World-first trial for paediatric brain cancer

Picture of Charlotte, 15

A world-first clinical trial has been launched at Sydney Children’s Hospital, Randwick for children with ependymoma – a rare and devastating form of paediatric brain cancer.

Professor David Ziegler and Dr Marion Mateos
The Deflexifol® At Relapse Trial (DART), proudly supported by Kids with Cancer Foundation, 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 children with high-risk or relapsed ependymoma. 

The treatment 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.

Ependymoma is the third most common paediatric brain tumour and forms in the tissues of the brain and spinal cord. Currently, it is treated with surgery and radiation, however if the cancer returns, treatment is ineffectual.

"Surgery and radiation are effective in treating some tumours 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," Professor Ziegler said.

"What we hope is that this trial will offer these patients a new option. 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.”

“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.

Fifteen-year-old Charlie was diagnosed with ependymoma when she was 15 months old. The tumour was one third the size of her brain.

Charlotte with Prof Ziegler
Under the care of Professor Ziegler, and a team of specialists in the Kids Cancer Centre, Charlie braved 18 months of intensive treatment, including 18 surgeries and many rounds chemotherapy and radiation. She also suffered multiple infections and seizures and her family were faced with the reality that Charlie may not be responding to treatment.

“There was so much uncertainty around Charlie’s treatment and if it was working. We were also very aware that if the treatment didn’t work, there were no other options and that was terrifying,” Charlie's mum, Rebecca said.

"My hope is that this trial will be the next step in finding better treatments for ependymoma and that it will change the trajectory for these children and their families.”

Dr Marion Mateos said the trial was made possible by the generosity of the community.

“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.

“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,” Mr Prees said.

For Charlie, she has thankfully recovered and while she does live with the long-term effects of treatment, such as mild deafness and cognitive delay, she is currently cancer-free.

“We are living proof of the amazing work that transpires at the hospital. We know what can be achieved with targeted research, the best clinicians, state-of-the-art equipment and the ongoing and generous support of the community,” Rebecca said.

"Charlie is a survivor. She doesn’t look back or wallow at some of the misfortunes the cancer has brought her over the years. Instead, she looks forward to all the greatness she is yet to achieve.”

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 and Sydney Children's Hospitals Foundation. If the trial is successful, it will then be expanded internationally.