An international clinical trial to test a combination of anticancer drugs for the treatment of the most aggressive form of children's cancer, diffuse midline glioma (DMG) otherwise known as diffuse intrinsic pontine glioma (DIPG) is only months away from Australian hospitals, based on ongoing research conducted by the Hunter Medical Research Institute and University of Newcastle.
Hunter biomedical scientist associate professor Matt Dun has been researching the treatment of DMG/DIPG since his daughter Josephine was diagnosed in February 2018, and said the new clinical trial had come from research started when he and his wife, Dr Phoebe Hindley, were trying to do all they could to help their daughter.
The trial will test the efficacy of a combination of drugs that Dr Dun identified for the treatment of DMG. One of the drugs, paxalisib was discovered for the treatment of DMG/DIPG by Dr Dun when his daughter was diagnosed. The combination of paxalisib with a second drug, ONC201, has shown promising results in laboratory and in several compassionate access cases around the world.
"We knew how insidious - and the word we use in cancer is heterogeneous - these kinds of paediatric brainstem gliomas are, so I knew that one drug wouldn't be enough to provide a long-term successful outcome for children with DMG," Dr Dun said, "But when you use paxalisib as a backbone and combine it with other brain penetrant drugs, the treatment works really nicely."
This most recent clinical trial comes less than two years after Matt and Phoebe's daughter died of the disease in December 2019. Josie was just four and half years old.
"We are excited to see the trial open, which will happen first in the US, then early next year in Australia, as the Dun Lab team has been working 24/7 for three and a half years. It's an opportunity to test this in clinical trial conditions, but mostly to provide options for kids diagnosed with DMG/DIPG in Australia," Dr Dun said, buoyed by the overwhelming support his research has received at home and internationally.
The promise of ongoing research as part of the this most recent clinical trial comes as both Drs Dun and Hindley prepare for the second RUN for RUN DIPG running event to be held on Sunday, September 26, as part of Australian Brain Cancer Awareness Day .
The event, which was originally planned to host around 100 runners on the Fernleigh Track to raise awareness and support for Dr Dun's research, had to be moved virtual to comply with NSW public health orders, but RUN DIPG general manager Troy Bailey said the support had nonetheless been phenomenal.
"We have over 250 registered participants now," he said, "And we're hoping for 500 on Sunday, really showing their support."
"We're encouraging all of our supporters anywhere in the world to get out on Sunday the 26th to follow COVID protocols - to use their hour or two of exercise - and use the #RUNFORRUNDIPG and create that online community and the need to fund research into childhood brain cancer."
Dr Hindley will lead the event, hitting the Fernleigh Track Sunday to complete a half-marathon of 21.1 kilometres, starting from around 7am.
To date, the RUN DIPG has supported $176,000 in grants to support DMG research and $200,000 in research scholarships, with plans to continue laboratory and collaborative grant funding in 2022.
Dr Dun described the next milestone in his research as leading to long-term survival for children diagnosed with the disease.
"Childhood brain cancers affect 150 families around Australia every year, and for those with high-grade tumours their children live on average for 12 months. Even locally, we have four beautiful girls diagnosed with these theoretically rare types of brain cancer in the last three and a half years.
"Without the support of RUN DIPG during our journey we would never have had the preliminary data to encourage the testing of some drugs that we were able to get internationally that now will form part of this international clinical trial.
"I like to think that one day we'll develop treatments that will lead to cures, but we don't have evidence of that yet. What we're trying to do is to treat DMG like a chronic disease - to extend survival to two years, then to four years and so on. And the information that we'll get from this trial, we hope, will uncover the things that will lead to the cures.
The trial is expected to open in October.
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