The CFU Assay in Preclinical Toxicity Testing: An In Vitro Tool for Predicting In Vivo Cytopenia

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Toxicity is a major cause of attrition in therapeutic drug development and is a key factor in decisionmaking around the advancement of candidate drugs through the development pipeline. The expansion of preclinical testing to incorporate assays that better predict potential toxicities earlier in the drug development process has obvious advantages for the selection of successful lead candidates. In vitro testing on primary cells can allow investigators to preview in vivo responses, thus facilitating design of better dosing strategies and optimization of animal and Phase I clinical studies.