Australia Prepares World-First mRNA Vaccine Trial for Children with Aggressive Brain Tumors
核心洞察
Australia is preparing to launch the world's first clinical trial of PaedNeoVax (PTX-108) (搜索), a personalized mRNA vaccine for children with aggressive brain tumors, though a start date has not yet been set.
The vaccine uses small samples of a patient's tumor to engage T cells to detect and attack cancer markers on tumor cell surfaces, with the trial led clinically by Professor Jordan Hansford of SAHMRI and the University of Adelaide.
The federal government has supported PTX-108 development with a $2.58 million Medical Research Future Fund grant, while the family of 16-year-old Coby Metzler, diagnosed with diffuse midline glioma (搜索), is seeking compassionate access outside the trial.
Australia is preparing to begin the world's first clinical trial of a personalized mRNA vaccine for children with aggressive brain tumors. The vaccine, called PaedNeoVax (PTX-108) (搜索), has not yet received a set start date, but the study will be rolled out at pediatric oncology centers across Australia under strict ethical and regulatory frameworks.
The trial is clinically led by Professor Jordan Hansford of the South Australian Health and Medical Research Institute (SAHMRI) and the University of Adelaide, and scientifically led by Brandon Wainwright of the University of Queensland (搜索)'s Frazer Institute and QIMR Berghofer Medical Research Institute.
"This is a new, personalised approach to treating these especially difficult brain cancers that we believe has huge potential," study chair Professor Hansford said.
How the Personalized Vaccine Works
PaedNeoVax uses mRNA technology to create a customized vaccine from small samples of a particular patient's tumor. The vaccine is designed to engage the immune system's T cells to identify and attack markers, or proteins, on the surface of tumor cells.
At the initial stage of the trial, researchers will determine the safest and most effective dosage, closely monitoring the first participants before expanding the study. Professor Hansford noted that the study protocol includes eligibility criteria approved by a human research ethics committee.
A Family's Race Against Time
The urgency of the trial is underscored by the case of 16-year-old Coby Metzler, who was diagnosed 14 months ago with level four brain cancer (搜索) and given a 12 to 18-month life expectancy. An MRI scan in June 2025 revealed a diffuse midline glioma (搜索) (DMG), an aggressive tumor that usually affects children and is particularly hard to treat because it spreads through the central nervous system and grows in the thalamus, brain stem, and spinal cord.
Coby was treated with radiation and gained access to another clinical drug trial in November last year. For a time, his tumor decreased in size, but by March this year his cancer began to progress again.
"We now understand that his cancer is progressing into his brain stem, which will take away his functions one by one," his mother, Brigette Metzler, said.
Facing the prospect that her son will not have a long enough life expectancy to qualify for the trial, Ms Metzler has prepared an appeal concerning her son's access to the vaccine outside the trial, with the help of family members and acquaintances with legal and medical expertise.
"The timeline for delivery of the trial does mean that some children will die waiting for a place," she said. "My son is likely to be one of them."
Access to Unapproved Medicines
Professor Hansford emphasized that PaedNeoVax is an experimental vaccine that cannot currently be purchased anywhere in the world. He said requests for access to unapproved medicines outside a clinical trial are considered directly by the medical team, the family, and the manufacturer, with no trial group involvement.
"Any study we perform has criteria for entry that is outlined in the trial protocol as approved by Human Research Ethics Committee," he said.
The Australian federal government has supported the development of PTX-108 with a grant of 2.58 million Australian dollars through the Medical Research Future Fund. Ms Metzler is asking the government to fund her son's access to treatment outside the trial when the vaccine becomes available.
"Australian law expressly permits access to therapies outside of clinical trials," Ms Metzler said. She added that the risk to a child seeking access outside the trial "is no greater than it would be if they were patient number one."
The Case for Compassionate Access and Clinical Trials
Oliver Jarrett, a board director of Brain Tumour Alliance Australia (搜索) and a former pharmaceutical executive, said pharmaceutical companies could provide experimental medicines to patients free of charge through compassionate access programs.
"If it hasn't been studied then provide it free of charge, that gives patients an option, and it doesn't force the government into a price point," he said.
Mr Jarrett noted that Australia currently secures only about 5-10 per cent of the world's phase two and three cancer trials, adding that "there are at least 10 times more potential treatments that are being studied around the world that people aren't able to access within Australia."
Liz Dawes, CEO and founder of the Robert Connor Dawes Foundation (搜索), which invested in the early stages of designing and developing the mRNA vaccine for pediatric brain cancer (搜索), stressed the importance of clinical trials for safety.
"Unfortunately, the reality we face with untested therapies is that without clinical trials, we simply don't have the safety blueprint needed to protect a sick child from accidental harm," Ms Dawes said.
Brain tumors kill more Australian children than any other disease, with 40 Australian children dying of brain cancer (搜索) every year.