A world-first Australian trial will create personalised mRNA vaccines for children with some of the deadliest brain tumours, manufactured locally within weeks of diagnosis.
Children with some of the most aggressive brain cancers will be offered bespoke vaccines designed from their own tumours in a landmark Australian trial that researchers say could redefine treatment for paediatric cancer.
The PaedNEO-VAX study will roll out across eight children’s hospitals nationwide, using genome sequencing to decode each patient’s tumour and identify unique markers that can be targeted with a personalised mRNA vaccine, manufactured in Queensland by Southern RNA.
Backed by a $2.578 million federal investment, the study is co-led by researchers from The University of Queensland and South Australian Health and Medical Research Institute, alongside mRNA medicines platform company Providence Therapeutics and mRNA manufacturer Southern RNA.
They will test whether custom-built vaccines can slow or stop cancers that are often resistant to standard treatment.
Clinical lead Professor Jordan Hansford, from SAHMRI and Adelaide University, said the study would target children with relapsed or treatment-resistant tumours, as well as those newly diagnosed with diffuse midline glioma, one of the most lethal childhood brain cancers.
“This is a new, personalised approach to treating these especially difficult brain cancers that we believe has huge potential,” he said.
“Phase I of the trial will help us determine the safest and most effective dose.
“Phase II will assess how well the treatment works including whether it slows cancer progression and improves overall survival and quality of life for participating children and their families.”
Brain tumours remain the leading cause of disease-related death in Australian children, underscoring the urgency behind the trial. Researchers hope the approach, which has shown promise in adult cancers such as melanoma and pancreatic cancer, can be translated to paediatric patients.
Scientific lead Professor Brandon Wainwright AM, from UQ’s Frazer Institute, said the process began with whole-genome sequencing and advanced bioinformatics to pinpoint tumour-specific targets, before a custom vaccine is designed and produced.
“We are excited that after many years of research in our laboratory, we might offer a glimmer of hope for children with some of the most aggressive and treatment-resistant tumours,’’ he said.
“After identifying unique cancer markers in each child’s tumour using genome sequencing and advanced data analysis, we will select tumour-specific vaccine targets.
“Customised vaccines for each child will then be manufactured in Queensland by Southern RNA and then sent to the trial sites for dosing.
“We expect to have a customised vaccine ready within about 10 weeks of them enrolling in the trial.”
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Each child in the trial will receive their own vaccine, with early phases focused on safety and dosing before expanding to assess whether the treatment can extend survival and improve quality of life.
The trial has received funding from the Medical Research Future Fund (MRFF), with platform mRNA technology and investment coming from Providence Therapeutics.
Providence Therapeutics founder and CEO Brad Sorenson said he was excited to partner with Australian researchers and oncologists to tackle these important cancers.
“If your child has life-threatening cancer, as a parent, you just want to have options, some way to fight back,” he said.
Families and patient advocates have been closely involved in the design of the trial, ensuring the research reflects the needs of those affected.
