Developing Humanised Immunocompetent Mouse Models of High-Risk Neuroblastoma

Grant awarded in 2024 following an in-depth review of grant proposals by our world-class Scientific Advisory Board. 

Dr. Toby Trahair from Sydney Children’s Hospital studied the potential for tailored treatment programmes by continuing developments in the field of immuno-oncology.

Developing Humanised Immunocompetent Mouse Models of High-Risk Neuroblastoma

For over a decade, neuroblastoma researchers have worked to develop patient-derived neuroblastoma models in order to study neuroblastoma biology, to test new drug candidates, and to inform individual treatment.  

Immunotherapy treatments for neuroblastoma, including antibody therapy, CAR-T cells, partial-matching transplants, and vaccination, are being increasingly used in clinical trials. While these treatments are effective for some children with neuroblastoma, they are not effective for all.  

Immuno-oncology, a form of cancer treatment that not only covers immune-based treatments but also interactions between cancer and the immune system, is an increasingly important area in neuroblastoma research. However, current patient-derived neuroblastoma models are not well suited for immuno-oncology research because these models are developed without a functioning immune system. 

Therefore, Dr. Trahair’s research project focuses on developing improved neuroblastoma models that contain patient-derived cancer cells as well as a working immune system from the same patient. Dr. Trahair’s research project focuses on developing improved neuroblastoma models that contain patient-derived cancer cells as well as a working immune system from the same patient. These “humanised” mouse models will allow us to study how neuroblastoma interacts with the immune system and, importantly, will provide the opportunity to test new immunotherapy treatments. 

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