Australian & New Zealand Childrens Haematology/Oncology Group


New clinical trial now open for children with deadly brain cancers.

August 2022

An international clinical trial testing the latest promising drugs for children diagnosed with a deadly brain cancer is now open in Australia and will open soon in New Zealand.

Diffuse midline gliomas (DMGs) are a group of brain tumours, including diffuse intrinsic pontine glioma (DIPG), which are extremely aggressive and grow rapidly, impacting critical brainstem functions. Despite years of research and much knowledge gained about the underlying biology of DMGs – especially those with H3K27M mutations – little progress has been made to improve outcomes for children diagnosed with these types of tumours. Surgery is usually not possible due to the location of the tumour. Rapid treatment is essential to try to control the growth of the tumour and maintain quality of life for as long as possible. In most cases, radiation therapy is given as an initial treatment and usually combined with chemotherapy. Although tumour control can sometimes be achieved, DMGs tend to progress within a year, and the limited treatment options available at this point are even less effective.

PNOC-022 has been developed by the Pacific Pediatric Neuro-Oncology Consortium (PNOC), a brain cancer clinical trial consortium based in the United States.  PNOC-022 will provide a trial framework to test current promising drugs and rapidly incorporate new agents as soon as favourable data is available.

“Participation in this trial will enable immediate access to a new and promising treatment strategy desperately needed for Australian and New Zealand children diagnosed with this deadly form of brain cancer,” said National Principal Investigator Associate Professor Geoffrey McCowage. “The Children’s Hospital at Westmead is the first hospital in Australia to offer PNOC-022, with other children’s cancer centres throughout Australia and New Zealand opening the trial in the coming months.”

PNOC-022 will include children who are at different stages of disease, including newly diagnosed, after completion of radiation therapy and patients who have evidence of disease progression. All children will be treated with the drug ONC201 and combined with novel agents that have shown additive or synergistic effects in preclinical models. Further details of the trial agents can be found here: PNOC022. There are limited places available on the trial, and recruitment may be paused as data is reviewed.

“The strength of this trial is the adaptive platform design, which allows us to constantly review emerging data, and rapidly incorporate new drugs without having to open a new clinical trial each time,” said Associate Professor McCowage. “Importantly, PNOC-022 is a global research effort, with researchers from all around the world – including Australia and New Zealand – coming together to put forward the most promising options to help these children.”

ANZCHOG will be the national sponsor for participating sites in Australian and New Zealand, working closely with the international PNOC trial team. PNOC-022 is proudly supported by the Isabella and Marcus Foundation, the Isabella and Marcus Paediatric Brainstem Tumour Fund (a sub-fund of Australian Communities Foundation), the Robert Connor Dawes Clinical Trial Program, the Australian Brain Cancer Mission and the Wayne Francis Charitable Trust.

“The Isabella and Marcus Foundation is proud to support this clinical trial, which provides children and young adults in Australia and New Zealand diagnosed with the most intractable brain cancers, access to the most promising new treatment. We hope that these medicines represent the breakthrough we have all been waiting for,” stated Rob DeRose, from the Isabella and Marcus Foundation.

“Robert Connor Dawes Foundation is proud to co-fund the PNOC-022 trial. It is one of two current trials supported through our Clinical Trials Program which we hope to expand into the future, working together with other funders and ANZCHOG,” said Dr Kim Wark, Research Manager at the Robert Connor Dawes Foundation.

“Opening and conducting a clinical trial is a complex and expensive process,” explained A/Professor McCowage. “Each trial opened in Australia and New Zealand needs funding to enable the essential work at each hospital and within ANZCHOG’s National Trial Centre. We appreciate the ongoing support of our funding partners – we couldn’t open paediatric trials in Australia and New Zealand without their support.”

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.