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Updated: Oct 3, 2025

Drug Screening of Primary Patient Derived Tumor Xenografts in Zebrafish
Published on: April 10, 2020
Preclinical testing of CAR T cells in zebrafish xenografts
Sarah Grissenberger1, Benjamin Salzer2, Susana Pascoal1
1St. Anna Children's Cancer Research Institute, Innovative Cancer Models, Vienna, Austria.
Abstract:
Engineered chimeric antigen receptor T cells (CAR T cells) have emerged as a promising immunotherapy for cancer and have proven to be effective for B cell malignancies. Currently, great efforts are undertaken to expand the application of CAR T cells to other cancer entities, to increase the efficacy of CAR T cell-mediated killing of cancer cells and to reduce possible side effects of CAR T cell therapy. This creates a need for preclinical models to test the many emerging novel CAR designs. Traditionally, mouse xenograft models are applied to investigate the efficacy of CAR T cells in vivo. Here, we describe a complementing xenograft protocol for testing CAR T cells against human leukemia cells in zebrafish embryos. The embryonic zebrafish xenograft promises to be a fast and cost-efficient model and particularly offers live imaging opportunities of CAR T cell distribution and killing of cancer cells in vivo.
Insights
Zebrafish xenografts offer a rapid, cost-effective preclinical model for testing engineered chimeric antigen receptor T cells (CAR T cells) against human leukemia. This model enables live imaging of CAR T cell activity and cancer cell killing in vivo.
Area of Science:
- Immunotherapy
- Oncology
- Preclinical Cancer Models
Background:
- Engineered chimeric antigen receptor T cells (CAR T cells) show promise in treating B cell malignancies.
- Expanding CAR T cell therapy to other cancers requires effective preclinical models for novel designs and efficacy testing.
Purpose of the Study:
- To describe a xenograft protocol using zebrafish embryos for testing CAR T cells against human leukemia.
- To establish a fast, cost-efficient, and visually informative preclinical model for CAR T cell therapy research.
Main Methods:
- Utilized embryonic zebrafish xenografts to model human leukemia.
- Developed a protocol for testing the efficacy of CAR T cells in vivo within the zebrafish embryo model.
Main Results:
- The embryonic zebrafish xenograft model provides a viable platform for evaluating CAR T cell efficacy against leukemia.
- This model facilitates live imaging of CAR T cell distribution and their direct interaction with cancer cells.
Conclusions:
- Zebrafish xenografts serve as a valuable, complementary preclinical model to traditional mouse xenografts for CAR T cell research.
- This approach supports the rapid development and testing of novel CAR T cell therapies for various cancers, including leukemia.

