Where did drugs come from?

Published on September 23, 2013   37 min

Other Talks in the Series: Small Molecule Drug Discovery

Hello, this is David Swinney. I'm going to be talking to you about where drugs come from. I'm currently at the Institute for Rare and Neglected Diseases Drug Discovery, a non-profit recently formed to discover drugs for rare and neglected diseases. Before that, I was at Roche in Palo Alto for 20 years, where we did much of the work that we will talk about in this presentation.
Drug discovery is an iterative process starting with an unmet medical need and an idea to address that need. From that idea, you'll get a starting point, which could be a small molecule or an assay, which will then eventually lead to a drug candidate through an optimization process. From that drug candidate, it will then go into clinical testing initially with safety, proof-of-concept and finally, larger phase three studies leading to registration. What I've tried to show in this slide is how it's really an iterative process with many different cycles that feed back on each other, which can gain knowledge. You can actually measure this through different kinds of markers, biomarkers, the success or lack of success at each stage in the process.
A challenge to productivity is the time and investment required to identify medicines that are both efficacious and safe. Candidates for clinical trials will succeed or fail based on their own merits. Most candidates will fail. How can candidates be identified with a better chance for success? The intention with this work was to learn from past success. We've evaluated the role of how a medicine will work at the molecular level, as well as work to understand how medicines and the respective molecular mechanisms were discovered.