Discovery of Novel 1,4-Diacylpiperazines as Selective and Cell-Active eIF4A3 Inhibitors

J Med Chem. 2017 Apr 27;60(8):3335-3351. doi: 10.1021/acs.jmedchem.6b01904. Epub 2017 Apr 11.

Abstract

Eukaryotic initiation factor 4A3 (eIF4A3), a member of the DEAD-box RNA helicase family, is one of the core components of the exon junction complex (EJC). The EJC is known to be involved in a variety of RNA metabolic processes typified by nonsense-mediated RNA decay (NMD). In order to identify molecular probes to investigate the functions and therapeutic relevance of eIF4A3, a search for selective eIF4A3 inhibitors was conducted. Through the chemical optimization of 1,4-diacylpiperazine derivatives identified via high-throughput screening (HTS), we discovered the first reported selective eIF4A3 inhibitor 53a exhibiting cellular NMD inhibitory activity. A surface plasmon resonance (SPR) biosensing assay ascertained the direct binding of 53a and its analog 52a to eIF4A3 and revealed that the binding occurs at a non-ATP binding site. Compounds 52a and 53a represent novel molecular probes for further study of eIF4A3, the EJC, and NMD.

MeSH terms

  • Adenosine Triphosphatases / antagonists & inhibitors
  • DEAD-box RNA Helicases / antagonists & inhibitors*
  • Drug Discovery
  • Eukaryotic Initiation Factor-4A / antagonists & inhibitors*
  • HEK293 Cells
  • Humans
  • Mass Spectrometry
  • Piperazines / chemistry
  • Piperazines / pharmacology*
  • Proton Magnetic Resonance Spectroscopy
  • Structure-Activity Relationship
  • Surface Plasmon Resonance

Substances

  • Piperazines
  • Eukaryotic Initiation Factor-4A
  • Adenosine Triphosphatases
  • EIF4A3 protein, human
  • DEAD-box RNA Helicases