Science in the Triangle

Large pharmaceutical companies already leave much of the translational research to biotech companies and startups. But now, turning an idea into a potential product is gaining importance at U.S. medical schools as more and more university scientists are taking on the development of disease treatments and preventions.

In North Carolina, researchers at Wake Forest University are about to test a novel vaccine booster in healthy volunteers. The New England Journal of Medicine this month published the results of the first clinical trial of a therapy developed at the University of North Carolina at Chapel Hill to replace a defective gene that causes Duchenne’s muscular dystrophy. And Duke University researchers have come up with treatments for two rare diseases, Krabbe disease and Pompe disease, and are working on three more.

Dr. Priya Kishnani

The three scientists that the Raleigh-based Carolinas Chapter of The Indus Entrepreneurs invited to its life science panel discussion Tuesday at Brier Creek Country Club reflected not only this research & development shift, but as women they also succeeded in a male-dominated field.

One of the panel members was Dr. Priya Kishnani, a Duke pediatrician and geneticist, who was instrumental in developing Myozyme, a Pompe disease treatment that was approved in 2006 and is marketed by Genzyme.

Prabhavathi Fernandes

Kishnani was joined by Prabhavathi Fernandes, chief executive of Cempra Pharmaceuticals, and Christy Shaffer, former chief executive of Inspire Pharmaceuticals.

Christy Shaffer

Research Triangle Park was established to bring together academia and industry and develop research-based products. In that respect, Cempra, a 4-year-old Chapel Hill startup that has raised $60 million in venture capital to develop new antibiotics, and Inspire, a publicly traded Durham company with about $100 million in annual revenue, are driving forces in the home-grown life cycle of drug development.

The trio talked about what inspires them, whether they believe in an entrepreneurial gene and what’s unique about translational research in RTP. They also fielded questions from the audience, including one from Leslie Alexandre, former chief executive of the N.C. Biotechnology Center, on pricing of new medicines in the face of rising health care costs.

What inspires them to press on?

Kishnani: Compassion for children who are born with rare genetic defects that are deadly and the desire to present parents hope, not just the number of months their children might live.

Fernandes: Taking ownership of a good idea and becoming its champion all the way to the pharmacy shelf.

Shaffer: Mentors and a good support system are very important.

Is there such a thing as an entrepreneurial gene?

Shaffer: To succeed in translational research requires tenacity and the willingness to take risks.

Fernandes: “You have to be an optimist.”

Kishnani: It takes certain characteristics to believe in a new treatment that is in development 10 years or 15 years. Call it an entrepreneurial gene if you want to.

Is there anything unique about doing translational research in the RTP area?

Kishnani, Shaffer, Fernandes: Yes. The atmosphere here is less competitive than in other R&D hot spots, such as Boston. The collaborative environment provides a unique ecosystem to start companies.

Is drug pricing out of whack?

Alexandre: New drugs that prolong patients’ lives for a few months at a cost of $50,000 or $100,000 become controversial when health spending makes up more than 17 percent of the gross national product ( the value of all the goods and services the U.S. produces in one year). Yes, we’re all living longer and need more medicines, but what are patients in their 80s willing to pay for another month of life?

Kishnani: The Orphan Drug Act protects companies that sell treatments for rare diseases from competition for seven years. That’s why there’s a lot of interest in so-called orphan drugs (treatments for diseases that affect 200,000 patients or fewer). Pricing is not an issue.

Shaffer: Champions a reward system for lifestyle changes to prevent chronic diseases, such as Type 2 diabetes, and decrease the need for expensive treatments. Also, the industry is under severe price pressures. About 80 percent of the drugs on the market will be cheaper generics next year. That’s a huge shift from about a decade ago, when generics made up about 20 percent of all drugs on the market.

Fernandes: “Competition is the best for pricing.”