Discovery of 2-Amino-3-cyanothiophene Derivatives as Potent STAT3 Inhibitors for the Treatment of Osteosarcoma Growth and Metastasis

J Med Chem. 2022 May 12;65(9):6710-6728. doi: 10.1021/acs.jmedchem.2c00004. Epub 2022 Apr 27.

Abstract

Osteosarcoma is one of the most common malignant bone tumors. However, the treatment and clinical outcomes of osteosarcoma have hardly changed over the past three decades due to the comprehensive heterogeneity and higher rate of mutation of osteosarcoma. Recent studies have shown that STAT3 has the potential to suppress the proliferation and metastasis of osteosarcoma. In this study, a novel class of 2-amino-3-cyanothiophene derivatives were designed and synthesized to inhibit osteosarcoma by targeting STAT3. Representative compound 6f showed potent antiproliferative effects against osteosarcoma cells, directly bound to the STAT3 SH2 domain with a KD of 0.46 μM, and inhibited the phosphorylation of STAT3 Y705 in a dose-dependent manner. Furthermore, compound 6f promoted osteosarcoma cell apoptosis in vitro and significantly suppressed the growth and metastasis of osteosarcoma in vivo. These findings demonstrate that targeting STAT3 may be a feasible therapeutic strategy for the treatment of metastatic osteosarcoma.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Apoptosis
  • Bone Neoplasms* / pathology
  • Cell Line, Tumor
  • Cell Proliferation
  • Humans
  • Osteosarcoma* / pathology
  • STAT3 Transcription Factor / metabolism

Substances

  • STAT3 Transcription Factor
  • STAT3 protein, human