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Advancing Biological Understanding and Therapeutics Discovery with Small-Molecule Probes

期刊

CELL
卷 161, 期 6, 页码 1252-1265

出版社

CELL PRESS
DOI: 10.1016/j.cell.2015.05.023

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资金

  1. NIH [U54 MH084681-01, 1 U54 HG005033-01, U54 MH084512-01, 1 U54 HG005032-01, 1 U54 HG005034-01, U54 MH084690-01, U54 MH084691-01, 1 U54 HG005031-01, U54 MH084659-01]
  2. Janssen
  3. Bristol-Myers Squibb
  4. AstraZeneca

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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.

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