Center for Clinical and Translational Science

On June 28, 2016, the Clinical Scholars and REPPS (Rockefeller Early Phase Physician Scientists) met with President Marc Tessier-Lavigne for an evening of questions and answers on drug development. The evening was the inaugural tutorial of the new Entrepreneurship Program directed by Dr. Sarah Schlesinger and Dr. Barry Coller. The program aims to foster knowledge of the complex process of turning scientific discoveries into therapeutics. Dr. Tessier-Lavigne’s insights into this process, which reflect his many years of experience in academia, the biotechnology and pharmaceutical industries and entrepreneurship, provided an ideal introduction to understanding the entire translational research continuum, leading to a product that improves the health and lives of the public.

Dr. Tessier-Lavigne focused on the importance of partnering with the right people, and that this should be based on both their experience and on personal chemistry. “When finding a company to work with, choose people who have been around the block. Find trusted colleagues who have genuine experience whom you can trust.” Through his experience he highlighted three key points in drug development where physician scientists can have a unique and significant impact. Finding the right target: This is achieved through a triangulation process of studying human samples, genetics, and mouse models. Finding the right drug: By developing drugs for new targets or re-purposing existing drugs. Finding the right patient: Identifying the right population of patients to treat. A striking example of this point was the fact that Herceptin was only beneficial in treating patients with Her2 expression on their cancer cells.  In each case combining medical knowledge with scientific insights is synergistic in making the best choices.

From the entrepreneurial point of view, Dr. Tessier-Lavigne stressed the importance of finding venture capitalists with a proven track record in the industry. He also noted that single target, single drug companies represent the greatest risk. Companies with platform technologies or multiple potential targets to address a disease or clinical problem have a greater likelihood of success and are thus more likely to be able to secure funding.

Despite the great improvements during the last 20 years, translating scientific discoveries into therapeutics is a challenging, costly and often lengthy process, requiring a broad array of expertise. Generating novel chemical matter is not easy and therapeutic targets have become more and more complex, making the field challenging but also very exciting. Dr. Tessier-Lavigne urged the Scholars and REPPS to focus on the solvable in their work, and to utilize their role as physician-scientists to help build bridges between the scientific and clinical communities. “Breakthroughs”, he noted, “come from careful and persistent application of expert knowledge.”