Drug DISCOVERY Specific Seminar – Donna Huryn, PhD

Pharmaceutical Sciences Seminar Series

(Drug DISCOVERY Specific Seminar)

• Donna Huryn, PhD
• University of Pennsylvania

Academic Drug Discovery: Playing to the Strengths to Address Challenging Targets and Unmet Medical Needs in Cancer

The academic setting provides unique opportunities to drug discovery scientists, as well as some challenges, compared to the more traditional industrial or biotech model.  Characteristics of a university setting, not usually associated with pharma are the ability to pursue high risk projects, a depth of expertise, infrastructure and capabilities in focused areas, and the willingness to pause to follow intriguing science.  This talk will highlight a cancer discovery projects that took advantage of the unique characteristics of an academic drug discovery environment.  Through the National Cancer Institute’s Chemical Biology Consortium, a team of scientists representing multiple institutions designed and optimized novel allosteric inhibitors of the AAA ATPase p97 (VCP), an exciting, emerging target in cancer.  We solved the first high-resolution structure of p97 bound to an inhibitor using cryo-electron microscopy, and further developed refined binding models of these molecules.  This exercise led to the observation of a previously unrecognized protein motif, a bis-threonine turnstile.  The team’s efforts have provided not only starting points for the development of new experimental therapeutics, but also valuable tool molecules that are being used to decipher the complex biological pathways regulated by p97 activity.

Hosted by Jennifer Golden