New cast for a new era: preclinical cancer drug development revisited

J Clin Invest. 2013 Sep;123(9):3639-45. doi: 10.1172/JCI68340. Epub 2013 Sep 3.


Molecularly targeted agents promise to revolutionize therapeutics by reducing morbidity and mortality in patients with cancer. However, despite an urgent need for more effective anticancer compounds, current preclinical drug evaluations largely fail to satisfy the demand. New preclinical strategies, including the improvement of sophisticated mouse models and co-clinical study designs, are being used to augment the predictive value of animal-based translational cancer research. Here, we review the development of successful preclinical antineoplastic agents, their associated limitations, and alternative methods to predict clinical outcomes.

Publication types

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

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use
  • Clinical Trials as Topic
  • Disease Models, Animal
  • Drug Discovery
  • Drug Screening Assays, Antitumor*
  • Humans
  • Mice
  • Mice, Transgenic
  • Neoplasms / drug therapy*


  • Antineoplastic Agents