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An Australian-first clinical trial giving hope to children diagnosed with a terrible brain tumour, Diffuse Intrinsic Pontine Glioma (DIPG), has opened at The Children鈥檚 黑料吃瓜群网 at Westmead (CHW).

CHW is currently one of nine sites worldwide to open the trial, known as PNOC-022, which is testing the effectiveness of promising new drug combinations, including the oral drug ONC201.

Associate Professor Geoffrey McCowage, National Principal Investigator for the trial and Senior Paediatric Oncologist at CHW, said the preliminary results of children treated with ONC201 overseas has already generated a lot of interest and excitement about the potential of the trial.

鈥淭he early results in children treated with ONC201 for DIPG have shown some tumour responses and periods of disease control that we haven鈥檛 seen with other drugs,鈥� McCowage said.

鈥淲e are hopeful that we will end up proving that ONC201 is a step forward and hopefully the other drugs in the trial will be as well.鈥�

For decades researchers have completed multiple clinical trials to find a drug to help change the outcomes for children diagnosed with DIPG, but with limited success.

An adaptive approach

By using an adaptive approach, researchers hope this trial will garner better results through the ability to test the effectiveness of ONC201, in combination with Paxalisib, and other new drugs as they become available.

鈥淭his research is a sophisticated international effort in evaluating new drugs in the treatment of this terrible disease,鈥� McCowage said.

鈥淚t鈥檚 only through adaptive clinical trials like this that we can work out if new drugs are effective without needing to keep opening multiple separate trials and determine if these drugs should be routinely added into treatment for future children.鈥�

While focused on DIPG, the trial will also evaluate the success of these combination therapies in treating Diffuse Midline Gliomas (DMG), cancers located in other parts of the brain. These cancers have recently been identified as having the same genetic make-up in the tumour cells as DIPG.

DIPG, DMG and a spontaneous mutation

Dr Dinisha Govender, local leader at CHW for the trial and Paediatric Neuro-oncologist, said because of this identification they believe the same treatments may work for both tumours.

鈥淪cientists found children with DIPG and DMG were born with normal DNA, but later on a brain cell made a mistake. That mistake was a spontaneous mutation in a particular gene, called H3K27M which was found to be present in DIPG tumours and DMG tumours occurring elsewhere in the brain,鈥� Govender said.

鈥淣ow we know about these identical abnormalities in the tumours, we can expect the same treatments to apply to both groups of patients, offering new hope to families who were previously told there was nothing that could be done.鈥�

The trial will run over the course of several years, with scheduled periods of pause to analyse the data.

The trial is expected to be rolled out at Sydney Children鈥檚 黑料吃瓜群网, Randwick, and other children鈥檚 cancer centres across Australia over the coming months.

鈥淎s a Network, we are excited to collaborate with colleagues internationally on this trial and continue advancing cancer research so one day families don鈥檛 have to hear the words 鈥榯here鈥檚 nothing more we can do鈥�,鈥� McCowage said.

The PNOC-022 trial has been developed by the Pacific paediatric Neuro-Oncology Consortium (PNOC) and is being sponsored in Australia and New Zealand by the Australian and New Zealand Children鈥檚 Haematology/Oncology Group.

Image credit: iStockphoto.com/Panuwat Dangsungnoen