Public Release: 

NIH helps UC San Diego researchers repurpose Sanofi pain drug for tropical disease

Researchers will evaluate existing Cathepsin S inhibitor as new potential Chagas disease therapeutic

University of California - San Diego


IMAGE: James McKerrow, M.D., Ph.D., is the director of the University of California, San Diego Skaggs School of Pharmacy and Pharmaceutical Sciences. view more

Credit: UC San Diego School of Medicine

The National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health (NIH), has awarded James McKerrow, MD, PhD, dean of the University of California, San Diego Skaggs School of Pharmacy and Pharmaceutical Sciences, with a 2015 New Therapeutic Uses Award. The grant will provide McKerrow and his team with SAR114137, a chronic pain drug owned by Sanofi, and support for testing the drug's efficacy in treating Chagas disease, a parasitic infection that is the leading cause of heart failure in Latin America.

NCATS' New Therapeutic Uses program matches researchers with a selection of pharmaceutical industry assets that have already undergone significant research and development, including safety testing in humans. When NCATS first shared its list of available industry-owned assets, Sanofi's SAR114137 immediately struck McKerrow as a potential treatment for Chagas disease. That's because previous studies showed that inhibitors of cruzain, a parasite-specific relative of Cathepsin S, can effectively treat the disease and prevent heart problems in infected animals. However, no data from human safety studies or clinical trials could validate the results in infected animals.

SAR114137 was originally designed to treat chronic pain but McKerrow thinks it may also be a good therapeutic candidate for Chagas disease. The drug has already undergone safety studies in humans.

Chagas is a neglected tropical disease that currently affects more than eight million people worldwide and is responsible for more than 10,000 deaths worldwide each year. It is an emerging infection in California.

"Yet there are currently no FDA-approved therapies for Chagas disease," McKerrow said. "We call Chagas a 'neglected' disease because pharmaceutical companies aren't interested in developing new therapies to treat it. This award will allow us to confirm SAR114137's antiparasitic activity in lab studies and in infected animals, then test its safety and effectiveness in humans with the disease. It's our hope that this work will lead to a more effective treatment for Chagas, thanks to NCATS' New Therapeutic Uses award."

According to NCATS, developing new therapeutics from scratch is a costly, complex and time-consuming process. The average length of time from target discovery to approval of a new drug is about 14 years. The failure rate during this process exceeds 95 percent, and the cost per successful drug can be $2 billion or more. The high therapeutic development failure rate means there are many existing, partially developed therapeutic candidates that could be repurposed for use in diseases other than those for which they were originally intended.

"By bringing together assets from pharmaceutical companies with new ideas from academic researchers, our New Therapeutic Uses program is aimed at producing new treatments much more quickly than starting from scratch," said Christine Colvis, PhD, director of Drug Development Partnership Programs.


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