World-first trial for paediatric brain cancer

World-first trial for paediatric brain cancer

4 June, 2023

The Deflexifol At Relapse Therapy (DART) trial is proudly supported by Kids with Cancer Foundation and is being led by Professor David Ziegler and Dr Marion Mateos at the Kids Cancer Centre (KCC). The trial will test deflexifol - a new formulation of 5-FU, which is a more effective, less toxic chemotherapeutic drug that is commonly used to treat adult cancers. 

Five-year funding for the DART National Study Coordinator and research costs has been provided by Kids with Cancer Foundation, which has pledged more than $1,200,000 to Sydney Children’s Hospital Foundation to directly support this lifesaving research.

Professor Ziegler said paediatric ependymoma is the third most common brain tumour in children.

“Currently, there are two treatments available; surgery to remove the tumour and radiation therapy,” Prof. Ziegler said.

“About 70 per cent of children are cured with the combination of these intense treatments, but for at least one in three children, they are ineffective. Sadly, almost all children or adolescents with high-risk or relapsed ependymoma will die from the disease.

“This treatment has shown some really promising results in treating tumours in adults and we are hopeful we will see similar results in our paediatric population.” 

Dr Marion Mateos said the trial will provide a new avenue of hope for children diagnosed with this life-threatening cancer. 

“If the trial is successful, it will mean we have something to offer kids with high-risk or relapsed disease who previously had to be told there was nothing more we could do,” Dr Mateos said.

“To commence a trial like this is a massive endeavour and is not something we can do alone. With the support of the Kids with Cancer Foundation, we have been able to raise the bar to find new and better treatments for our patients.”

Peter Bodman, Founder and Director of Kids with Cancer Foundation, welcomed the announcement.

“We are proud to have been working with Sydney Children’s Hospital, Randwick for 25 years, providing funding to the hospital and support to families of kids with cancer being treated there – as well as to children’s hospitals and families of kids with cancer all around Australia,” Mr Bodman said.

“The research and trial could help save the lives of children with relapsed paediatric ependymoma – a condition where there is currently no further treatment options, and we are honoured to be able to fund it through the help of our wonderful donors and fundraisers.”

The Deflexifol At Relapse Therapy (DART) trial, proudly supported by Kids with Cancer Foundation, is operating at Sydney Children’s Hospital, Randwick, with support from the Cancer Institute NSW. The trial will open at children’s cancer centres nationally for children across Australia, sponsored by ANZCHOG with support from the Robert Connor Dawes Foundation. If the trial is successful, it will then be expanded internationally. 

“Our ultimate goal is to make sure every child is cured and no child is left behind and that’s exactly what we’re doing here,” Prof Ziegler said.


MEDIA CONTACT | Sarah Palmer
0428 112 023 |

Last updated Wednesday 25th October 2023