Combining hit identification strategies: fragment-based and in silico approaches to orally active 2-aminothieno[2,3-d]pyrimidine inhibitors of the Hsp90 molecular chaperone

J Med Chem. 2009 Aug 13;52(15):4794-809. doi: 10.1021/jm900357y.

Abstract

Inhibitors of the Hsp90 molecular chaperone are showing considerable promise as potential molecular therapeutic agents for the treatment of cancer. Here we describe novel 2-aminothieno[2,3-d]pyrimidine ATP competitive Hsp90 inhibitors, which were designed by combining structural elements of distinct low affinity hits generated from fragment-based and in silico screening exercises in concert with structural information from X-ray protein crystallography. Examples from this series have high affinity (IC50 = 50-100 nM) for Hsp90 as measured in a fluorescence polarization (FP) competitive binding assay and are active in human cancer cell lines where they inhibit cell proliferation and exhibit a characteristic profile of depletion of oncogenic proteins and concomitant elevation of Hsp72. Several examples (34a, 34d and 34i) caused tumor growth regression at well tolerated doses when administered orally in a human BT474 human breast cancer xenograft model.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Oral
  • Animals
  • Antineoplastic Agents / chemical synthesis*
  • Antineoplastic Agents / pharmacology
  • Binding, Competitive
  • Crystallography, X-Ray
  • Female
  • Fluorescence Polarization
  • HSP90 Heat-Shock Proteins / antagonists & inhibitors*
  • Humans
  • Male
  • Mice
  • Mice, Inbred BALB C
  • Pyrimidines / chemical synthesis*
  • Pyrimidines / pharmacology
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • HSP90 Heat-Shock Proteins
  • Pyrimidines