Activity-based proteome profiling of potential cellular targets of Orlistat--an FDA-approved drug with anti-tumor activities

J Am Chem Soc. 2010 Jan 20;132(2):656-66. doi: 10.1021/ja907716f.

Abstract

Orlistat, or tetrahydrolipstatin (THL), is an FDA-approved antiobesity drug with potential antitumor activities. Cellular off-targets and potential side effects of Orlistat in cancer therapies, however, have not been extensively explored thus far. In this study, we report the total of synthesis of THL-like protein-reactive probes, in which extremely conservative modifications (i.e., an alkyne handle) were introduced in the parental THL structure to maintain the native biological properties of Orlistat, while providing the necessary functionality for target identification via the bio-orthogonal click chemistry. With these natural productlike, cell-permeable probes, we were able to demonstrate, for the first time, this chemical proteomic approach is suitable for the identification of previously unknown cellular targets of Orlistat. In addition to the expected fatty acid synthase (FAS), we identified a total of eight new targets, some of which were further validated by experiments including Western blotting, recombinant protein expression, and site-directed mutagenesis. Our findings have important implications in the consideration of Orlistat as a potential anticancer drug at its early stages of development for cancer therapy. Our strategy should be broadly useful for off-target identification against quite a number of existing drugs and/or candidates, which are also covalent modifiers of their biological targets.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / metabolism
  • Antineoplastic Agents / pharmacology*
  • Cell Proliferation / drug effects
  • Dose-Response Relationship, Drug
  • Drug Design
  • Drug Screening Assays, Antitumor
  • Humans
  • Lactones / chemical synthesis
  • Lactones / chemistry
  • Lactones / metabolism
  • Lactones / pharmacology*
  • Molecular Conformation
  • Molecular Probes / chemical synthesis
  • Molecular Probes / chemistry
  • Molecular Probes / pharmacology
  • Orlistat
  • Proteome / chemistry
  • Proteome / metabolism*
  • Structure-Activity Relationship
  • Tumor Cells, Cultured
  • United States
  • United States Food and Drug Administration

Substances

  • Antineoplastic Agents
  • Lactones
  • Molecular Probes
  • Proteome
  • Orlistat