Stereospecific targeting of MTH1 by (S)-crizotinib as an anticancer strategy

Nature. 2014 Apr 10;508(7495):222-7. doi: 10.1038/nature13194. Epub 2014 Apr 2.

Abstract

Activated RAS GTPase signalling is a critical driver of oncogenic transformation and malignant disease. Cellular models of RAS-dependent cancers have been used to identify experimental small molecules, such as SCH51344, but their molecular mechanism of action remains generally unknown. Here, using a chemical proteomic approach, we identify the target of SCH51344 as the human mutT homologue MTH1 (also known as NUDT1), a nucleotide pool sanitizing enzyme. Loss-of-function of MTH1 impaired growth of KRAS tumour cells, whereas MTH1 overexpression mitigated sensitivity towards SCH51344. Searching for more drug-like inhibitors, we identified the kinase inhibitor crizotinib as a nanomolar suppressor of MTH1 activity. Surprisingly, the clinically used (R)-enantiomer of the drug was inactive, whereas the (S)-enantiomer selectively inhibited MTH1 catalytic activity. Enzymatic assays, chemical proteomic profiling, kinome-wide activity surveys and MTH1 co-crystal structures of both enantiomers provide a rationale for this remarkable stereospecificity. Disruption of nucleotide pool homeostasis via MTH1 inhibition by (S)-crizotinib induced an increase in DNA single-strand breaks, activated DNA repair in human colon carcinoma cells, and effectively suppressed tumour growth in animal models. Our results propose (S)-crizotinib as an attractive chemical entity for further pre-clinical evaluation, and small-molecule inhibitors of MTH1 in general as a promising novel class of anticancer agents.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aminoquinolines / pharmacology
  • Animals
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacology*
  • Colonic Neoplasms / drug therapy
  • Colonic Neoplasms / genetics
  • Colonic Neoplasms / pathology
  • Crizotinib
  • Crystallization
  • DNA Breaks, Single-Stranded / drug effects
  • DNA Repair
  • DNA Repair Enzymes / antagonists & inhibitors*
  • DNA Repair Enzymes / biosynthesis
  • DNA Repair Enzymes / chemistry
  • DNA Repair Enzymes / metabolism*
  • Disease Models, Animal
  • Female
  • Homeostasis / drug effects
  • Humans
  • Mice
  • Mice, SCID
  • Models, Molecular
  • Nucleotides / metabolism
  • Phosphoric Monoester Hydrolases / antagonists & inhibitors*
  • Phosphoric Monoester Hydrolases / biosynthesis
  • Phosphoric Monoester Hydrolases / chemistry
  • Phosphoric Monoester Hydrolases / metabolism*
  • Protein Conformation
  • Protein Kinase Inhibitors / chemistry
  • Protein Kinase Inhibitors / pharmacology*
  • Proteomics
  • Proto-Oncogene Proteins / genetics
  • Proto-Oncogene Proteins p21(ras)
  • Pyrazoles / chemistry
  • Pyrazoles / pharmacology*
  • Pyridines / chemistry
  • Pyridines / pharmacology*
  • Substrate Specificity
  • Xenograft Model Antitumor Assays
  • ras Proteins / genetics

Substances

  • Aminoquinolines
  • Antineoplastic Agents
  • KRAS protein, human
  • Nucleotides
  • Protein Kinase Inhibitors
  • Proto-Oncogene Proteins
  • Pyrazoles
  • Pyridines
  • SCH 51344
  • Crizotinib
  • Phosphoric Monoester Hydrolases
  • 8-oxodGTPase
  • Proto-Oncogene Proteins p21(ras)
  • ras Proteins
  • DNA Repair Enzymes

Associated data

  • PDB/4C9W
  • PDB/4C9X