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Updated: Sep 18, 2025
Drug Screening of Primary Patient Derived Tumor Xenografts in Zebrafish
Published on: April 10, 2020
Narrative Review of Patient Cancer Tissue-Derived Zebrafish Xenograft Models for Evaluating Drug Sensitivity as an
Yusuke Sugino1,2, Xin Bao1,2, Takumi Kageyama1
1Department of Nephro-Urologic Surgery and Andrology, Mie University Graduate School of Medicine, Tsu, Japan.
Abstract:
In the pursuit of optimal medical care, treatment selection based on the molecular analysis of genomes, transcriptomes, and proteomes has been explored; however, this approach relies on data from large patient groups, resulting in limited accuracy in predicting treatment efficacy. Diseases involve complex pathological networks, requiring treatments that target multiple key molecules in these networks. Drug screening using these networks, which cannot be achieved through a gene expression analysis alone, requires animal models. Zebrafish embryos have an immature immune system, allowing for a high engraftment rate of human cancer cells transplanted within 48 h after fertilization. Consequently, the time required for engraftment is also reduced. Less than 500 human cancer cells are required for transplantation, enabling the assessment of drug efficacy from clinical samples within approximately 1 week. The cost of raising zebrafish is low, drug efficacy can be evaluated using small amounts of drugs, and their use aligns closely with animal welfare standards. This review aims to discuss the technical aspects of evaluating drug efficacy using zebrafish patient cancer tissue-derived xenograft (zPDX) models and summarize previous studies using zPDX as an avatar model for personalized medicine.
Insights
Zebrafish patient cancer tissue-derived xenografts (zPDX) offer a rapid and cost-effective method for evaluating drug efficacy. This model enables personalized medicine by assessing treatment effectiveness on individual patient cancer cells.
Area of Science:
- Oncology
- Translational Medicine
- Animal Models
Background:
- Current molecular profiling for treatment selection has limitations in predicting efficacy due to reliance on large patient groups.
- Complex diseases require treatments targeting multiple molecular targets within pathological networks.
- Gene expression analysis alone is insufficient for comprehensive drug screening.
Purpose of the Study:
- To discuss the technical aspects of evaluating drug efficacy using zebrafish patient cancer tissue-derived xenograft (zPDX) models.
- To summarize previous studies utilizing zPDX models for personalized cancer medicine.
- To highlight the advantages of zPDX models in drug screening and personalized treatment selection.
Main Methods:
- Utilizing zebrafish embryos (within 48 hours post-fertilization) with immature immune systems for high human cancer cell engraftment rates.
- Transplanting a small number of human cancer cells (<500) to establish xenografts.
- Employing zPDX models for rapid drug efficacy assessment from clinical samples within approximately one week.
Main Results:
- Zebrafish embryos facilitate rapid engraftment of human cancer cells, reducing experimental timelines.
- The zPDX model allows for drug efficacy evaluation using minimal drug quantities.
- This model demonstrates potential for personalized medicine by predicting individual treatment responses.
Conclusions:
- Zebrafish patient cancer tissue-derived xenograft (zPDX) models provide a technically feasible and efficient platform for drug efficacy evaluation.
- The zPDX model supports personalized medicine by enabling rapid assessment of anti-cancer drug effectiveness on patient-derived tissues.
- Advantages include low cost, reduced drug usage, and adherence to animal welfare standards, making zPDX a promising tool in oncology research.
