Validating cancer drug targets

Nature. 2006 May 25;441(7092):451-6. doi: 10.1038/nature04873.


A cancer drug target is only truly validated by demonstrating that a given therapeutic agent is clinically effective and acts through the target against which it was designed. Nevertheless, it is desirable to declare an early-stage drug target as 'validated' before investing in a full-scale drug discovery programme dedicated to it. Although the outcome of validation studies can guide cancer research programmes, strictly defined universal validation criteria have not been established.

Publication types

  • Review

MeSH terms

  • Animals
  • Cells / drug effects
  • Cells / metabolism
  • Disease Models, Animal
  • Drug Evaluation, Preclinical / methods*
  • Drug Evaluation, Preclinical / standards
  • Humans
  • Neoplasms / drug therapy*
  • Neoplasms / genetics
  • Neoplasms / metabolism*
  • Neoplasms / pathology
  • Reproducibility of Results
  • Substrate Specificity