BACKGROUND:  Muscular dystrophy (MD) is a group of genetic, degenerative diseases affecting muscles. There is no cure and no effective treatments and interventions for the disease. The disease will progress and become worse. In particular, an X-linked form of MD, Duchenne Muscular Dystrophy (DMD), occurs every 3,500 male births. However, there are no FDA-approved drugs or treatments for DMD. Therefore, there is a need for pharmaceutical targets for the treatments of MD.

INNOVATION:  Researchers at UCLA have identified a host factor that mediates the disease progression in muscular dystrophy. Abnormal levels of the factor were found both in MD patients and in the mdx mouse model of the disease. Furthermore, eliminating this factor has been shown to ameliorate the disease in muscular dystrophic mice. Therefore, this host factor is a potential therapeutic target for the treatment of muscular dystrophy, and also other human diseases causing muscular atrophy, such as amyotrophic lateral sclerosis (ALS) or those that cause muscle inflammation and scar tissue formation such as myositis and limb girdle muscular dystrophy.

POTENTIAL APPLICATIONS 

• A pharmaceutical target for the treatment of muscular dystrophy.
• A pharmaceutical target for the treatment of other muscular atrophy conditions, such as ALS.
• A pharmaceutical target for diseases that involve the development of scar tissue.

ADVANTAGES

• A new lead, filling the blank in the drug discovery for the treatment of muscular dystrophy.

DEVELOPMENT-TO-DATE:  The UCLA researchers have demonstrated the proof-of-concept of the discovery using an in vivo mouse model of MD.

Related Papers (Selected)

• Vetrone SA, Montecino-Rodriguez E, Kudryashova E, Kramerova I, Hoffman EP, Liu SD, Miceli MC, Spencer MJ. Osteopontin promotes fibrosis in dystrophic mouse muscle by modulating immune cell subsets and intramuscular TGF-beta. J Clin Invest. 2009 May 18. pii:37662. doi:10.1172/JCI37662. [Epub ahead of print] [more]

Reference: UCLA Case No. 2008-733