Cancer remains one of the most pressing global health challenges, with millions of new diagnoses each year. While precision medicine and targeted therapies have transformed treatment strategies, many patients still face drug resistance and disease recurrence. A new review published in Genes & Diseases highlights the critical role of patient-derived xenograft (PDX) models, offering a powerful preclinical platform that more accurately mimics human tumors. These models could revolutionize drug development, improve treatment efficacy, and guide personalized therapies.

PDX models are created by implanting patient-derived tumor tissue into immunodeficient mice, preserving essential features such as tumor genetics, microenvironment, and drug response patterns. Unlike traditional cancer models, which rely on cell lines, PDX models provide a more faithful representation of the complexity of human cancer, making them a superior tool for testing novel therapies before they reach clinical trials.

A key advantage of PDX models is their ability to support co-clinical trials, where patients and their corresponding PDX models are treated in parallel. This approach allows researchers to evaluate treatment responses in real time, helping to identify the most effective therapies for individual patients. These models have already demonstrated significant promise in areas such as breast, lung, colorectal, and ovarian cancer research.

Despite their potential, PDX models face several challenges that need to be addressed to maximize their impact. High costs, long development times, and the need for highly specialized facilities are among the barriers to widespread adoption. Additionally, tumor evolution within PDX models may not always mirror the progression of cancer in human patients, posing limitations for long-term studies.

To overcome these obstacles, scientists are actively working on next-generation PDX models, incorporating technologies such as CRISPR gene editing, organoid co-cultures, and humanized mouse models. These innovations aim to enhance the reliability and scalability of PDX systems, accelerating the development of breakthrough cancer therapies.

With ongoing advancements in biobanking and artificial intelligence-driven drug discovery, PDX models are expected to become an indispensable tool in precision oncology.

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Genes & Diseases publishes rigorously peer-reviewed and high quality original articles and authoritative reviews that focus on the molecular bases of human diseases. Emphasis is placed on hypothesis-driven, mechanistic studies relevant to pathogenesis and/or experimental therapeutics of human diseases. The journal has worldwide authorship, and a broad scope in basic and translational biomedical research of molecular biology, molecular genetics, and cell biology, including but not limited to cell proliferation and apoptosis, signal transduction, stem cell biology, developmental biology, gene regulation and epigenetics, cancer biology, immunity and infection, neuroscience, disease-specific animal models, gene and cell-based therapies, and regenerative medicine.
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Reference
Minqi Liu, Xiaoping Yang, Patient-derived xenograft models: Current status, challenges, and innovations in cancer research, Genes & Diseases, Volume 12, Issue 5, 2025, 101520, https://doi.org/10.1016/j.gendis.2025.101520

Funding Information:
National Natural Science Foundation of China 82172653
Intra Institutional Open Fund of School of Medicine, Hunan Normal University KF2022001
Key Project of Developmental Biology and Breeding from Hunan Province, China 2022XKQ0205
Research Team for Reproduction Health and Translational Medicine of Hunan Normal University 2023JC101