As one of the last validating steps before entering clinical phase, our preclinical model generates results integrative with current available in vivo models. Response rates of classic cytotoxic drugs and targeted therapies in PDXs demonstrate a high similarity with those observed in patients in the clinic treated with the same agents. Based on a selected tumor type and/or a predefined molecular subtype, a number of PDXs can be enrolled in a ‘mouse clinical trial’ and treated with new or existing agents. By comparing the molecular profile of responders and non-responders, PDXs facilitate the identification of potential drug efficacy biomarkers and allows predicting drug susceptibility and resistance.
Study endpoints like complete remission, stable disease, etc. needs to be carefully determined based on the mechanism of action of the investigated agent.