A groundbreaking trial of personalized vaccines for children suffering from deadly brain cancers is set to commence across eight hospitals in Australia later this year. This four-year trial will involve approximately 70 children facing “dismal prognoses” and aims to test the safety and effectiveness of a novel treatment that leverages the immune system to combat tumors.
Scientific lead Professor Brandon Wainwright from the University of Queensland (UQ) explained that small samples of each child’s tumor will be genetically sequenced to identify unique cancer markers. These markers will then serve as targets for the therapy, resulting in a customized vaccine manufactured within eight weeks of the child’s enrollment in the trial. Participants will receive eight doses of the vaccine, administered bi-weekly, with a booster shot following a few months later.
Harnessing the Power of mRNA Technology
The trial will utilize messenger RNA (mRNA) technology, which gained prominence during the COVID-19 pandemic. Professor Wainwright, in collaboration with Providence Therapeutics and other partners, adapted this technology to treat childhood brain cancer. He noted that mRNA cancer vaccines have already shown promising results in adults with pancreatic cancer and melanoma.
Professor Wainwright emphasized the challenge posed by childhood brain cancers, which often evade detection by the immune system. The mRNA vaccines are designed to expose the cancer, allowing the immune system to recognize and attack cancer markers on tumor cells.
“These patients have a variety of terrible symptoms. If we can reduce the severity of disease and improve their overall survival, we’re on a path that hasn’t been possible at all,” Professor Wainwright said.
Addressing the Deadliest Pediatric Cancer
Brain cancer remains the deadliest of all pediatric cancers, claiming the lives of about 40 Australian children each year. Despite decades of research, survival rates for the most aggressive childhood brain cancers have remained stagnant.
The trial, overseen by the Australian and New Zealand Children’s Haematology/Oncology Group (ANZCHOG), will recruit children from hospitals in New South Wales, Victoria, Queensland, South Australia, and Western Australia. The study targets children with newly diagnosed diffuse midline glioma and relapsed high-grade brain tumors, such as medulloblastoma and ependymoma.
Ethical Considerations and Trial Design
Professor Jordan Hansford, the trial’s clinical lead, stressed the ethical considerations in the trial design. “We don’t use placebos. We think that’s unethical in children who have very, very poor outcome disease,” he stated. Instead, all children in the trial will receive a personalized vaccine, with safety and optimal dosing tested in the initial phase before moving on to assess effectiveness.
“We hope we see some amazing responses, like we have in pancreatic cancer,” Professor Hansford said. “We’re hopeful from the adult studies that at least half of our kids who will be eligible for the study may have some sort of immune response.”
A Personal Mission Driven by Loss
The trial is partially funded by the Marcus Rosin Fund, established by Melbourne mother Marisa Rosin in memory of her son Marcus, who died of brain cancer in 2014 at the age of nine. Marcus had undergone extensive treatment, including a 15-hour surgery and radiation, but the cancer returned after three years.
Ms. Rosin expressed mixed emotions about the trial, saying, “I’m sad that it didn’t happen earlier. It’s a little bit bittersweet for us.” She remains hopeful that the trial will provide other children with more time with their families.
“If they can just give these kids a little bit more time — whether it be one year, two years, three years with their parents, we’ve done our job,” she said.
Future Implications and Broader Applications
The potential success of this trial could pave the way for similar personalized treatments for other types of cancer. Professor Nigel McMillan from Griffith University, although not involved in the trial, praised the approach as “really appropriate” for tackling such a devastating disease.
Professor McMillan explained that the personalized mRNA vaccines function by providing the immune system with a “mug shot” of cancer markers, enabling it to recognize and destroy cancer cells. This innovative approach could revolutionize cancer treatment by allowing the immune system to target unique proteins produced by cancer cells.
As the trial progresses, researchers and families alike remain hopeful that this pioneering approach will offer a new lease on life for children battling brain cancer, potentially transforming the landscape of pediatric oncology.
