Telethon Backs Groundbreaking CAR T-Cell Research for Children with Neuroblastoma

The RPH Research Foundation is proud to announce that the Channel 7 Telethon Trust is supporting an innovative research project aimed at advancing a potentially life-saving treatment for children with one of the most aggressive forms of cancer.

Led by A/Prof Zlatibor Velickovic, the project will establish the foundations of CAR T-cell therapy for children with high-risk neuroblastoma, a devastating childhood cancer with survival rates that have remained alarmingly low for decades.

Neuroblastoma is the most common solid tumour of childhood, typically occurring between birth and five years of age. It results from immature nerve cells within the sympathetic nervous system.  Neuroblastoma accounts for approximately 11% of all paediatric cancer deaths. While some children respond well to treatment, those diagnosed with high-risk and relapsed neuroblastoma have limited treatment options and face a survival rate of less than 10%, representing an urgent unmet medical need.

Thanks to support from Telethon, Cell and Tissue Therapies WA (CTTWA) will undertake a pilot study to establish manufacturing feasibility for GD2-directed Chimeric Antigen Receptor (CAR) T-cells to help vulnerable children who have exhausted conventional treatment options. This innovative approach targets the GD2 ganglioside antigen, highly expressed on neuroblastoma cells, offering new hope where traditional therapies have failed.

The team will develop Good Manufacturing Practice protocols, validate production processes, and assess scalability to enable future early-phase clinical trials in Western Australia for this promising immunotherapy approach.

This research represents the first initiative in Australia to establish GMP manufacturing capabilities specifically for GD2-targeted CAR T-cell therapy in paediatric neuroblastoma. While CAR T-cell manufacturing has been developed for haematological malignancies, solid tumour applications remain extremely limited globally, making this a pioneering effort.

By establishing local manufacturing capabilities, this research will improve access to cutting-edge immunotherapy for WA children, reducing the need for interstate or overseas treatment and providing hope for WA families.  

Chief Investigator Zlatibor Velickovic with Cell and Tissue Therapies WA Team