Investigational steroid mirrors prednisone’s benefits while taming its side effects

This would become a really big deal if the drug studies continue to show the same pattern….

Children’s-led research also reveals nine potential biomarkers for inflammatory diseases.

A head-to-head trial comparing the decades-old steroid, prednisone, and a promising new steroid, vamorolone, finds both act on the same key set of genetic pathways involved in controlling inflammation, indicates a new study led by Children’s National Health System researchers. However, the study suggests the new investigational steroid doesn’t activate several additional pathways involved in prednisone’s bevy of undesirable side effects.

The findings reveal a molecular mechanism behind vamorolone’s encouraging results in experimental models and human volunteers and eventually could lead to even better therapies for health problems that steroids are designed to treat.

“Our goal is to eventually have a new and improved steroid that replicates the efficacy of prednisone without its side effects,” says Christopher R. Heier, Ph.D., an assistant professor at Children’s Research Institute and senior study author. “Our results show that with vamorolone, we could be closing in on that goal.”

Prednisone long has been prescribed short-term for conditions that involve inflammation, such as allergicreactions, psoriasisasthma and bronchitis. However, for chronic conditions, such as Duchenne muscular dystrophy, prednisone is often prescribed for years or even decades, explains study lead author Alyson A. Fiorillo, Ph.D., also an assistant professor at Children’s Research Institute.

Over time, several serious side effects typically arise after prednisone use, including stunted growth, bone fragility and weight gain. But better alternatives don’t yet exist to help patients with Duchenne retain their strength and motor abilities.

Vamorolone, which was developed at Children’s National, has the potential to be a game-changer for patients who need to use prednisone long-term, say Fiorillo and Heier. Although preclinical models and now human trials have suggested that vamorolone could offer the same benefits as prednisone without the burden of its side effects, the mechanism behind these effects was unknown

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