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

Luca Pompili, Manuela Porru, Carla Caruso, Annamaria Biroccio, Carlo Leonetti

Research output: Contribution to journalReview articlepeer-review


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.

Original languageEnglish
Article number189
JournalJournal of Experimental and Clinical Cancer Research
Issue number1
Publication statusPublished - Dec 5 2016


  • Immunosuppressed mice
  • Orthotopic
  • Patient-derived-xenografts
  • Predictive value
  • Subcutaneous
  • Tumor heterogeneity

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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