2017 Stanford Drug Discover Conference

On Monday April 24, 2017 at Stanford University, a standing-room audience of entrepreneurs, venture capitalists, students, and researchers from academia and industry attended the second annual Stanford Drug Discovery Conference sponsored by the Stanford Cardiovascular and Cancer  Institutes and also the California Institute for Regenerative Medicine. This one-day event featured senior executives from the pharmaceutical industry such as Sean Harper, MD, executive vice president of Amgen, as well as local and invited speakers such as UCSF cardiovascular professor Shaun Coughlin, MD, PhD, and UT Southwestern stem-cell researcher in myocardial repair Eric Olsen, PhD, culminating in the keynote address by former FDA Commissioner Robert Califf, MD, now Professor of Cardiology at Duke University. Notable presentations were by Dr. Thomas Sudhof, MD, PhD, Professor, Stanford School of Medicine and 2013 Nobel Laureate about his current research of neuronal circuitry in the brain and the interactions of apolipoprotein E, a cholesterol-carrying protein in the brain, with a kinase cascade, which may be contributory to Alzheimer’s pathology as seen in murine models. His observations on this kinase pathway suggest that drugs targeting kinases for other diseases may need to be reviewed for selectivity to avoid potential unfavorable neuronal side effects. Shaun Coughlin, MD, PhD (UCSF) described his lab’s research on the platelet-initiated blood clot (thrombosis) cascade initiated by the protease-activated receptors (PARs) located on the surface of platelets and the inside surface of veins and arteries. He highlighted key differences between this cascade in mice, whose platelets express PAR3 and PAR4, and humans, whose platelets express PAR1 and PAR4. Consequently, researchers must be cognizant about extrapolating pathways and drug targets from mice to humans when conducting drug research.

The afternoon session was dedicated to “Bench to Bedside—Cancer therapies” in line with the focus of the conference being on the translation of basic research to drug research to help treat or even cure patients of their disease. Professor Alan Ashworth, PhD (UCSF), who contributed to the discovery of the inherited breast cancer gene BRCA2 (breast cancer 2), described the use of drugs targeting the DNA repair enzyme poly (ADP-ribose) polymerase to improve cancer treatment in patients who have inherited either the BRCA1 or BRCA2 mutation. Dr. Gideon Bollag, CEO of Plexxikon, presented on how they used their novel scaffold-based drug discovery platform to discover the melanoma drug Zelboraf® (vemurafenib), which targets a specific mutation in the BRAF gene that codes for a cell division signaling protein; the mutation is present in 60% of late-stage melanomas. Shivaani Kummar, MD, Director of the Phase 1 Clinical Research Program in Cancer at Stanford, presented the evolution and design of early phase clinical trials in drug development. Finally, the session ended with a thought-provoking speech by Dr. Robert Califf, who proposed the formation of a publically curated database for preclinical studies (e.g. animal studies) akin to the human clinical trial database www.clinicaltrials.gov. Additionally, he emphasized the need to make clinical trial data publically available, which has only been modestly accomplished by www.clinicaltrials.gov.

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