'Gentler' Corticosteroid Drug Shows Promise in DMD Mice

A drug that may provide the benefits of corticosteroid medications such as prednisone and deflazacort without some of their notorious side effects — growth retardation, bone loss and suppression of the immune system — has been shown to protect and strengthen muscles in mice with a disease that mimics Duchenne muscular dystrophy (DMD).

The findings were reported by a U.S.-based team of investigators who published their results online Sept. 9, 2013, in EMBO Molecular Medicine. MDA provided funding for this study.

The drug, known as VBP15, has been called a "gentler" corticosteroid (glucocorticoid), is being developed by ReveraGen BioPharma, a biotechnology company in Rockville, Md. It's gentler because its chemical structure is designed to eliminate or reduce several of the undesirable, harsh effects of prednisone and similar drugs.

In mice with a disease resembling human DMD, VBP15 treatment:

  • improved muscle strength;
  • decreased muscle inflammation;
  • preserved immune system function;
  • preserved growth; and
  • preserved bone structure.

Additional MDA support for VBP15

In May 2012, MDA awarded a $1.5 million grant to ReveraGen for development of VBP15 for DMD, a disease caused by a lack of the dystrophin protein, fragile muscle-fiber membranes and muscle inflammation.

In August 2013, the Association awarded $300,000 over three years to Jyoti Jaiswal at George Washington University in Washington, D.C., to investigate the possibility of using VBP15 to treat type 2B limb-girdle muscular dystrophy (LGMD2B) or Miyoshi myopathy. Both these conditions arise from a deficiency of the dysferlin protein and involve poor muscle-fiber membrane repair and muscle inflammation.

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