Southern RNA to manufacture personalised vaccines to treat children with deadly brain cancers

Key points

A world-first clinical trial will test cancer vaccines designed specifically for children with advanced or treatment-resistant brain tumours, following a $2.578 million investment from the Australian Government.

The study – co-led by The University of Queensland and the South Australian Health and Medical Research Institute (SAHMRI) and partnered with mRNA medicines platform company Providence Therapeutics and mRNA manufacturer Southern RNA – will provide personalised vaccines to children and adolescents.

Trial sites for the PaedNEO-VAX study will be rolled out at 8 paediatric hospitals in Queensland, South Australia, NSW, Victoria and Western Australia from April.

Clinical lead, Professor Jordan Hansford, from SAHMRI and Adelaide University, said the trial was available for paediatric patients with relapsed and unresponsive high-grade tumours, medulloblastoma, ependymoma, high-grade glioma and newly diagnosed diffuse midline glioma.

“Families should ask their child’s oncologist for information about the trial, with about half of all participants screened likely to be suitable,” Professor Hansford said.

“This is a new, personalised approach to treating these especially difficult brain cancers that we believe has huge potential.”

“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 kill more Australian children than any other disease.

Scientific lead Professor Brandon Wainwright AM, from UQ’s Frazer Institute, 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.

“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.”

The Australia and New Zealand Children’s Haematology and Oncology Group (ANZCHOG) is the trial sponsor and will manage the study.

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.

Hope for children diagnosed with incredibly deadly cancer

Childhood cancer specialist Jordan Hansford, of the Women’s and Children’s Hospital in Adelaide, the trial’s clinical lead, said it would be open to children with newly diagnosed diffuse midline glioma and relapsed and unresponsive high-grade brain tumours, including medulloblastoma and ependymoma.

“We hope we see some amazing responses, like we have in pancreatic cancer,” Professor Hansford said.

The safety and optimal dosing of the personalised vaccines would be tested in the first seven to 10 patients before moving on to phase two of the trial to assess effectiveness.  All children in the trial, which has received funding from the Medical Research Future Fund (MRFF), will be given a personalised vaccine.

Gold Coast-based company Southern RNA will produce the personalised vaccines.

The Australian and New Zealand Children’s Haematology/Oncology Group (ANZCHOG) — the peak body for the region’s paediatric cancer specialists — will oversee the trial.

Collaboration and acknowledgements

• Providence Therapeutics is a Canadian mRNA medicines platform company with expertise in RNA technologies dedicated to developing treatments for conditions with significant unmet medical needs, including oncology and infectious diseases.

• ANZCHOG is the peak body for paediatric cancer clinical trials across Australia and New Zealand, bringing together clinicians, researchers and hospitals to deliver high quality, collaborative research.

• Southern RNA is a leading mRNA contract development and manufacturing organisation based in Queensland, specialising in the production of mRNA raw materials and vaccines.

• The Queensland Children’s Hospital Foundation provided more than $1 million to The University of Queensland’s Children’s Brain Cancer Centre at the Frazer Institute for research that led to the trial.

• The project is supported by the 2024 MRFF Clinical Trials Activity grant (MRF2045405) administered by Monash University; in partnership with Providence Therapeutics, Southern RNA, the ZERO2 Childhood Cancer platform consortium, and Consortium for Australian Children’s Trials in Brain Cancer (MRF2038070).

• ZERO2 Childhood Cancer platform will provide tumour whole-genome sequencing and QIMR Berghofer will complete advanced bioinformatics.

• The discovery and applied research underpinning this trial and future improvements has been funded by an ongoing commitment by not-for-profit organisation The Robert Connor Dawes Foundation (RCD Foundation) to the Wainwright laboratory, and the Waddell laboratory (QIMR Berghofer). RCD Foundation drives global paediatric brain cancer research advancements and supports young patients on their brain cancer journey.

• The Children’s Cancer Foundation (CCF) and The Marcus Rosin Fund in Australia are partnering with RCD Foundation to provide additional funding for the clinical trial.

Southern RNA has locations at Cohort Innovation Space – Gold Coast Health and Knowledge Precinct, and Brisbane Technology Park, Eight Mile Plains, Queensland Australia.