World first trial for paediatric brain cancer launched in NSW


A world-first clinical trial, known as The Deflexifol® At Relapse Trial (DART), proudly supported by Kids with Cancer Foundation, has been launched at Sydney Children’s Hospital, Randwick for children with ependymoma – a rare and devastating form of paediatric brain cancer.

The trial is being led by Professor David Ziegler and Dr Marion Mateos at the Kids Cancer Centre (KCC) and will test Deflexifol® – a new Australian-developed co-formulation of a chemotherapy called 5-FU and leucovorin, in treating ependymoma in children.

Deflexifol® is expected to be a less toxic and more effective chemotherapeutic compared with standard of care versions of these drugs commonly used to treat adult cancers.

Associate Professor David Ziegler, Children’s Cancer Institute

Five-year funding for the DART National Study Coordinator and research costs has been provided by Kids with Cancer Foundation, who have pledged more than $1,200,000 to Sydney Children’s Hospitals 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.

“However for at least one in three children, the tumour will grow back again after this treatment. Sadly, almost all children or adolescents with relapsed ependymoma will die from the disease.

“This treatment has shown some really promising results in treating tumours in adults. Excitingly the old drug 5-FU has been discovered to be active against ependymoma, but until now it hasn’t been possible to get high enough concentrations into the brain.

With this new formulation, we are aiming to use higher doses, with less side effects that should also be much more effective.”

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.”

Kids with Cancer Foundation CEO, Todd Prees, welcomed the announcement.

Todd Press. Photo: linkedin

“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 Prees 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.”

NSW Health Minister, Ryan Park, said: “I am proud and grateful that NSW is at the forefront of this trial. This is world leading research which will make a real difference to the lives of these children and their families.”

The Deflexifol® At Relapse Trial (DART), 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.




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