Inhibition of Cancer-Associated Mutant Isocitrate Dehydrogenases by 2-Thiohydantoin Compounds

J Med Chem. 2015 Sep 10;58(17):6899-6908. doi: 10.1021/acs.jmedchem.5b00684. Epub 2015 Aug 26.

Abstract

Somatic mutations of isocitrate dehydrogenase 1 (IDH1) at R132 are frequently found in certain cancers such as glioma. With losing the activity of wild-type IDH1, the R132H and R132C mutant proteins can reduce α-ketoglutaric acid (α-KG) to d-2-hydroxyglutaric acid (D2HG). The resulting high concentration of D2HG inhibits many α-KG-dependent dioxygenases, including histone demethylases, to cause broad histone hypermethylation. These aberrant epigenetic changes are responsible for the initiation of these cancers. We report the synthesis, structure-activity relationships, enzyme kinetics, and binding thermodynamics of a novel series of 2-thiohydantoin and related compounds, among which several compounds are potent inhibitors of mutant IDH1 with Ki as low as 420 nM. X-ray crystal structures of IDH1(R132H) in complex with two inhibitors are reported, showing their inhibitor-protein interactions. These compounds can decrease the cellular concentration of D2HG, reduce the levels of histone methylation, and suppress the proliferation of stem-like cancer cells in BT142 glioma with IDH1 R132H mutation.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / chemistry*
  • Antineoplastic Agents / pharmacology
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Crystallography, X-Ray
  • Drug Screening Assays, Antitumor
  • Glutarates / metabolism
  • Histones / metabolism
  • Humans
  • Isocitrate Dehydrogenase / antagonists & inhibitors*
  • Isocitrate Dehydrogenase / chemistry
  • Isocitrate Dehydrogenase / genetics
  • Kinetics
  • Methylation
  • Models, Molecular
  • Mutation
  • Neoplastic Stem Cells / drug effects
  • Neoplastic Stem Cells / pathology
  • Protein Binding
  • Protein Conformation
  • Structure-Activity Relationship
  • Thermodynamics
  • Thiohydantoins / chemical synthesis
  • Thiohydantoins / chemistry*
  • Thiohydantoins / pharmacology

Substances

  • Antineoplastic Agents
  • Glutarates
  • Histones
  • Thiohydantoins
  • alpha-hydroxyglutarate
  • Isocitrate Dehydrogenase