New muscular dystrophy treatment shows promise in early study

11-Sep-2013 - Germany

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 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.

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.

Original publication

Christopher R. Heier, Jesse M. Damsker, Qing Yu, Blythe C. Dillingham, Tony Huynh, Jack H. Van der Meulen, Arpana Sali, Brittany K. Miller, Aditi Phadke, et al.; "VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects."; EMBO Molecular Medicine

https://www.bionity.com/en/news/144898/new-muscular-dystrophy-treatment-shows-promise-in-early-study.html