Discovery of potent and selective benzothiazole hydrazone inhibitors of Bcl-XL

J Med Chem. 2013 Jul 11;56(13):5514-40. doi: 10.1021/jm400556w. Epub 2013 Jun 26.

Abstract

Developing potent molecules that inhibit Bcl-2 family mediated apoptosis affords opportunities to treat cancers via reactivation of the cell death machinery. We describe the hit-to-lead development of selective Bcl-XL inhibitors originating from a high-throughput screening campaign. Small structural changes to the hit compound increased binding affinity more than 300-fold (to IC50 < 20 nM). This molecular series exhibits drug-like characteristics, low molecular weights (Mw < 450), and unprecedented selectivity for Bcl-XL. Surface plasmon resonance experiments afford strong evidence of binding affinity within the hydrophobic groove of Bcl-XL. Biological experiments using engineered Mcl-1 deficient mouse embryonic fibroblasts (MEFs, reliant only on Bcl-XL for survival) and Bax/Bak deficient MEFs (insensitive to selective activation of Bcl-2-driven apoptosis) support a mechanism-based induction of apoptosis. This manuscript describes the first series of selective small-molecule inhibitors of Bcl-XL and provides promising leads for the development of efficacious therapeutics against solid tumors and chemoresistant cancer cell lines.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Apoptosis / drug effects*
  • Benzothiazoles / chemical synthesis
  • Benzothiazoles / metabolism
  • Benzothiazoles / pharmacology*
  • Binding, Competitive
  • Cell Line, Tumor
  • Cells, Cultured
  • Drug Discovery
  • Embryo, Mammalian / cytology
  • Fibroblasts / cytology
  • Fibroblasts / drug effects
  • Fibroblasts / metabolism
  • Hydrazones / chemical synthesis
  • Hydrazones / metabolism
  • Hydrazones / pharmacology*
  • Kinetics
  • Mice
  • Mice, Knockout
  • Models, Chemical
  • Molecular Structure
  • Myeloid Cell Leukemia Sequence 1 Protein / deficiency
  • Myeloid Cell Leukemia Sequence 1 Protein / genetics
  • Surface Plasmon Resonance
  • bcl-2 Homologous Antagonist-Killer Protein / deficiency
  • bcl-2 Homologous Antagonist-Killer Protein / genetics
  • bcl-2-Associated X Protein / deficiency
  • bcl-2-Associated X Protein / genetics
  • bcl-X Protein / antagonists & inhibitors*
  • bcl-X Protein / chemistry
  • bcl-X Protein / metabolism

Substances

  • Benzothiazoles
  • Hydrazones
  • Mcl1 protein, mouse
  • Myeloid Cell Leukemia Sequence 1 Protein
  • bcl-2 Homologous Antagonist-Killer Protein
  • bcl-2-Associated X Protein
  • bcl-X Protein
  • benzothiazole