A HUNTER charity hopes to sponsor a new clinical trial for DIPG in Australia this year, offering an option to the 20 children likely to be diagnosed in 2021.
Dr Matt Dun, a cancer researcher at the University of Newcastle and HMRI, is part of an international research group for DIPG - the brain stem cancer that claimed the life of his own little girl, Josephine, in 2019.
Ahead of the latest RUN DIPG fundraiser on Sunday - a 20 kilometre swim at Merewether baths - Dr Dun said the money already raised by the charity had helped take his team's ideas "from the bench in the lab" through the pre-clinical pipeline.
Now, an international clinical trial based on those discoveries is beginning in the US with Dr Dun's global collaborators.
If all goes to plan, RUN DIPG will support the introduction of the trial - called "DMG-ACT" - in Australia in the coming months.
"We have given the protocol to our regulatory body here in Australia," he said. "It is looking good, and we're looking to get the trial started here in the first half of this year."
We are hoping this platform will give Australian families some options at least, because at the moment, there isn't any.Dr Matt Dun
Dr Dun's daughter "Josie" had not responded to the standard-of-care radiotherapy for DIPG, excluding her from any trials.
"The way we have written our trial is that all patients will be included at diagnosis following radiotherapy, and when the disease progresses following radiotherapy," Dr Dun said.
"It's a patient-centred trial. It's adaptive. We are hoping this platform will give Australian families some options at least, because at the moment, there isn't any."
So far, the combination of drugs they are proposing to test have only been used under compassionate access for children with advanced DIPG.
It is the drug combination that Josie received for the last 40 weeks of her life.
"What we really want to do is test the utility of the combination in patients immediately after they have their standard of care radiotherapy, and see if we can get a benefit," he said.
"And if we can get a benefit, it gives us more time - both locally and internationally - to develop better, more innovative, more curable approaches."
Dr Dun said there were some "sophisticated" cell-based therapies under investigation in the US, which they hoped would provide a benefit.
"But the cost of going onto that trial is more than a million dollars," he said. "And we need something now.
"We need something to help every Australian kid diagnosed now. I'm not saying ours is the silver bullet, but we are hoping we can buy some time. In the meantime, we are working incredibly hard in the lab to come up with the next plan."
Dr Dun said a group of "average swimmers", including himself, planned to swim 20 kilometres at Merewether Baths on Sunday - one kilometre for every Australian child that would be diagnosed with DIPG this year.
The event was originally intended to be held in Lake Macquarie, with participants preparing to swim to Pulbah Island. The recent shark attack in the lake had prompted the move to the baths.
"This swim will help us keep this real discovery-based research going, and in the Australian research climate right now, governments are hesitant, or almost aren't, funding discovery research anymore," Dr Dun said.
"They want to take the drugs that have been synthesised and developed already and move them through the clinical trial pipeline. But for DIPG, that just doesn't work. We don't have the drugs made yet that we know will work specifically in the brain stem, that can cross the blood-brain barrier, that can be an oral formulation so kids can take it at home, that shows efficacy in a kid with a brain stem glioma. So we are making drugs and testing them, and it is all hugely expensive."
Dr Dun said the international DIPG research community was a welcoming one that "knew what it was up against". It was the "Holy Grail" of cancer research. The "unbeatable".
"It's landing on the moon times 100," he said. "Neil Armstrong landed on the moon a few years after his daughter died of DIPG, and yet we still don't have a treatment."
Dr Dun said DIPG had essentially become the "Hunter's paediatric cancer" due to its over-representation in the region.
"If you do the stats on how many we should have, we should have one case every four or five years," he said.
"We have had three deaths in three years."
Support the swim at justgiving.com/campaign/takeonthelake.
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