Patient-derived xenografts: a relevant preclinical model for drug development

Luca Pompili†,
Manuela Porru†,
Carla Caruso,
Annamaria Biroccio and
Carlo LeonettiEmail author View ORCID ID profile

†Contributed equally

Journal of Experimental & Clinical Cancer Research201635:189

DOI: 10.1186/s13046-016-0462-4

Received: 29 October 2016

Accepted: 22 November 2016

Published: 5 December 2016

Abstract

Identifying appropriate preclinical cancer models remains a major challenge in increasing the efficiency of drug development. A potential strategy to improve patient outcomes could be selecting the ‘right’ treatment in preclinical studies performed in patient-derived xenografts (PDXs) obtained by direct implants of surgically resected tumours in mice. These models maintain morphological similarities and recapitulate molecular profiling of the original tumours, thus representing a useful tool in evaluating anticancer drug response. In this review, we will present the state-of-art use of PDXs as a reliable strategy to predict clinical findings. The main advantages and limitations will also be discussed.