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Regenerative Medicine

The future of patient-derived tumor xenografts in cancer treatment

    Daniela Sia

    Barcelona-Clínic Liver Cancer Group, HCC Translational Research Laboratory, Liver Unit, Hepato-biliary Surgery, Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Hospital Clínic, CIBERehd, Universitat de Barcelona, C/Rossello 153, Barcelona, Catalonia, Spain

    Gastrointestinal Surgery & Liver Transplantation Unit, Department of Surgery, National Cancer Institute, via Venezian, 1, Milan, Italy

    Liver Cancer Program, Division of Liver Diseases, Department of Medicine, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, 1425 Madison Avenue NY 10029, USA

    ,
    Agrin Moeini

    Barcelona-Clínic Liver Cancer Group, HCC Translational Research Laboratory, Liver Unit, Hepato-biliary Surgery, Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Hospital Clínic, CIBERehd, Universitat de Barcelona, C/Rossello 153, Barcelona, Catalonia, Spain

    Liver Cancer Program, Division of Liver Diseases, Department of Medicine, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, 1425 Madison Avenue NY 10029, USA

    ,
    Ismail Labgaa

    Liver Cancer Program, Division of Liver Diseases, Department of Medicine, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, 1425 Madison Avenue NY 10029, USA

    &
    Augusto Villanueva

    *Author for correspondence:

    E-mail Address: augusto.villanueva@mssm.edu

    Liver Cancer Program, Division of Liver Diseases, Department of Medicine, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, 1425 Madison Avenue NY 10029, USA

    Division of Hematology & Medical Oncology, Department of Medicine, Icahn School of Medicine at Mount Sinai, 1425 Madison Avenue, NY, USA

    Published Online:https://doi.org/10.2217/pgs.15.102

    Over the last decades, major technological advancements have led to a better understanding of the molecular drivers of human malignancies. Nonetheless, this progress only marginally impacted the cancer therapeutic approach, probably due to the limited ability of experimental models to predict efficacy in clinical trials. In an effort to offset this limitation, there has been an increasing interest in the development of patient-derived xenograft (PDX) models where human tumors are xenotransplanted into immunocompromised mice. Considering their high resemblance to human tumors and their stability, PDX models are becoming the preferred translational tools in preclinical studies. Nonetheless, several limitations hamper a wider use of PDX models and tarnish the concept that they might represent the missing piece in the personalized medicine puzzle.

    Papers of special note have been highlighted as: • of interest

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