September 2013: VBP15 drug shows early promise for treatment of Duchenne muscular dystrophy

VBP15 drug shows early promise for treatment of Duchenne muscular dystrophy Skip to content Menu Medical Home Life Sciences Home Become a Member Search Medical Home Life Sciences Home About Functional Food News Health A-Z Drugs Medical Devices Interviews White Papers More... 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The results, published in EMBO Molecular Medicine , show that the drug, VBP15, decreases inflammation and 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. 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. Related Stories New laser-fabricated plasmonic chip accurately identifies and sorts cancer cells Proteomic signatures reveal early signs of cardiovascular-kidney-metabolic disease in children Standard pediatric blood pressure thresholds miss early trauma warning signs 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." Source: Children's National Medical Center Posted in: Medical Research News | Medical Condition News | Pharmaceutical News Comments (2) Download PDF Copy Suggested Reading Subcutaneous tarlatamab exhibits low toxicity rates in phase 1b trial Tumor DNA clearance reaches 39% in novel immunomodulator and ADC combination Childhood chemotherapy leaves lasting DNA damage in healthy cells Elusive stem cells offer clues to treat lumbar spinal stenosis Phase 2 study highlights flexible dosing options for tarlatamab in relapsed SCLC A large US study found no overall link between fluoride in pregnancy and children’s behavior Leicester's cardiovascular research hub shines spotlight on life-saving innovations Gut microbiome may reveal inflammation and future disease risk better than age Comments Sanjogita Gosavi on VBP15 drug shows early promise for treatment of Duchenne muscular dystrophy Sanjogita Gosavi says: December 31, 2013 at 9:19 AM Whether this drug VBP15 is used in older children of DMD ? my means at the age of 18 yrs 0 0 Reply Amaravathi Ganesham on VBP15 drug shows early promise for treatment of Duchenne muscular dystrophy Amaravathi Ganesham says: April 10, 2014 at 8:15 AM Hi, I am from India, one of my nephew (Age 10 Years) is suffering from this DMD? Can we use this VBP15? 0 0 Reply The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical. Cancel reply to comment Post a new comment Login (Logout) Quirky Comment Title Post Sign in to keep reading We're committed to providing free access to quality science. By registering and providing insight into your preferences you're joining a community of over 1m science interested individuals and help us to provide you with insightful content whilst keeping our service free. Sign in with Google Sign in with Apple Sign in with LinkedIn Sign in with Facebook or Sign in with email Trending Stories Latest Interviews Top Health Articles Psilocybin microdosing sounds promising, but the strongest studies tell a different story Coffee after colorectal cancer? 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