New muscular dystrophy treatment shows promise in early study

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 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-kB, 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-kB 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 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.

More information: VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects, DOI: 10.1002/emmm.201302621

add to favorites email to friend print save as pdf

Related Stories

Researchers review muscular dystrophy therapies

Jun 22, 2012

Leading muscular dystrophy researcher Dean Burkin, of the University of Nevada School of Medicine summarizes the impact of a new protein therapeutic, MG53, for the treatment of Duchenne muscular dystrophy ...

Protein injection points to muscular dystrophy treatment

Nov 27, 2012

Scientists have discovered that injecting a novel human protein into muscle affected by Duchenne muscular dystrophy significantly increases its size and strength, findings that could lead to a therapy akin to the use of insulin ...

New breakthrough could help treat muscular dystrophy

Aug 13, 2012

A researcher in the Faculty of Medicine & Dentistry at the University of Alberta improved Duchenne muscular dystrophy symptoms in non-human lab models, using a new drug cocktail. The drug combination targets ...

Recommended for you

New biomedical implants accelerate bone healing

4 hours ago

A major success in developing new biomedical implants with the ability to accelerate bone healing has been reported by a group of scientists from the Department of Restorative Dentistry, University of Malaya. ...

User comments