New Drug Reduces Fibrosis and Prevents DMD

by Bidita Debnath on Dec 11 2017 11:43 PM

New Drug Reduces Fibrosis and Prevents DMD
Duchenne muscular dystrophy (DMD) is a fatal form of a muscle wasting disorder that is caused by mutations in a gene on the X chromosome. Researchers have found a new drug that reduces fibrosis (scarring) and prevents loss of muscle function in an animal model of DMD.
The researchers from Saint Louis University (SLU) in the US, in a paper published in the Nature journal Scientific Reports, said this could provide a promising approach in designing new medications for those suffering from DMD.

With treatment, those with DMD have an average lifespan of around 25 years. Boys with the illness typically need to use a wheelchair by age 12 and require mechanical ventilation to help with breathing.

Many eventually suffer cardiac or respiratory failure.

Thomas Burris and Colin Flaveny from Saint Louis University aim to develop synthetic compounds to target these receptors in order to create drugs to treat diseases by understanding how the body’s natural hormones operate.

"Recently, we found that REV-ERB appears to play unique roles for each stage of muscle tissue development," Flaveny said.

REV-ERB regulates key processes in the body, from sleep to cholesterol, and, most recently, muscle regeneration.

The team showed that REV-ERB is a regulator of muscle differentiation and that a drug that inhibits this receptor, called SR8278, stimulates muscle regeneration after acute injury.

The research team also discovered that SR8278 increased lean mass and muscle function and decreased muscle fibrosis and muscle protein degradation in mice.

"These results suggest that REV-ERB is a potent target for the treatment of DMD," Burris said. "This is an encouraging finding as we search for better treatments for those with this debilitating illness."