Inhibiting aberrant signal transducer and activator of transcription protein activation with tetrapodal, small molecule Src homology 2 domain binders: promising agents against multiple myeloma

J Med Chem. 2013 Sep 26;56(18):7190-200. doi: 10.1021/jm3017255. Epub 2013 Sep 13.

Abstract

The signal transducer and activator of transcription (STAT) proteins represent a family of cytoplasmic transcription factors that regulate a pleiotropic range of biological processes. In particular, Stat3 protein has attracted attention as it regulates the expression of genes involved in a variety of malignant processes, including proliferation, survival, migration, and drug resistance. Multiple myeloma (MM) is an incurable hematologic malignancy that often exhibits abnormally high levels of Stat3 activity. Although current treatment strategies can improve the clinical management of MM, it remains uniformly incurable with a dismal median survival time post-treatment of 3-4 years. Thus, novel targeted therapeutics are critically needed to improve MM patient outcomes. We herein report the development of a series of small molecule Stat3 inhibitors with potent anti-MM activity in vitro. These compounds showed high-affinity binding to Stat3's SH2 domain, inhibited intracellular Stat3 phosphorylation, and induced apoptosis in MM cell lines at low micromolar concentrations.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents / chemistry*
  • Antineoplastic Agents / metabolism
  • Antineoplastic Agents / pharmacology*
  • Antineoplastic Agents / therapeutic use*
  • Cell Line, Tumor
  • Humans
  • Inhibitory Concentration 50
  • Molecular Docking Simulation
  • Multiple Myeloma / drug therapy*
  • Multiple Myeloma / pathology
  • STAT3 Transcription Factor / antagonists & inhibitors*
  • STAT3 Transcription Factor / chemistry*
  • STAT3 Transcription Factor / metabolism
  • src Homology Domains / drug effects*

Substances

  • Antineoplastic Agents
  • STAT3 Transcription Factor
  • STAT3 protein, human