News Release 

Australia to lead world-first trial in children with brain cancer

Australia will join an international clinical trial to test a new treatment in children with brain cancer, due to launch in later this year

Children's Cancer Institute Australia

Australia will join an international clinical trial to test a new treatment in children with brain cancer, due to launch in later this year. Brain cancer kills more children in Australia than any other cancer, with the Australian Institute of Health and Welfare reporting about 40 deaths each year in children under 15 years of age.

Funded by a $1.1 million grant from the National Health and Medical Research Council, part of a $3 million investment in childhood brain cancer research announced by the Australian Government this week, the trial will be led from Sydney Children's Hospital, Randwick, and opened at every children's hospital in Australia.

"This trial will be focusing on children with the most commonly diagnosed type of brain tumour, low-grade gliomas," said Associate Professor David Ziegler, senior paediatric oncologist at Kids Cancer Centre, Sydney Children's Hospital, Randwick, and head of the Brain Tumour Group at Children's Cancer Institute, who will head the clinical trial in Australia. "Gliomas can be very difficult to treat. When a tumour isn't able to be removed by surgery, the child may face years of treatment and often suffers major morbidities. For such patients the only treatment options are cytotoxic chemotherapy and/or radiotherapy, resulting in significant short and long term toxicity. We need new treatments that are not only more effective, but also much safer to use in children."

The new treatment being tested in the clinical trial could be just what's needed. One of a new generation of drugs known as 'targeted therapies', it has been developed to target cancer cells directly and leave healthy cells unharmed, thereby causing far fewer side effects.

"The targeted therapy we will be testing has been specifically designed to shut off the genetic driver of this type of tumour, and can be taken as a daily pill" said Ziegler. "If shown to be successful, it could replace chemotherapy as the standard treatment for this type of cancer, significantly improving quality of life for survivors."

A media release put out by The Hon Greg Hunt MP, Minister for Health, on 7 June, said the government recognised the essential nature of clinical trials for evaluating the effectiveness and safety of medicines. "We will be monitoring the outcomes of this research closely and we look forward to seeing much needed support and access to better treatments roll out to children and their families," it stated.


About Kids Cancer Centre, Sydney Children's Hospital, Randwick

The Kids Cancer Centre (KCC) at Sydney Children's Hospital, Randwick has been treating children with cancer and blood diseases in NSW, Australia and the Asia-Pacific region for nearly 50 years. Almost two thirds of children treated for cancer or leukaemia at the Centre are enrolled on clinical trials, in a unique model where research and clinical care are one, ensuring the best possible care for children and their families. During that time the survival rates for children with cancer have gone from 10 per cent to nearly 80 per cent. Clinical and research staff from the Centre have made major international and national contributions to the expansion of knowledge in the area: from important discoveries around bone marrow transplantation, chemotherapy for relapsed solid tumours and leukaemia, to the invention of novel anti-cancer drug combinations and minimal residual disease testing in acute lymphoblastic leukaemia. Centre staff have been leaders in devising new methods of outreach and home nursing, and in developing modern approaches to the bereaved family. These achievements have been founded on academic excellence and clinical expertise. In the past five years alone, the Centre's staff have published over 200 papers in peer-reviewed medical and scientific journals and been awarded more than $60 million in competitive grant funding. The past 20 years has also seen a total of eight clinical staff receive the Order of Australia honours for their work. More at or

About Children's Cancer Institute

Originally founded by two fathers of children with cancer in 1976, Children's Cancer Institute is the only independent medical research institute in Australia wholly dedicated to research into the causes, prevention and cure of childhood cancer. Forty years on, our vision is to save the lives of all children with cancer and improve their long-term health, through research. The Institute has grown to now employ over 300 researchers, operational staff and students, and has established a national and international reputation for scientific excellence. Our focus is on translational research, and we have an integrated team of laboratory researchers and clinician scientists who work together in partnership to discover new treatments which can be progressed from the lab bench to the beds of children on wards in our hospitals as quickly as possible. These new treatments are specifically targeting childhood cancers, so we can develop safer and more effective drugs and drug combinations that will minimise side-effects and ultimately give children with cancer the best chance of a cure with the highest possible quality of life. More at

Tami Bradley -
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