A cell-based high throughput screening assay for the discovery of cGAS-STING pathway agonists

Antiviral Res. 2017 Nov:147:37-46. doi: 10.1016/j.antiviral.2017.10.001. Epub 2017 Oct 2.

Abstract

Stimulator of interferon genes (STING) is an endoplasmic reticulum transmembrane protein that serves as a molecular hub for activation of interferon and inflammatory cytokine response by multiple cellular DNA sensors. Not surprisingly, STING has been demonstrated to play an important role in host defense against microorganisms and pharmacologic activation of STING is considered as an attractive strategy to treat viral diseases and boost antitumor immunity. In light of this we established a HepAD38-derived reporter cell line that expresses firefly luciferase in response to the activation of cyclic GMP-AMP synthase (cGAS)-STING pathway for high throughput screening (HTS) of small molecular human STING agonists. This cell-based reporter assay required only 4 h treatment with a reference STING agonist to induce a robust luciferase signal and was demonstrated to have an excellent performance in HTS format. By screening 16,000 compounds, a dispiro diketopiperzine (DSDP) compound was identified to induce cytokine response in a manner dependent on the expression of functional human STING, but not mouse STING. Moreover, we showed that DSDP induced an interferon-dominant cytokine response in human skin fibroblasts and peripheral blood mononuclear cells, which in turn potently suppressed the replication of yellow fever virus, dengue virus and Zika virus. We have thus established a robust cell-based assay system suitable for rapid discovery and mechanistic analyses of cGAS-STING pathway agonists. Identification of DSDP as a human STING agonist enriches the pipelines of STING-targeting drug development for treatment of viral infections and cancers.

Keywords: Antiviral; High throughput assay; Innate immune; STING.

MeSH terms

  • Animals
  • Antiviral Agents / chemistry
  • Antiviral Agents / pharmacology*
  • Cell Survival / drug effects
  • Cells, Cultured
  • Drug Discovery / methods*
  • Flavivirus / drug effects
  • Gene Expression / drug effects
  • High-Throughput Screening Assays*
  • Humans
  • Immunity, Innate / drug effects*
  • Interferon Inducers / chemistry
  • Interferon Inducers / pharmacology*
  • Lethal Dose 50
  • Membrane Proteins / agonists*
  • Membrane Proteins / genetics
  • Mice
  • Mutation
  • Nucleotidyltransferases / antagonists & inhibitors*
  • Nucleotidyltransferases / genetics
  • Piperazines / chemistry
  • Piperazines / pharmacology*
  • Signal Transduction / drug effects
  • Species Specificity
  • Spiro Compounds / chemistry
  • Spiro Compounds / pharmacology*
  • Transcription Factors / genetics
  • Virus Replication / drug effects

Substances

  • Antiviral Agents
  • Interferon Inducers
  • Membrane Proteins
  • Piperazines
  • STING1 protein, human
  • Spiro Compounds
  • Transcription Factors
  • Nucleotidyltransferases
  • cGAS protein, human