New muscular dystrophy treatment shows promise

A potential new treatment for the condition muscular dystrophy could be in the works, after early study results showed promise.

The preclinical study, led by researchers at Children’s National Medical Center, found that a new oral drug known as VBP15 shows early promise for the treatment of Duchenne muscular dystrophy (DMD), by decreasing inflammation and protecting and strengthening muscle, without the harsh side-effects that are linked to current treatments with glucocorticoids.

The results have been published in EMBO Molecular Medicine and, though preliminary, are very promising for advancing the treatment of this disease, which causes disability in thousands of children across the world, explained Dr Eric Hoffman, 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," he added.

The team also observed that VBP15 inhibits the transcription factor NF-kB, a key cell-signaling molecule found in most cell types that plays a role in inflammation and tissue damage.

They had previously found that NF-kB is active in dystrophin-deficient muscle years before the onset of symptoms, suggesting that very early treatment of DMD patients with VBP15 may delay or even prevent the onset of some clinical symptoms.

Dr Kanneboyina Nagaraju, lead author of the study, and principal investigator in the Center for Genetic Medicine at Children’s National, explained that VBP15 has an additional property of addressing membrane defects in dystrophic muscle cells.

He added: "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.

"Increasing pre-clinical data suggests that VBP15 may show efficacy in these other disorders and in many other indications where steroids are used."

Initial clinical trials of VBP15 are planned, pending Food and Drug Administration (FDA) review and approval, which is anticipated to occur in 2014.