Pharmacophore Identification and Scaffold Exploration to Discover Novel, Potent, and Chemically Stable Inhibitors of Acid Ceramidase in Melanoma Cells

J Med Chem. 2017 Jul 13;60(13):5800-5815. doi: 10.1021/acs.jmedchem.7b00472. Epub 2017 Jun 27.

Abstract

Acid ceramidase (AC) hydrolyzes ceramides, which are central lipid messengers for metabolism and signaling of sphingolipids. A growing body of evidence links deregulation of sphingolipids to several diseases, including cancer. Indeed, AC expression is abnormally high in melanoma cells. AC inhibition may thus be key to treating malignant melanoma. Here, we have used a systematic scaffold exploration to design a general pharmacophore for AC inhibition. This pharmacophore comprises a 6 + 5 fused ring heterocycle linked to an aliphatic substituent via a urea moiety. We have thus identified the novel benzimidazole derivatives 10, 21, 27, and 30, which are highly potent AC inhibitors. Their chemical and metabolic stabilities are comparable or superior to those of previously reported AC inhibitors. Moreover, they are potent against endogenous AC in intact melanoma cells. These novel inhibitors merit further characterization and can serve as a promising starting point for the discovery of new antimelanoma therapeutics.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acid Ceramidase / antagonists & inhibitors*
  • Acid Ceramidase / metabolism
  • Animals
  • Antineoplastic Agents / blood
  • Antineoplastic Agents / chemistry*
  • Antineoplastic Agents / pharmacology*
  • Benzimidazoles / blood
  • Benzimidazoles / chemistry*
  • Benzimidazoles / pharmacology*
  • Cell Line, Tumor
  • Cell Survival / drug effects
  • Drug Stability
  • Enzyme Inhibitors / blood
  • Enzyme Inhibitors / chemistry*
  • Enzyme Inhibitors / pharmacology*
  • HEK293 Cells
  • Humans
  • Melanoma / drug therapy
  • Melanoma / metabolism
  • Mice

Substances

  • Antineoplastic Agents
  • Benzimidazoles
  • Enzyme Inhibitors
  • benzimidazole
  • Acid Ceramidase