Fragment-based drug discovery applied to Hsp90. Discovery of two lead series with high ligand efficiency

J Med Chem. 2010 Aug 26;53(16):5942-55. doi: 10.1021/jm100059d.

Abstract

Inhibitors of the chaperone Hsp90 are potentially useful as chemotherapeutic agents in cancer. This paper describes an application of fragment screening to Hsp90 using a combination of NMR and high throughput X-ray crystallography. The screening identified an aminopyrimidine with affinity in the high micromolar range and subsequent structure-based design allowed its optimization into a low nanomolar series with good ligand efficiency. A phenolic chemotype was also identified in fragment screening and was found to bind with affinity close to 1 mM. This fragment was optimized using structure based design into a resorcinol lead which has subnanomolar affinity for Hsp90, excellent cell potency, and good ligand efficiency. This fragment to lead campaign improved affinity for Hsp90 by over 1,000,000-fold with the addition of only six heavy atoms. The companion paper (DOI: 10.1021/jm100060b) describes how the resorcinol lead was optimized into a compound that is now in clinical trials for the treatment of cancer.

MeSH terms

  • Aminopyridines / chemical synthesis
  • Aminopyridines / chemistry*
  • Antineoplastic Agents / chemistry*
  • Crystallography, X-Ray
  • Databases, Factual
  • Drug Design
  • HSP90 Heat-Shock Proteins / antagonists & inhibitors*
  • HSP90 Heat-Shock Proteins / chemistry*
  • Ligands
  • Magnetic Resonance Spectroscopy
  • Models, Molecular*
  • Phenols / chemical synthesis
  • Phenols / chemistry*
  • Protein Binding
  • Protein Structure, Tertiary
  • Resorcinols / chemical synthesis
  • Resorcinols / chemistry
  • Structure-Activity Relationship

Substances

  • Aminopyridines
  • Antineoplastic Agents
  • HSP90 Heat-Shock Proteins
  • Ligands
  • Phenols
  • Resorcinols

Associated data

  • PDB/2XAB
  • PDB/2XDK
  • PDB/2XDL
  • PDB/2XDS
  • PDB/2XDU
  • PDB/2XDX
  • PDB/2XHR
  • PDB/2XHT
  • PDB/2XHX