New muscular dystrophy treatment shows promise

Duchenne muscular dystrophy results in severe muscle degeneration and affects approximately 180,000 patients worldwide, mostly children. The current standard treatment uses glucocorticoids, but is limited due to serious side effects leading to fragile bones and suppression of both the immune system and growth.

"These findings, while preliminary, are very promising for advancing the treatment of this disease, which causes disability in so many children worldwide," said Eric P. Hoffman, PhD, director of the Center for Genetic Medicine Research at Children's National. "The study also suggests the potential for new strategies in very early treatment, which could further benefit patients." The Children's National research team also observed that VBP15 inhibits the transcription factor NF-κB, a key cell-signaling molecule found in most cell types that plays a role in inflammation and tissue damage. The team previously found that NF-κB is active in dystrophin-deficient muscle years before the onset of symptoms, suggesting that very early treatment of Duchenne muscular dystrophy patients with VBP15 may prevent or delay the onset of some clinical symptoms.

"VBP15 has an additional property of addressing membrane defects in dystrophic muscle cells," remarked Kanneboyina Nagaraju, DVM, PhD, the lead author of the study, and principal investigator in the Center for Genetic Medicine at Children's National. "It is becoming increasingly clear that membrane integrity and repair are crucial factors not only in muscle, but also in cardiovascular, neurodegenerative, and airway disorders. In-creasing pre-clinical data suggest that VBP15 may show efficacy in these other disorders and in many other indications where steroids are used." Dr. Nagaraju noted.

Initial clinical trials of VBP15 are planned, pending Food and Drug Administration (FDA) review and approval, anticipated in 2014. ReveraGen Biopharma, Inc., the first Children's National private spin-off company, is working closely with Children's National investigators to translate the pre-clinical findings to DMD patients. ReveraGen Biopharma collaborated with NIH/TRND on VBP15, which was selected through a national com-petitive process, one of the few inaugural compounds selected through this novel pro-gram.

"The partnership with NIH and support from many others is illustrative of the importance of private-public partnerships that are essential in expediting the development of orphan disease therapeutics," remarked Edward Connor, MD, CEO, and CMO of ReveraGen Biopharma and Director of Innovation Development at Children's National. "We are optimistic that the new drug will translate well to patients, and are moving this forward as quickly as possible, while assuring safety."