Discovery of a Potent and Selective Sphingosine Kinase 1 Inhibitor through the Molecular Combination of Chemotype-Distinct Screening Hits

J Med Chem. 2017 Mar 23;60(6):2562-2572. doi: 10.1021/acs.jmedchem.7b00070. Epub 2017 Mar 6.

Abstract

Sphingosine kinase (SphK) is the major source of the lipid mediator and G protein-coupled receptor agonist sphingosine-1-phosphate (S1P). S1P promotes cell growth, survival, and migration and is a key regulator of lymphocyte trafficking. Inhibition of S1P signaling has been proposed as a strategy for treatment of inflammatory diseases and cancer. Two different formats of an enzyme-based high-throughput screen yielded two attractive chemotypes capable of inhibiting S1P formation in cells. The molecular combination of these screening hits led to compound 22a (PF-543) with 2 orders of magnitude improved potency. Compound 22a inhibited SphK1 with an IC50 of 2 nM and was more than 100-fold selective for SphK1 over the SphK2 isoform. Through the modification of tail-region substituents, the specificity of inhibition for SphK1 and SphK2 could be modulated, yielding SphK1-selective, potent SphK1/2 dual, or SphK2-preferential inhibitors.

MeSH terms

  • Amination
  • Benzimidazoles / chemistry
  • Benzimidazoles / pharmacology
  • Drug Discovery
  • Humans
  • Models, Molecular
  • Phosphotransferases (Alcohol Group Acceptor) / antagonists & inhibitors*
  • Phosphotransferases (Alcohol Group Acceptor) / metabolism
  • Protein Kinase Inhibitors / chemistry*
  • Protein Kinase Inhibitors / pharmacology*
  • Pyrrolidines / chemistry
  • Pyrrolidines / pharmacology

Substances

  • Benzimidazoles
  • Protein Kinase Inhibitors
  • Pyrrolidines
  • Phosphotransferases (Alcohol Group Acceptor)
  • sphingosine kinase
  • pyrrolidine