Category:

New Use Therapeutic

Description:

The well known drug rapamycin is a fungal macrolide that inhibits mammalian target of rapamycin (mTOR) (See Figure), an important molecule for both growth and survival signaling. Rapamycin is currently used to inhibit inflammation during surgery, however its utility in other therapeutic areas is limited due to its toxicity. Studies have shown both beneficial and harmful effects of administering rapamycin in the heart. The present invention, developed by inventors at the Medical University of South Carolina (MUSC), relates to the use of rapamycin to treat pressure overload hypertrophy, a symptom of a cardiomyopathy. Hypertrophy of cardiac tissue initially compensates for the increased pressure, however it ultimately results in heart failure. The present invention determines specific effective dosages and time intervals of rapamycin administration that result in a beneficial effect and eliminate most or all negative effects. Using a relatively small dose of rapamycin, inventors at MUSC have augmented survival signaling via increased Akt activation and increased clearance of deleterious proteins via the ubiquitin proteasome system (in vivo and in vitro data is attached). The goal is to switch the growth response of hypertrophy to be purely physiological and therefore eliminate or reduce the risk of heart failure. This invention seeks to define the parameters of rapamycin administration as a therapy to reduce the development of heart failure. There are also other rapamycin derivatives and analogs with no known therapeutic effect. These derivatives and analogs may also be useful as part of the present invention. Since rapamycin is not currently used to treat heart conditions, the present invention is a novel and non obvious use for rapamycin and its derivatives.

Potential Applications:

• First test of its kind.
• New Use for Known (and FDA approved) Compound.
• Large Potential Population to be Treated

Inventor(s):

D. Kuppuswamy, Ph.D., and R. Johnston, Ph.D.

Patent Status:

Provisional Application Filed, February 12, 2009

Availability:

Available for:
exclusive
non-exclusive
licensing.

Technology Status:

Licensing Contact:

Ryan N. Fiorini, Ph.D., MBA, MHA
MUSC Foundation for Research Development
PO Box 250828
Charleston, SC 29425
843.876.1906
fiorinir@musc.edu