A Newcastle researcher will receive a major honour for his work to improve the survival of children diagnosed with an aggressive and deadly cancer.
Professor Matt Dun will receive the Australian Society for Medical Research medal at the National Press Club on Tuesday.
Professor Dun, of University of Newcastle and Hunter Medical Research Institute, said it was "a huge honour and extremely humbling".
"It comes with a good opportunity to advocate for health and medical research, and for families that have faced and will face this terrible monster."
Professor Dun is focused on diffuse intrinsic pontine glioma (DIPG), having lost his daughter Josephine to this disease.
He leads a team of more than 20 researchers, who are analysing DIPG tumours to pinpoint disease subtypes and further refine treatments.
In a press club address, Professor Dun will share his personal journey and discuss his pioneering research, which has extended lives and offered fresh hope.
Josephine was diagnosed in 2018, shortly before she turned three.
Most children with DIPG survive only six to 12 months after diagnosis.
Josephine died at age four in December 2019, almost two years after diagnosis.
"I knew I had to act immediately. Knowing there were no effective treatments for DIPG, I couldn't just accept it," Professor Dun said.
"I reached out to the world's leading DIPG researchers and gathered information on potential treatments that were in development.
"My goal was to find a way to prolong Josephine's survival, to buy enough time until more effective therapies became available."
He and his team pinpointed treatments that extended her life by a year.
"The therapies stabilised the disease and Josie learnt to walk again, swim and have her one and only dance concert," Professor Dun says.
"It gave my family precious time filled with joy and memories, something we will be forever grateful for.
"We had a great Christmas with her cousins and our family."
When Josephine began her experimental treatment, she regained the ability to walk and swim, but only in circles as the tumour had paralysed the right side of her body.
She became well enough for Professor Dun to leave her bedside, return to the lab and focus on developing the next steps.
"I knew the tumour would eventually adapt to the therapies that we had discovered," he said.
Through the Zero Childhood Cancer program, Professor Dun's team pinpointed specific mutations in Josephine's tumour, providing potential drug targets.
"By chance, I discovered a drug called paxalisib that could penetrate the brain and suppress the activity of one of these key mutations."
Since then, his team's research showed that "this gene is essential for the growth of all DIPGs, highlighting a therapeutic vulnerability that could be targeted".
Four other key mutations in Josephine's tumour informed the team that "one drug alone wouldn't be enough".
His lab identified the most suitable therapy to combine with paxalisib.
"After proving the combination was effective in the lab, we gained access to both therapies, allowing Josephine to become the first child to receive this treatment," he said.
Since then, clinical trials showed children treated in this way "survive an average of 8.7 additional months when started at disease progression".
This was four times longer than historical data and 110 per cent longer than children who received an additional dose of radiation therapy alone.
"However, tragically, children treated at diagnosis with this combination still only survive around 13 months," he said.
Professor Dun's data suggested that the drugs cause "immune cells to invade the brain that help drive resistance to the treatment".
"We are now focusing on understanding these mechanisms better and finding ways to harness and capitalise on these observations to improve outcomes further," he said.
On the medal, Professor Dun said he was "incredibly proud of the work of my team, the support of my collaborators and my institute".
He was grateful for the "generosity of DIPG families".
"Together we can make a difference for those facing the most devastating challenges.
"Collaboration and generosity can triumph over isolation and adversity, showing that real progress is possible when we work together."
He said "significant investment" in Australian health and medical research was needed.
"Health is the foundation of life and diseases like DIPG can be beaten," he said.
"Investing in research promotes education, wellbeing and quality of life, creating a ripple effect that benefits society as a whole.
"We need sustained funding and support to ensure researchers can continue to develop innovative treatments and make meaningful progress against the toughest diseases."
Donate to Professor Dun's charity at rundipg.org.
