Multi-species toxicology approaches for oncology drugs: the US perspective

Eur J Cancer. 2004 Apr;40(6):907-13. doi: 10.1016/j.ejca.2003.11.024.

Abstract

The Toxicology and Pharmacology Branch (T&PB) of the National Cancer Institute (NCI) performs pharmacological and toxicological evaluations of new oncology agents according to an agent-directed paradigm in which all studies are tailored to each agent. The United States Food and Drug Administration (US FDA) requires that preclinical toxicology studies be conducted in two species, a rodent and a non-rodent for all small molecules, and T&PB has successfully used this formula. While pharmacokinetic (PK) studies are considered optional, T&PB routinely develops new methods for plasma/tissue drug analysis and employs this methodology throughout development to determine kinetics in various species and toxicokinetics in the toxicity studies. In the current era of molecular target-based development, the T&PB also develops or employs methodology to evaluate effects of the new chemical entity on appropriate biomarkers in tumour and normal tissues. In this comprehensive programme, T&PB is able to correlate safety and toxicity with both plasma drug levels and biomarker modulation in two species for a seamless entry into Phase I.

Publication types

  • Review

MeSH terms

  • Animals
  • Antineoplastic Agents / toxicity*
  • Biotechnology
  • Drug Design
  • Drug Evaluation / methods
  • Forecasting
  • Maximum Tolerated Dose
  • Models, Animal
  • Neoplasms / drug therapy*
  • United States

Substances

  • Antineoplastic Agents