Children’s Cancer Institute and Sydney Children’s Hospital, Randwick announce ground breaking clinical trial for childhood cancer in Australia

02 Sep 2014

New trial offers exciting new treatment approach for aggressive relapsed neuroblastoma

Children’s Cancer Institute and Sydney Children’s Hospital, Randwick have today announced a major, new international clinical trial designed to help combat neuroblastoma – the most common solid tumour to affect infants and young children today.

Coinciding with International Childhood Cancer Awareness Month in September, the clinical trial offers an exciting new treatment for children diagnosed with relapsed neuroblastoma. The trial uses an existing small molecule drug, called Difluoromethylornithine (DFMO), previously used for treating African Sleeping Sickness.

Children’s Cancer Institute’s research has shown that DFMO, when used in combination with modern chemotherapy, attacks neuroblastoma much more effectively than chemotherapy drugs alone.

The clinical trial, led by Dr David Ziegler, Paediatric Oncologist for the Kids Cancer Centre at Sydney Children’s Hospital, Randwick and Group Leader of Targeted Therapies Program at Children’s Cancer Institute, is an international trial being run across 14 hospitals in North America.

As one of Australia’s leading specialist medical centres for children, Sydney Children’s Hospital, part of The Sydney Children’s Hospitals Network, is the only site in the country to host the trial.

Dr David Ziegler said “The drug DFMO essentially makes the current chemotherapies more effective in killing neuroblastoma cells. To initiate this trial we developed a new formulation of DFMO that can be given to children as a syrup. We hope that this will be a new way forward for children diagnosed with high-risk neuroblastoma.”

Professor Michelle Haber AM, Executive Director, Children’s Cancer Institute said: “Currently,a child with high-risk neuroblastoma who relapses after initial treatment is considered incurable. Children’s Cancer Institute, along with our partner Sydney Children’s Hospital, is determined to put an end to this devastating disease. If the DFMO clinical trial proves successful it could fundamentally change the way we approach neuroblastoma treatment.

“Neuroblastoma accounts for 15 per cent of all paediatric cancer deaths in Australia and high-risk neuroblastoma has a survival rate of just 1 in 2. This is why it’s so critical that we progress our discoveries into actual treatments for kids as quickly as possible.”

The trial is supported by Australian Lions Childhood Cancer Research Foundation, The Kids’ Cancer Project, Cancer Institute NSW and the National Health and Medical Research Council (NHMRC), and internationally by New Approaches to Neuroblastoma Therapy (NANT).