On the use of pharmacogenetics in cancer treatment and clinical trials

Eur J Cancer. 2014 Oct;50(15):2532-43. doi: 10.1016/j.ejca.2014.07.013. Epub 2014 Aug 4.


There are an increasing number of studies devoted to the identification of associations between anticancer drug efficacy and toxicity and common polymorphisms present in the patients' genome. However, many articles presenting the results of such studies do not bring the simple and necessary background information allowing the evaluation of the relevance of the study, its significance and its potential importance for patients' treatment. This position paper first addresses clinical oncologists with the aim of giving them the basic knowledge on pharmacogenetics and on the potential use of gene polymorphisms as predictive biomarkers in routine and clinical research. A secondary objective is to give molecular biologists some recommendations on how to conceive protocols and how to publish their results when they develop pharmacogenetic studies appended to clinical trials or with autonomous goals.

Keywords: Anticancer drug response; Anticancer drug toxicity; Biomarkers; Cancer clinical trials; Cancer treatments; Gene polymorphisms; Pharmacogenetics.

Publication types

  • Review

MeSH terms

  • Clinical Trials as Topic / methods*
  • Clinical Trials as Topic / statistics & numerical data
  • Gene Frequency
  • Genetic Predisposition to Disease / genetics
  • Genotype
  • Humans
  • Neoplasms / drug therapy*
  • Neoplasms / genetics*
  • Pharmacogenetics / methods
  • Pharmacogenetics / statistics & numerical data
  • Polymorphism, Genetic*
  • Treatment Outcome