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

A lack of practical resources in Japan has limited preclinical discovery and testing of therapies for pediatric relapsed and refractory acute lymphoblastic leukemia (ALL), which has poor outcomes. Here, we established 57 patient-derived xenografts (PDXs) in NOD.Cg-Prkdc(scid)ll2rg(tm1Sug)/ShiJic (NOG) mice and created a biobank by preserving 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 PDX models reproduced treatment-mediated clonal selection. Our PDX biobank is a useful scientific resource for capturing drug sensitivity features of pediatric patients with ALL, providing an essential tool for the development of targeted therapies.

Automated summary

A lack of practical resources in Japan has hindered preclinical discovery and testing of therapies for pediatric relapsed and refractory acute lymphoblastic leukemia (ALL). In this study, a PDX biobank was established, consisting of 57 patient-derived xenografts in mice, to mimic the biological features and treatment-mediated clonal selection of relapsed ALL. This biobank provides a valuable scientific resource for capturing drug sensitivity features of pediatric patients with ALL and aids in the development of targeted therapies.