Children with brain cancer given hope in trial of personalised vaccines across eight Australian hospitals
Marcus Rosin (far right), with his sister Mia and parents Fabian and Marisa, died from brain cancer in 2014, aged nine. (Supplied: Marisa Rosin)
In short:
A personalised vaccine for children with deadly brain cancers is to be trialled in eight Australian hospitals.
The trial will last four years and include about 70 children with "dismal prognoses", with a customised vaccine manufactured for each patient.
What's next?
The trial will be rolled out later this year, overseen by the Australian and New Zealand Children's Haematology/Oncology Group.
A world-first trial of a personalised vaccine for children with deadly brain cancers will be rolled out in eight hospitals throughout Australia later this year.
The four-year trial of about 70 children with "dismal prognoses", will test the safety and effectiveness of the experimental treatment, designed to harness the immune system to produce an army of killer T cells — or medical missiles — to attack a child's tumour.
Scientific lead Brandon Wainwright, of the University of Queensland (UQ), said small samples of each child's tumour would be genetically sequenced and assessed for individual cancer markers that could be used as targets for the therapy.
A customised vaccine would then be manufactured for each child within about eight weeks of them enrolling in the trial.
They would receive eight doses — injected into the arm, two weeks apart — followed by a booster shot a couple of months later.
Professor Wainwright, who has spent 30 years researching childhood brain cancers, said the trial would be open to children who "have completely run out of other therapeutic options".
"Because these diseases are so serious at the point at which they'll enter the trial … we will know whether there has been a response in any one individual patient within 12 months of them starting," he said.
Brandon Wainwright says he's "excited" at the prospect of extending the lives of critically ill patients. (Supplied)
The hope was not just to increase survival. Quality of life was also important.
"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.
"We'd be over the moon. We're a bit stressed, we're a bit excited."
Brain cancer kills 40 children every year
Survival rates for the worst childhood brain cancers have remained unchanged for decades.
About 40 Australian children die annually of brain cancer, the deadliest of all paediatric cancers.
The trial will harness messenger RNA (mRNA) technology proven effective in COVID-19 vaccines.
The vaccines are designed to attack cancer markers on the surface of tumour cells. (Supplied)
Professor Wainwright, from UQ's Frazer Institute, worked with Canadian mRNA medicines platform company Providence Therapeutics and other collaborators to leverage the technology into a childhood brain cancer treatment.
He said personalised mRNA cancer vaccines had already shown promising results in adults with pancreatic cancer and melanoma.
Professor Wainwright said childhood brain cancers were adept at hiding from the immune system and needed to be exposed before they could be targeted.
He said the mRNA vaccines were designed to allow the body's immune system to detect the cancer and mount an attack on cancer markers, or proteins, on the surface of tumour cells.
Hospitals in NSW, Victoria, Queensland, South Australia and Western Australia will recruit children for the study.
Jordan Hansford is "really hopeful" the vaccines will be a game changer for young cancer sufferers. (Supplied)
'We don't use placebos'
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.
"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.
"Whether or not that's significant to clear disease or not, obviously we're hopeful for that, we don't know.
"Do I think it's going to work for absolutely every single child? No. But we are really hopeful it will in some."
Professor Hansford, of Adelaide University and the South Australian Health and Medical Research Institute, 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.
"We don't use placebos. We think that's unethical in children who have very, very poor outcome disease," Professor Hansford said.
Clinical trial funded by family who lost 9yo son
Marcus Rosin (left) had a brain operation, but three years later his cancer returned. (Supplied: Marisa Rosin)
Melbourne mum Marisa Rosin's youngest child Marcus died of brain cancer in 2014 after being diagnosed with an ependymoma tumour when he was four.
He had a 15-hour operation to remove the tumour from his brain, followed by radiation, but three years later the cancer returned.
The little boy, who loved playing with Lego and superheroes, was just nine years old when he died.
"It was devastating," Ms Rosin said. "We knew in our hearts that we tried everything."
The Marcus Rosin Fund has provided funding for the clinical trial, dubbed PaedNEO-VAX.
"I'm sad that it didn't happen earlier. It's a little bit bittersweet for us," Ms Rosin said.
"If Marcus was here, I'd put him in the trial, 110 per cent."
Professor Hansford was Marcus's treating doctor.
Ms Rosin said she told him at her son's wake: "I have a feeling that you are going to come up with a cure. Something's going to happen with you."
"To be part of this is truly amazing," she said.
"If this works, it will be incredible and being that Dr Jordan Hansford is part of it … such a beautiful human being.
"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.
"I'm excited. Let's cross everything."
Technology could be applied to other cancers
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.
Professor Nigel McMillan says the vaccines provide a "mug shot" of cancer markers to target. (Supplied: Griffith University)
Griffith University Professor of Medical Sciences, Nigel McMillan, who is not involved in the trial, described the study design as a "really appropriate approach to take to what is a really devastating disease".
Professor McMillan said the personalised mRNA vaccines were designed to provide a patient's immune system with an "identikit photo or mug shot" of cancer markers to target, specific to their individual tumour.
"This allows the immune system to now recognise what is cancer in your body and to go to work," he said.
"By making the immune system recognise those new and unique proteins that the cancer's making, this allows an opportunity for the immune system to now target and kill those cancer cells.
"I think that this is the sort of technology that can be applied to any cancer in the future."