Australia Launches World-First Personalized Vaccine Trial for Childhood Brain Cancers
Australia is set to initiate a world-first clinical trial later this year for a personalized vaccine designed to treat children with deadly brain cancers. This four-year trial, involving approximately 70 children with severe prognoses, will be conducted across eight hospitals nationwide.
The trial aims to evaluate the safety and effectiveness of an experimental treatment that leverages the immune system to target a child's tumor.
"Small samples of each child's tumor would be genetically sequenced to identify individual cancer markers for therapeutic targeting. A customized vaccine will then be manufactured for each child within about eight weeks of enrollment." — Professor Brandon Wainwright
Professor Brandon Wainwright of the University of Queensland (UQ), the scientific lead, highlighted that participants will receive eight doses, administered two weeks apart, followed by a booster shot several months later.
"The trial is intended for children who have exhausted other treatment options. The objective is to increase survival rates and improve the quality of life for patients."
Responses in individual patients are expected to be observable within 12 months of starting the treatment.
The Urgency: A Critical Need
Brain cancer is responsible for approximately 40 childhood deaths annually in Australia, establishing it as the deadliest pediatric cancer.
"Survival rates for severe childhood brain cancers have not changed significantly for decades."
Leveraging mRNA Technology
The trial capitalizes on messenger RNA (mRNA) technology, previously utilized in COVID-19 vaccines. These experimental vaccines are designed to enable the body's immune system to detect and attack specific cancer markers, or proteins, on the surface of tumor cells.
Professor Wainwright, from UQ's Frazer Institute, collaborated with Providence Therapeutics, a Canadian mRNA medicines platform company, and other partners to adapt this technology for childhood brain cancer treatment. Personalized mRNA cancer vaccines have already demonstrated promising results in adult patients with pancreatic cancer and melanoma.
Trial Details and Patient Eligibility
Recruitment for the study, known as PaedNEO-VAX, will occur at hospitals in New South Wales, Victoria, Queensland, South Australia, and Western Australia.
Professor Jordan Hansford, a childhood cancer specialist at the Women's and Children's Hospital in Adelaide and the trial's clinical lead, confirmed the trial is open to children with newly diagnosed diffuse midline glioma, as well as those with relapsed and unresponsive high-grade brain tumors, including medulloblastoma and ependymoma.
The initial phase will assess the safety and optimal dosing in the first seven to ten patients, before proceeding to phase two to evaluate effectiveness. All children in the trial will receive a personalized vaccine, as placebos are considered unethical in these severe cases.
Funding and Oversight
The trial received funding from the Medical Research Future Fund (MRFF) and additional support from The Marcus Rosin Fund. This fund was established by Marisa Rosin, whose son Marcus tragically died from brain cancer in 2014 at age nine.
Gold Coast-based company Southern RNA will be responsible for producing the personalized vaccines. The trial will be overseen by the Australian and New Zealand Children's Haematology/Oncology Group (ANZCHOG).
Professor Nigel McMillan of Griffith University, who is not involved in the trial, described the study design as an appropriate approach for this disease, explaining that the personalized mRNA vaccines provide the patient's immune system with specific...