We have funded the Australian and New Zealand Haematology and Oncology Group (ANZCHOG) to administer paediatric brain cancer trials. To date, RCD has contributed funding towards 7 trials, all of which are still open for recruitment except for AIM BRAIN. This funding has allowed increased access and participation for Australian and New Zealand children with brain cancer in international and national trials which otherwise may not have occurred. These include:

NICHE-HHG trial (PNOC-019)

The first pilot study to examine the effectiveness of combining two biological agents (nivolumab and ipilimumab) which boost the natural immune response against the tumour (known as immunotherapy) for children and young adults with recurrent or progressive high-grade gliomas.

LGG Avastin Trial

A Phase II trial testing a known chemotherapy agent (vinblastine) and biological agent (bevacizumab – an antibody therapy) in combination, in children with progressive low-grade glioma that can’t be surgically removed and haven’t had chemotherapy before. Each of these agents has previously demonstrated effectiveness in other cancer types so hopefully it will also be effective with this type of brain cancer.


An adaptive trial testing a small drug molecule (ONC201), previously shown to be effective in diffuse midline gliomas, in combination with a novel agent paxalisib, together with standard treatment to see if this can improve outcomes for children and young adults with DMG including DIPG at diagnosis, post radiotherapy or time of progression.


A study to determine the specificity and sensitivity of a particular genetic test on brain tumours for improved diagnosis and treatment. This four-year study yielded successful results which have allowed the test to be transitioned into the clinic and accredited by the regulators. It is now considered part of standard of care when typing a tumour.


The Phase I component of this trial will determine the best dose of a new co-formulation of a chemotherapy (Deflexifol®) in children and young adults with refractory or recurrent brain tumours, or patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG)/diffuse midline glioma (DMG) who have completed radiotherapy. The Phase II component will test to see if Deflexifol® is an effective treatment for patients with relapsed ependymoma.


Investigates the safety and effectiveness of a biological agent (tocilizumab, an antibody therapy) to induce an anit-tumour response in children with adamantinomatous craniopharyngioma (ACP), a highly debilitating paediatric brain tumour that has very limited treatment options.


A Phase 2 trial in which children with adamantinomatous craniopharyngioma (ACP) will receive a small molecule inhibitor of an important biological pathway (MAPK/ERK) associated with the tumour to assess its effectiveness.