The first Japanese biobank of patient‐derived pediatric acute lymphoblastic leukemia xenograft models

Abstract

Preclinical discovery and testing of therapies for pediatric relapsed and refractory acute lymphoblastic leukemia (ALL), which has poor outcomes, is limited, in Japan, due to a lack of practical resources. We have established 57 patient-derived xenografts (PDXs) in NOD.Cg-Prkdcscidll2rgtm1Sug/ShiJic (NOG) mice and created a biobank by preserving the PDX cells, including three extramedullary relapsed ALL PDXs. We demonstrated that our PDX mice and PDX cells mimicked the biological features of relapsed ALL and that the PDX models reproduced treatment-mediated clonal selection. Our PDX biobank is a useful resource for capturing features of drug sensitivity in pediatric patients with ALL, providing an essential tool for the development of targeted therapies.