Australian & New Zealand Childrens Haematology/Oncology Group


World First: Australian child first in the world to trial new brain cancer treatment

January 2023

A world-first cancer treatment trial, tackling a rare and debilitating paediatric brain tumour, has commenced at the Perth Children’s Hospital. The first Australian patient will be joined by more than 30 other children across the world to trial a treatment for Adamantinomatous Craniopharyngioma (ACP) over the next two years.

ACP is a highly debilitating paediatric brain tumour that currently lacks effective anti-tumour therapies. Current therapies for ACP depend largely on surgery and radiation, both of which are associated with poor quality of life and become more challenging and risky if the disease persists. Recent discoveries with regard to the biological characteristics of ACP indicate that a drug, tocilizumab, currently used in the treatment of arthritis and other diseases, may be effective in the control of this uncommon paediatric brain tumour.  

The new study, “CONNECT1905”, developed by the international clinical trials group, the COllaborative Network for NEuro-oncology Clinical Trials (CONNECT), seeks to identify better ways to treat and improve outcomes for children with recurrent and progressive ACP. The Australian sponsor for the study, the Australian and New Zealand Children’s Haematology and Oncology Group (ANZCHOG) is thrilled to partner with CONNECT to offer the global trial at several Australian childhood cancer centres.

Dr Maryam Fouladi, Chair of CONNECT, said, “We at CONNECT are so excited to partner with ANZCHOG to open the CONNECT1905 study at our Australian sites. Craniopharyngiomas are uncommon paediatric brain tumours that account for about 6% of all brain tumours in children. Resection and/or radiotherapy are currently the standard therapies for patients with this tumour, with few other treatment options. This study plans to assess the efficacy of tocilizumab in children and young adults with this condition who have progressed, despite other interventions, or who are too young to consider radiotherapy as an option, given its long-term side effects. As such, this study provides an alternative therapy for patients for whom few other options exist.”

“We look forward to providing this therapeutic option to patients and families in Australia through our partnership with ANZCHOG and our colleagues at paediatric oncology centers throughout Australia,” Dr Fouladi said.

Australian Principal Investigator Dr Neevika Manoharan said, “CONNECT1905 will be the first study open to Australian children with craniopharyngioma, a rare and often debilitating brain tumour, in decades. It provides these children with a novel treatment option where they have previously had none. We are very excited for children in Australia to have access to this ground-breaking study.”

A patient at Perth Children’s Hospital will be the first in the world to be treated under this study, to be followed by eligible patients at Queensland Children’s Hospital and Sydney Children’s Hospital. The treatment has the potential to benefit ACP patients who may or may not have undergone surgery or radiation as it may help shrink this type of brain tumour or slow its growth.

The CONNECT1905 trial in Australia is co-funded by the Robert Connor Dawes Foundation and Carrie’s Beanies 4 Brain Cancer Foundation.

ANZCHOG is a non-profit organisation committed to ensuring Australian and New Zealand children receive world-class cancer care. We are the peak professional body for paediatric oncologists and health professionals who care for children with cancer, spearheading national initiatives to enhance clinical care through communication, research, networking and education. We are also the national cooperative clinical trials group for childhood cancer, actively working with trial consortia around the globe to ensure Australian and New Zealand children have the opportunity to access the latest promising cancer treatments.