Discovery of novel drugs for promising targets.

Abstract Background Once a promising drug target is identified, the steps to actually discover and optimize a drug are diverse and challenging. Objective The goal of this study was to provide a road map to navigate drug discovery. Methods Review general steps for drug discovery and provide illustrating references. Results A number of approaches are available to enhance and accelerate target identification and validation. Consideration of a variety of potential mechanisms of action of potential drugs can guide discovery efforts. The hit to lead stage may involve techniques such as high-throughput screening, fragment-based screening, and structure-based design, with informatics playing an ever-increasing role. Biologically relevant screening models are discussed, including cell lines, 3-dimensional culture, and in vivo screening. The process of enabling human studies for an investigational drug is also discussed. Conclusions Drug discovery is a complex process that has significantly evolved in recent years.