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Updated: Mar 10, 2026

Development and Maintenance of a Preclinical Patient Derived Tumor Xenograft Model for the Investigation of Novel Anti-Cancer Therapies
Published on: September 30, 2016
Patient-derived xenografts: a relevant preclinical model for drug development
Luca Pompili1,2, Manuela Porru1, Carla Caruso2
1UOSD SAFU, Department of Research, Diagnosis and Innovative Technologies, Traslational Research Area, Regina Elena National Cancer Institute, Via Elio Chianesi 53, Rome, 00144, Italy.
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.
Insights
Patient-derived xenografts (PDXs) offer a promising strategy for improving cancer drug development. These models accurately reflect patient tumors, aiding in the selection of effective anticancer treatments.
Area of Science:
- Oncology
- Translational Research
- Drug Development
Background:
- Selecting effective preclinical cancer models is crucial for efficient drug development.
- Patient-derived xenografts (PDXs) are generated from direct surgical tumor implants in mice.
- PDXs preserve the original tumor's morphology and molecular characteristics.
Purpose of the Study:
- To review the current applications of PDXs in predicting clinical outcomes.
- To highlight the advantages of using PDXs in anticancer drug evaluation.
- To discuss the limitations associated with PDX models.
Main Methods:
- Review of existing literature on patient-derived xenografts.
- Analysis of PDX model utility in preclinical cancer research.
- Discussion of PDX model advantages and limitations.
Main Results:
- PDXs serve as valuable tools for evaluating anticancer drug response.
- These models recapitulate key features of the original human tumors.
- PDXs show potential in predicting clinical findings.
Conclusions:
- PDX models represent a reliable strategy for predicting clinical drug responses.
- Utilizing PDXs can enhance the efficiency of cancer drug development.
- Further understanding of PDX model advantages and limitations is essential.

