2019 Volume 56 Issue 5 Pages 407-413
Compared with adult cancers, pediatric tumors are rare, and limited access to tumor cells and tissues is a barrier to developing translational research. To overcome these limitations, there are international efforts to develop patient-derived xenograft (PDX) banks for pediatric leukemias and solid tumors. A PDX model can be developed by implanting tumor cells or tissues from a patient’s tumor into an immune-deficient mouse via the tail vein, subcutaneously or orthotopically, thereby preserving both cell–cell interactions and the tumor microenvironment within the limitation of cross talk between mouse and human cells. PDXs not only expand the tumor cells but also recapitulate the pathology of the human disease in vivo. In addition, the PDX model has shown advantages as a preclinical model in drug screening and preclinical trials. In Japan, Fukushima Medical University and Kyoto University established the first nationwide publicly available repository of PDXs transplanted with pediatric leukemias in 2013. This repository will enable investigators from both academic and industrial laboratories to conduct therapeutic tests using patient-derived samples. In this review, we will summarize the developmental history of immune-deficient mice, efforts of overcoming the limitations of PDXs, and current applications of this model in preclinical research.
