact with both the mineralocorticoid receptor (MR) and glucocorticoid receptor (GR) since these two drug targets evolved from a common ancestor.

“However, we find these two drug targets can play distinctly different roles in heart and skeletal muscle. The GR regulates muscle inflammation, while the MR plays a key role in heart health,” say experts.

The experimental drug Vamorolone is currently in Phase IIb clinical trials and is particularly exciting for its unique potential to simultaneously treat chronic inflammation and heart pathology with improved safety

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