Identification of novel small molecules that inhibit STAT3-dependent transcription and function

PLoS One. 2017 Jun 21;12(6):e0178844. doi: 10.1371/journal.pone.0178844. eCollection 2017.

Abstract

Activation of Signal Transducer and Activator of Transcription 3 (STAT3) has been linked to several processes that are critical for oncogenic transformation, cancer progression, cancer cell proliferation, survival, drug resistance and metastasis. Inhibition of STAT3 signaling has shown a striking ability to inhibit cancer cell growth and therefore, STAT3 has become a promising target for anti-cancer drug development. The aim of this study was to identify novel inhibitors of STAT-dependent gene transcription. A cellular reporter-based system for monitoring STAT3 transcriptional activity was developed which was suitable for high-throughput screening (Z' = 0,8). This system was used to screen a library of 28,000 compounds (the ENAMINE Drug-Like Diversity Set). Following counter-screenings and toxicity studies, we identified four hit compounds that were subjected to detailed biological characterization. Of the four hits, KI16 stood out as the most promising compound, inhibiting STAT3 phosphorylation and transcriptional activity in response to IL6 stimulation. In silico docking studies showed that KI16 had favorable interactions with the STAT3 SH2 domain, however, no inhibitory activity could be observed in the STAT3 fluorescence polarization assay. KI16 inhibited cell viability preferentially in STAT3-dependent cell lines. Taken together, using a targeted, cell-based approach, novel inhibitors of STAT-driven transcriptional activity were discovered which are interesting leads to pursue further for the development of anti-cancer therapeutic agents.

MeSH terms

  • Antineoplastic Agents / pharmacology*
  • Apoptosis / drug effects
  • Cell Proliferation / drug effects
  • High-Throughput Screening Assays / methods*
  • Humans
  • Neoplasms / drug therapy
  • Neoplasms / pathology
  • STAT3 Transcription Factor / antagonists & inhibitors*
  • Signal Transduction
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / pharmacology*
  • Tumor Cells, Cultured

Substances

  • Antineoplastic Agents
  • STAT3 Transcription Factor
  • Small Molecule Libraries

Grants and funding

Support was provided by The Swedish Research Council; 521-2012-2037 [www.vr.se] (DG) The Swedish Cancer Foundation; 15 0768 [www.cancerfonden.se] (DG); Radiumhemmet Research Foundation; 144063 [www.rahfo.se] (DG); Ruth and Rickard Julins Fond; 2016juli46442, managed by Karolinska Institutet Funds [www.fonder.ki.se] (IK); Robert Lundbergs Memmorial Fund; 2015lund45360, managed by Karolinska Institutet Funds [fonder.ki.se] (IK); Jakob Linder's Memorial Foundation, [www.carlsonlinder.se] (IK); Swedish Society for Medical Research (SSMF), [www.ssmf.se] (BDGP); The David and Astrid Hagelén Foundation; managed by Karolinska Institutet Funds [fonder.ki.se] (BDGP). The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.