Lead Selection & Optimization

This course covers the stages of drug discovery in which promising compounds (leads) are identified, their optimal and suboptimal characteristics determined, and the suboptimal characteristics optimized to create a candidate suitable for the clinic.  The course is built around a case study on the discovery of an antimalarial therapy.  Assays useful for screening new compounds are outlined, examples of selection criteria for leads are discussed - such as potency, efficacy, and pharmacokinetics - and the structure activity relationships that contribute to the optimization of a lead series are considered.  The phenotypic approach used by the antimalarial program will be briefly contrasted with a target-based program on a different target. Target audience: This course is suitable for life scientists, clinicians, and individuals from fields that support drug discovery (e.g., patents, finance, licensing, etc.) interested in learning more about the pharmaceutical/biotechnology sector. Advanced undergraduate coursework or practical familiarity/working knowledge in biological sciences and organic chemistry is recommended.

• Lead Selection & Optimization, part 1
• Welcome, by the end of the course students will be able to: Recognize malaria as a neglected tropical disease and understand its significance as an unmet medical need. Identify and describe some of the characteristics an effective small molecule therapeutic should possess, and liabilities to avoid. Describe the principles and common approaches used in defining and optimizing a lead compound's pharmacophore.
• Lead Selection & Optimization, part 2
• Lead Selection & Optimization, part 3