Australia Launches World-First Pediatric Brain Cancer Trial Using Personalized mRNA Vaccines

Feb. 3, 2026-Australia has launched a world-first clinical trial testing personalised mRNA cancer vaccines for children and adolescents with advanced or treatment-resistant brain tumours, supported by a $2.578 million investment from the Australian Government.

The PaedNEO-VAX study will deliver custom-built mRNA vaccines tailored to each patient’s tumour, using genome sequencing to identify unique cancer-specific markers. The trial is co-led by The University of Queensland and the South Australian Health and Medical Research Institute (SAHMRI), in partnership with mRNA medicines platform company Providence Therapeutics.

The mRNA-based cancer vaccine trial will be rolled out across eight paediatric hospitals in Queensland, South Australia, New South Wales, Victoria and Western Australia. Eligible patients include children with relapsed or refractory high-grade brain tumours, medulloblastoma, ependymoma, high-grade glioma and newly diagnosed diffuse midline glioma.

Using advanced genomic analysis, each child’s tumour will be sequenced to identify tumour-specific antigens, which will then be encoded into bespoke mRNA vaccine constructs. Phase I of the PaedNEO-VAX trial will evaluate the safety and optimal dosing of the personalised mRNA vaccines, while Phase II will assess clinical efficacy, including effects on tumour progression, survival outcomes and quality of life.

The trial builds on growing evidence that mRNA cancer vaccines can stimulate targeted immune responses and have already shown promise in adult cancers such as melanoma and pancreatic cancer. The Australia and New Zealand Children’s Haematology and Oncology Group (ANZCHOG) is sponsoring and managing the study, with funding from the Medical Research Future Fund (MRFF) and platform mRNA support from Providence Therapeutics.

Brain tumours remain the leading cause of disease-related death in Australian children, underscoring the urgent need for innovative mRNA-based therapeutic strategies.