Perth Children’s Hospital (PCH) is expected to soon be able to trial a treatment for children with brain tumours, based specifically on a patient’s individual brain tumour.
The hospital is one of eight sites to take part in a world first clinical trial of cancer vaccines personalised for children with advanced or treatment-resistant brain tumours.
The PaedNEO-VAX study is being rolled out in eight paediatric hospitals across Australia.
Paediatric oncologist Dr Santosh Valvi is the deputy principal investigator of the trial and the principal investigator at PCH.
While it is referred to as a cancer vaccine, due to it being based on mRNA technology, what is being trialled is actually a treatment.
Dr Valvi said the trial would focus on the category of patients who do not have any other treatment options.
“The intent is for it to be curative, but obviously we will have to wait to see how things work out,” he said.
Brain tumours kill more Australian children than any other disease.
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Paediatric patients with relapsed and unresponsive high-grade tumours, medulloblastoma, ependymoma, high-grade glioma and newly diagnosed diffuse midline glioma may be eligible to take part.
Once a biopsy of a tumour is done, it will be sent offsite to be analysed and genome sequencing will identify unique cancer markers in each child’s tumour.
Based on the antigen expression, or neoantigens, a customised vaccine will be developed and it is expected that each customised vaccine will be ready within 10 weeks.
The manufacturing of the vaccines will take place in Queensland.
Dr Valvi said the vaccine could create an immunological reaction in the body and potentially make antibodies against the tumour itself.
He said while personalised vaccines had been used in adults in clinical trial settings on pancreatic cancers and melanomas, this would be the first time such an approach would be trialled in children.
Dr Valvi accessed a similar vaccine for two of his previous patients on compassionate grounds, however their vaccines were made up of what he said was a more generic vaccine with about five commonly expressed neoantigens.
He estimated about five patients per year in Perth may fit the criteria for the upcoming trial.
The personalised vaccines will be administered as nine intramuscular injections over a six-month period.
Work on the trial in Perth is anticipated to begin in a few months, once ethics approval has been granted.
The study is being 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 Therapeutic
Clinical lead, Professor Jordan Hansford, from SAHMRI and Adelaide University said the first phase of the trial would help determine the safest and most effective dose.
“Phase two 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,” he said.
Scientific lead Professor Brandon Wainwright, from the University of Queensland said personalised mRNA cancer vaccines had shown promising results in adults with rare and hard-to-treat cancers, including pancreatic cancer and melanoma.
“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,’’ Professor Wainwright said.
The federal government has provided $2.5 million towards funding the trial.
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