Pharmacological cancer research using cancer cell line-derived xenograft (CDX) mouse models

A cell line-derived xenograft（CDX）model, prepared by transplanting human cancer cell line into mice, has been used in pharmacological cancer research for more than half a century. Even now, CDX models are adopted to evaluate pharmacological characteristics of various therapeutic modalities, including low-molecular-weight compounds, peptide, nucleic acid, antibody therapeutics, and cell-based therapy. However, in recent years, there are many reports indicating that tumor microenvironments in CDX tumors do not reflect that in clinical tumors. Therefore, it has been considered that CDX models are not an optimal cancer model for drug screening in preclinical study. Nevertheless, we believe that CDX model is still an important and useful model for pharmacological research, especially for an education of DDS efficacy due to its case of model generation, high reproducibility and high throughput. In this review, we’d like to introduce the immunodeficient mice and transplantation for preparation of CDX models, and pharmacological cancer research using them. Moreover, we describe our previous works as an example of pharmacological cancer research using CDX models.