Advancing biological understanding and therapeutics discovery with small-molecule probes

Stuart L. Schreiber, Joanne D. Kotz, Min Li, Jeffrey Aubé, Christopher P. Austin, John C. Reed, Hugh Rosen, E. Lucile White, Larry A. Sklar, Craig W. Lindsley, Benjamin R. Alexander, Joshua A. Bittker, Paul A. Clemons, Andrea De Souza, Michael A. Foley, Michelle Palmer, Alykhan F. Shamji, Mathias J. Wawer, Owen McManus, Meng WuBeiyan Zou, Haibo Yu, Jennifer E. Golden, Frank J. Schoenen, Anton Simeonov, Ajit Jadhav, Michael R. Jackson, Anthony B. Pinkerton, Thomas D.Y. Chung, Patrick R. Griffin, Benjamin F. Cravatt, Peter S. Hodder, William R. Roush, Edward Roberts, Dong Hoon Chung, Colleen B. Jonsson, James W. Noah, William E. Severson, Subramaniam Ananthan, Bruce Edwards, Tudor I. Oprea, P. Jeffrey Conn, Corey R. Hopkins, Michael R. Wood, Shaun R. Stauffer, Kyle A. Emmitte

Research output: Contribution to journalReview articlepeer-review

95 Scopus citations


Small-molecule probes can illuminate biological processes and aid in the assessment of emerging therapeutic targets by perturbing biological systems in a manner distinct from other experimental approaches. Despite the tremendous promise of chemical tools for investigating biology and disease, small-molecule probes were unavailable for most targets and pathways as recently as a decade ago. In 2005, the NIH launched the decade-long Molecular Libraries Program with the intent of innovating in and broadening access to small-molecule science. This Perspective describes how novel small-molecule probes identified through the program are enabling the exploration of biological pathways and therapeutic hypotheses not otherwise testable. These experiences illustrate how small-molecule probes can help bridge the chasm between biological research and the development of medicines but also highlight the need to innovate the science of therapeutic discovery.

Original languageEnglish (US)
Pages (from-to)1252-1265
Number of pages14
Issue number6
StatePublished - 2015

ASJC Scopus subject areas

  • General Biochemistry, Genetics and Molecular Biology


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