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Children with an inoperable type of brain cancer may benefit from an advanced immunotherapy treatment, according to new research.

Usually occurring in children aged five to seven years old, Diffuse Intrinsic Pontine Glioma (DIPG) is an aggressive type of brain tumour that affects 20 children in Australia each year.

The fast-growing tumour forms in the part of a child鈥檚 brain responsible for vital functions like breathing, swallowing and movement, meaning it is unable to be surgically removed.

Children diagnosed with DIPG are unlikely to survive a year beyond diagnosis and there is currently no treatment for this devastating condition.

Researchers from the (WEHI) have found promise in treating DIPG with an innovative new immunotherapy treatment, called CAR T therapy.

Published in , the research showed CAR T therapy was effective at targeting DIPG tumours.

CAR T cell therapy involves isolating a patient鈥檚 immune cells, engineering them to become 鈥榮uper killer cells鈥� and then re-infusing them into the patient to fight their cancer.

The research, led by Associate Professor Misty Jenkins, Laboratory Head at WEHI and , showed in preclinical models these specifically engineered CAR T cells were able to enter the brain and have an anti-cancer effect by reducing the tumour burden.

鈥淯nlike radiation, which is a blunt instrument that kills cancerous as well as healthy brain tissue, CAR T cell therapy uses a patient鈥檚 own immune cells and engineers them to recognise and kill the tumour,鈥� she said.

鈥淐AR T cells offer a possibility of cure, with no long-term side effects.

鈥淭hese genetically modified white blood cells act as a 鈥榣iving drug鈥�, which means the patient will retain a living memory in their body of the anti-tumour response that may also work to kill the tumour again if it ever returns.

鈥淭he way these cells can completely eliminate the tumours and persist in the body into the future is what is so exciting about CAR T cell therapy, and why immunotherapy is the future of precision medicine.鈥�

The study found DIPG brain tumours can be targeted using CAR T cells that recognise a cancer-specific protein called HER2. Once in the brain, the CAR T cells recognised this protein and signalled the T cells to kill the tumour cells.

Associate Professor Jenkins said the use of CAR T cell therapy had effectively eliminated the DIPG tumours in the study鈥檚 preclinical models.

鈥淭his paper shows that an approach to HER2 does work, and we hope to see this treatment incorporated into current and future clinical trials available to children in Australia,鈥� she said.

鈥淲hile there are no long-term studies yet, this research builds on other work that has shown this treatment to be effective in reducing DIPG tumours and improving the quality of life for these patients, who currently have no treatment options.

鈥淥ur hope is that this treatment will be included in future clinical trials and will eventually be used in combination with other drugs to treat DIPG.鈥�

The research was a promising step forward in treating DIPG, Associate Professor Jenkins said.

鈥淭his lays the groundwork for us to interrogate thousands of potential therapies for anti-cancer immune cells to fight DIPG,鈥� she said.

鈥淥ther studies have demonstrated this treatment approach is safe in children, so we are hopeful it won鈥檛 be long before it is clinically available.鈥�

The research was made possible in part by funding from the Victorian Paediatric Cancer Consortium, Isabella & Marcus Foundation, Robert Connor Dawes Foundation and The Brain Cancer Centre.

Image credit: iStock.com/Realpictures