Advancing Biological Understanding and Therapeutics Discovery With Small-Molecule Probes

Cell. 2015 Jun 4;161(6):1252-65. doi: 10.1016/j.cell.2015.05.023.

Abstract

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.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Animals
  • Drug Discovery*
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacology
  • High-Throughput Screening Assays
  • Humans
  • National Institutes of Health (U.S.)
  • Small Molecule Libraries*
  • United States

Substances

  • Enzyme Inhibitors
  • Small Molecule Libraries