Small Molecule Drug Discovery

Published September 2013 Updated October 2014 22 lectures
Dr. Michelle R. Arkin
University of California, San Francisco, USA

It is well known that the pharmaceutical industry has a productivity problem. Many reviews and news articles have pointed out the disconnection between the resources invested in research and development and the number and value of drugs coming out the other side. Nevertheless, there are still enormous unmet medical needs... read morethat could be served by having new and better drugs. This series will outline traditional and novel approaches to finding new drug leads, with an eye towards explaining how innovations in drug discovery research can help to bridge the productivity gap.

This series will focus on the early stages of small molecule drug discovery. For the sake of focus, we will not discuss biologic drugs (antibodies, therapeutic proteins, vaccines, and nucleic acids) because many of their discovery and development challenges are distinct from those of small molecule drugs. Drug discovery is a highly interdisciplinary, team-based research effort, and knowing the end game – clinical development – is critical to execute the early stages.

For these reasons, this series will include detailed explanations of actions a scientist needs to do, and conceptual overviews of topics (s)he needs to understand. Many of the presentations will center on the first phases of drug discovery – target selection, methods of hit identification and validation, hit-to lead chemistry, in vitro ADME and in vivo pharmacokinetics. In addition, the series will summarize the later stages of drug discovery and development (lead optimization, toxicology, and clinical trials).