What is the Research for?

The Centre will conduct preclinical and translational research to:

  • Discover and characterise new targets for immunotherapies
  • Design models to test the effectiveness of paediatric immunotherapies
  • Develop new treatments and treatment regimens
  • Monitor the effectiveness of existing immunotherapies to predict treatment response
  • Study the mechanism of action of immunotherapy alone or in combination with other therapies
  • Improve the understanding of tumour immunity in paediatric cancer patients

To learn more about the research projects, see below.


PiggyBac transposon UCB-CAR19-NK cells: a novel off-the-shelf cellular immunotherapy for children with CD19+ blood cancers

Professor Maher Gandhi, The University of Queensland


Chimeric Antigen Receptor (CAR) T cell immunotherapies have resulted in unprecedented responses in relapsed/refractory acute lymphoblastic leukemia and chronic lymphocytic leukemia. But cost and State-based inequality of access prevent their revolutionary potential from being realised. Specifically, three problems remain: 1) the delay in manufacture means that many children will die before the immunotherapy is generated - an off-the-shelf product is needed; 2) side-effects (cytokine release syndrome) which can be devastating; 3) Application to other blood cancer and solid tumours.


This study aims to lay the foundations to generate an affordable, effective and safe off-the-shelf CAR cellular immunotherapy, to produce a ready-to-go treatment for acute lymphoblastic leukemia and chronic lymphocytic leukemia, that can be safely infused into patients without graft versus host disease.


This research is an important step towards allowing future generations of cancer patients to benefit from CAR cellular immunotherapies and will tackle problems 1-3 above. The future plan is to extend this technology to other types of cancer including other blood cancers and solid tumours.


Novel human preclinical tools to better predict outcomes for immunotherapy

Associate Professor Kristen Radford, The University of Queensland


Over 230 Australian children are diagnosed with leukemia each year. They receive intensive chemotherapy which can have long term side-effects and some have poor chances of survival. Immunotherapies have enormous potential for the treatment of paediatric cancers, especially those for which current treatments are ineffective or associated with toxicity and/or high risk of relapse.


New treatments, which use the patient’s immune system, are desperately needed and hold promise of improving survival whilst reducing treatment-related toxicity for leukemia patients. Preclinical studies are designed to test the safety and effectiveness of new treatments before testing in humans and primarily use animal models. However, there are no reliable preclinical models that can evaluate new human-specific immunotherapies and prioritise those most likely to be successful for clinical trial.


This project aims to establish a new model that will help researchers test how the human immune system responds to a potential treatment before reaching humans. The model will be comprised of human leukemia and a patient matched human immune system that can more effectively identify the best new immunotherapies to trial in childhood leukemias. The researchers have developed a new vaccine they would like to test before proceeding to clinical trial.


Targeting and eliminating paediatric cancers with chimeric antigen receptor engineered natural killer cells, a new hope for cancer immunotherapy.

Dr Fernando Guimaraes, The University of Queensland


In Queensland, brain cancers are the leading cause of disease-related death in children, and bone cancers in adolescents. Medulloblastoma and osteosarcoma are the most common malignant brain and bone tumours, respectively, both with marginal survival chances when these tumours are refractory or recurrent. Current cancer immunotherapies largely focus on genetic engineering of T cells, a type of white cells, taken from the patient's own blood. However, such approaches suffer from significant negative side effects and technical challenges related to the need to build the T cells from each patient, specifically.


Natural killer (NK) cells are a type of white cells known for their ability to kill cancer cells. NK cells are very promising as cancer treatment as they do not attack healthy tissues and are less toxic than conventional therapies. To address the unmet need for effective, widely and rapidly accessible immunotherapies, the research team will employ genetic engineering techniques to generate NK cells that can kill cancer cells more efficiently and be used in any patient. This project will investigate the efficacy of these cells in these two types of cancers – medulloblastoma and osteosarcoma.


The vision of this project is to create “super-killer” NK cells to deliver a new, safe, and highly effective cancer treatment, with the aim to develop a clinic-ready super-killer NK product for generic off-the-shelf use against solid cancers. This project will have a significant health and economic impact and will attract investments and commercial opportunities to further develop the technology to treat this devastating disease in children.

Find out more

Children’s Hospital Foundation

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