Sep. 9, 2013 — A preclinical study led by researchers in the United States has found that a new oral drug shows early promise for the treatment of muscular dystrophy. The results, which are published today in EMBO Molecular Medicine, show that VBP15 decreases inflammation in mice with symptoms similar to those found in patients with Duchenne muscular dystrophy. The authors found that the drug protects and strengthens muscle without the harsh side effects linked to current treatments with glucocorticoids such as prednisone.
Duchenne muscular dystrophy results in severe muscle degeneration and affects approximately 180,000 patients worldwide, mostly children. Treatment with the current standard therapy, glucocorticoids, can only be used for a short time due to serious side effects leading to fragile bones and suppression of both the immune system and growth hormone production.
The researchers also observed that VBP15 inhibits the transcription factor NF-κB, a key cell-signaling molecule found in most animal cell types that plays a role in inflammation and tissue damage.
The study authors previously found out 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.
"It is becoming increasingly clear that membrane integrity and repair are crucial factors in muscle, cardiovascular, neurodegenerative and airway disorders. The chemical properties of VBP15 also suggest potential for the treatment of other diseases." remarked Kanneboyina Nagaraju, DVM, PhD, the lead author of the study and a principal investigator in the Center for Genetic Medicine Research, Children's National Medical Center in Washington, DC.
The authors conclude that VBP15 merits further investigation for efficacy in clinical trials.
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."
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