An in vivo multiplexed small-molecule screening platform

Nat Methods. 2016 Oct;13(10):883-889. doi: 10.1038/nmeth.3992. Epub 2016 Sep 12.

Abstract

Phenotype-based small-molecule screening is a powerful method to identify molecules that regulate cellular functions. However, such screens are generally performed in vitro under conditions that do not necessarily model complex physiological conditions or disease states. Here, we use molecular cell barcoding to enable direct in vivo phenotypic screening of small-molecule libraries. The multiplexed nature of this approach allows rapid in vivo analysis of hundreds to thousands of compounds. Using this platform, we screened >700 covalent inhibitors directed toward hydrolases for their effect on pancreatic cancer metastatic seeding. We identified multiple hits and confirmed the relevant target of one compound as the lipase ABHD6. Pharmacological and genetic studies confirmed the role of this enzyme as a regulator of metastatic fitness. Our results highlight the applicability of this multiplexed screening platform for investigating complex processes in vivo.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Adhesion / drug effects
  • Cell Line, Tumor
  • Cell Movement / drug effects
  • Cell Proliferation / drug effects
  • Dose-Response Relationship, Drug
  • Drug Evaluation, Preclinical / methods*
  • Gene Knockdown Techniques
  • High-Throughput Screening Assays / methods*
  • Humans
  • Liver Neoplasms / metabolism
  • Liver Neoplasms / secondary
  • Mice
  • Mice, SCID
  • Molecular Imaging / methods*
  • Monoacylglycerol Lipases / antagonists & inhibitors
  • Monoacylglycerol Lipases / genetics
  • Neoplasm Transplantation
  • Pancreatic Neoplasms / metabolism
  • Pancreatic Neoplasms / pathology
  • Small Molecule Libraries / pharmacology*

Substances

  • Small Molecule Libraries
  • ABHD6 protein, human
  • ABHD6 protein, mouse
  • Monoacylglycerol Lipases