Methods and Compositions for the Treatment and Diagnosis of Statin-Induced Myopathy (BIDMC 1015)

Category:    Therapeutics - Drugs

KeyWords:  Biochemistry;  

BIDMC ID:    1015

Abstract:

Over 100 million patients worldwide are taking statins (HMG CoA reductase inhibitors) to lower cholesterol levels and thus reduce the risk of adverse cardiovascular events and coronary heart disease. Statin-mediated myopathy ranging from muscle pain to severe muscle damage is a debilitating complication of statin treatment. Currently, muscle toxicity of statins may prevent as many as half the patients with indications for statins from receiving this therapy. Investigators at BIDMC revealed that atrogin-1, a muscle-specific ubiquitin ligase involved in skeletal muscle atrophy, is a mediator of statin-mediated myopathy. The scientists suggest to treat and prevent statin-mediated myopathy by administration of atrogin-1 inhibitors (siRNA, antibodies, blocking peptides etc). Diagnosing a patient?s propensity for developing statin-mediated myopathy is suggested to be performed by measuring atrogin-1 levels in a patient and detecting possible atrogin-1 mutations. Investigators also suggest methods of identification of drug candidates for treatment of statin-mediated myopathy, as well as methods of identification of statin compounds having the propensity to induce a statin-mediated myopathy.

Inventor:   Lead Inventors: V.P. Sukhatme, S.H. Lecker

Commercial Opportunity:

1. Treatment and prevention of statin-mediated myopathy via inhibition of atrogin-1. 2. Diagnostics of statin-mediated myopathy and propensity to develop it via measurement of atrogin-1 level and detection of possible atrogin-1 mutations. 3. Identification of a compound for treatment of statin-mediated myopathy via measuring atrogin-1 level and activity in statin-treated cells (non)treated with a candidate compound. 4. Identification a propensity of a statin compound to induce a statin-mediated myopathy via measuring atrogin-1 level and activity in cells (non)treated with a statin compound.

Competitive Advantages:

Currently there are no known methods of statin-induced myopathy treatment, prevention or a patient susceptibility assessment.

Related Publications:

Hanai J, Cao P, Tanksale P, Imamura S, Koshimizu E, Zhao J, Kishi S, Yamashita M, Phillips PS, Sukhatme VP, Lecker SH.  The muscle-specific ubiquitin ligase atrogin-1/MAFbx mediates statin-induced muscle toxicity.  J Clin Invest.  2007; 117(12):3940-51.

State of Development:

Data to support the invention were obtained from humans with statin-induced myopathy, zebrafish, murine skeletal muscle cells, and cultured mouse myotubes. Technology licensing opportunities are available for the research and development of commercially valuable products.

Related Technology URL:

Patent Status:

US Provisional Application # 60/930774, PCT Application in progress

TVO Contact Info:
     Christine Jost
     Associate Director TVO
     cjost@bidmc.harvard.edu
     Phone: 617-667-4239   Fax: 617-667-0646

     Beth Israel Deaconess Medical Center
     Technology Ventures Office  Room: BR-0200
     330 Brookline Avenue
     Boston, MA 02215