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The Foundation has awarded £350,000 to Great Ormond Street Hospital Children's Charity (GOSH Charity) for researchers at University College London (UCL) and GOSH who hope to develop a new treatment for an aggressive type of brain tumour, diffuse midline glioma (DMG), until recently known as DIPG or Diffuse Intrinsic Pontine Glioma, using a patient’s own immune system to attack the cancer cells.

The team of researchers is being led by Dr Karin Straathof, Associate Professor at the UCL Cancer Institute and Honorary Consultant at GOSH. Dr Straathof will be collaborating with GOSH Charity Clinical Professor in Paediatric Neuro-Oncology and Honorary Consultant at GOSH, Professor Darren Hargrave and the team will use CAR T-cells (patient immune cells engineered to recognise and eradicate cancer cells) in a clinical study for up to 12 patients with DMG at GOSH.

CAR T-cell therapy has previously been used to successfully treat some childhood leukaemias and this team have also used the technology in clinical study for patients with neuroblastoma, a rare cancer that develops in nerve cells. They killed neuroblastoma cells by engineering T-cells to target a specific protein called GD2, and this protein is also found on the surface of DMG cancer cells, making it their next focus.

DMG develops in important parts of the brain that control bodily function. The tumour cells also become mixed with normal brain cells rather than a defined tumour, which means that removing the cancer via surgery is impossible. Although radiation therapy can halt the tumour growth for some time, it unfortunately usually grows back. With results from a study at Stanford University in the USA already showing promise of CAR T-cell therapy in DMG, it is hoped that this clinical trial at GOSH will be a crucial first step in developing effective CAR T-cell treatments for DMG and other high-risk brain tumours that can be devastating to patients and their families.

It is hoped that up to 12 children with DMG at GOSH will be able to enroll and be treated with the therapy as part of the clinical trial that will start in early 2023.