Patient-derived xenograft platform of OSCC: a renewable human bio-bank for preclinical cancer research and a new co-clinical model for treatment optimization

Abstract

Advances in next-generation sequencing and bioinformatics have begun to reveal the complex genetic landscape in human cancer genomes, including oral squamous cell carcinoma (OSCC). Sophisticated preclinical models that fully represent intra- and inter-tumoral heterogeneity are required to understand the molecular diversity of cancer and achieve the goal of personalized therapies. Patient-derived xenograft (PDX) models generated from human tumor samples that can retain the histological and genetic features of their donor tumors have been shown to be the preferred preclinical tool in translational cancer research compared with other conventional preclinical models. Specifically, genetically well-defined PDX models can be applied to accelerate targeted antitumor drug development and biomarker discovery. Recently, we have successfully established and characterized an OSCC PDX panel as part of our tumor bio-bank for translational cancer research. In this paper, we discuss the establishment, characterization, and preclinical applications of the PDX models. In particular, we focus on the classification and applications of the PDX models based on validated annotations, including clinicopathological features, genomic profiles, and pharmacological testing information. We also explore the translational value of this well-annotated PDX panel in the development of co-clinical trials for patient stratification and treatment optimization in the near future. Although various limitations still exist, this preclinical approach should be further tested and improved.

Keywords

patient-derived xenograft models / personalized medicine / co-clinical trial / patient stratification / oral squamous cell carcinoma

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Acknowledgments

This work was supported by grants from the National Natural Science Foundation of China (Nos. 81202131 and 81572656), the China Postdoctoral Science Foundation (No. 2013M531191), and the Shanghai Postdoctoral Sustentation Fund, China (No. 13R214 15100).

Compliance with ethics guidelines

Shuyang Sun and Zhiyuan Zhang declare that they have no conflict of interest. This manuscript is a commentary article and does not involve a research protocol requiring approval from a relevant institutional review board or ethics committee.

RIGHTS & PERMISSIONS

2016 Higher Education Press and Springer-Verlag Berlin Heidelberg