The significance of mitochondrial toxicity testing in drug development

Drug Discov Today. 2007 Sep;12(17-18):777-85. doi: 10.1016/j.drudis.2007.07.013. Epub 2007 Aug 22.


Mitochondrial dysfunction is increasingly implicated in the etiology of drug-induced toxicities. Members of diverse drug classes undermine mitochondrial function, and among the most potent are drugs that have been withdrawn from the market, or have received Black Box warnings from the FDA. To avoid mitochondrial liabilities, routine screens need to be positioned within the drug-development process. Assays for mitochondrial function, cell models that better report mitochondrial impairment, and new animal models that more faithfully reflect clinical manifestations of mitochondrial dysfunction are discussed in the context of how such data can reduce late stage attrition of drug candidates and can yield safer drugs in the future.

Publication types

  • Review

MeSH terms

  • Animals
  • Drug Design
  • Drug Evaluation, Preclinical / methods*
  • Drug Industry
  • Drug Labeling / legislation & jurisprudence
  • Drug-Related Side Effects and Adverse Reactions*
  • Humans
  • Mitochondria / drug effects*
  • Mitochondria / physiology
  • United States
  • United States Food and Drug Administration