Diffuse midline glioma, formerly known as diffuse intrinsic pontine glioma (DIPG)*, is a type of primary, malignant brain tumour occurring in children. With less than 1% of diagnosed patients surviving after 5 years, it is one of the most fatal paediatric brain tumours.
The dismal prognosis of diffuse midline glioma can be attributed to its location within the brain, the brain stem. The brain stem is a difficult to access region of the brain, making it impossible to surgically remove the tumour using current technology.
The current standard of care prescribed to treat diffuse midline glioma is radiation therapy. However, radiation therapy is not a cure and serves only to stabilise the symptoms temporarily, making it essential to develop innovative treatments for this tumour type.
One such innovative treatment is being developed and tested by the research team, led by Professor Okada and Dr Sabine Mueller, at the University of California San Francisco. They are currently leading a phase 1 clinical trial in children with diffuse midline glioma to test a new immunotherapy.
Immunotherapy is a type of treatment that uses the patient's immune system to attack the tumour cells. In order to accomplish this, the immune cells need to distinguish cancerous cells from healthy ones. The research team developed a vaccine that would essentially train the immune cells to recognize a specific abnormal protein that is only found on the surface of cancer cells.
This particular protein, also known as a “neoantigen", has been altered in approximately 70% of the diffuse midline glioma cases tested.
"This may be an ideal case of a tumour neoantigen," said Professor Okada. "Most neoantigens in cancer are unique to individual patients, but this is one of very, very few examples of a shared, common neoantigen that may have the potential to be used in many patients."
This innovative treatment is addressing the acute need to effectively treat this highly aggressive cancer. If the clinical trials are successful, this research could have a significant impact on children diagnosed with diffuse midline glioma, as it could provide them with a new treatment option.
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*Following the 2016 revision to WHO classification, this tumour is now known clinically as “Diffuse Midline Glioma- Pontine Location H3 K27M.