Projects we fund

Overview

In the last 10 years, immunotherapy has emerged as a major new opportunity to treat many kinds of cancers, particularly those which are difficult to treat with surgery or conventional therapies. Diffuse Intrinsic Pontine glioma (DIPG) is one of those cancers – a virtually incurable childhood tumour that is characterised by a recurring mutation. Radiotherapy is used to treat children with this condition, but it cannot cure them, and surgery isn’t an option because of where the tumours develop, and the way that they spread into surrounding tissues. Unlike chemotherapy or radiotherapy, which target normal cells as well as cancer cells, immunotherapy with engineered T-cells has the capability to target specific cells, and doctors have found a possible way to identify and target DIPG cells whilst leaving non-mutated cells untouched. This raises hopes for their ability to use immunotherapy to treat this condition in the next five years. A major challenge is to find a way to deliver the therapeutic T-cells in a way that allows them to enter, and survive within, the tumour. In this research project, Professor Anderson (pictured above) and Dr Okada will be looking at ways to get the immunotherapy treatment directly into the cancer.

Potential impact

This project stems from discoveries made in Dr Okada’s lab in California. This international collaborative project will help doctors and lab scientists to further understand and overcome potential barriers for development of safe and effective T-cell therapy in children with DIPG. Because of the expertise within the research teams they will aim to develop what they find in the lab into clinical trials in children. The infrastructure at UCSF and GOSH will enable them to deliver first in child clinical trials of gene and cell therapy, so our aim is to identify the optimal design of a phase 1 clinical trial by the end of the project (24 months) and then go on to carry out the trials; either for separate trials or a single trial in both UK and USA. By doing this we aim to develop a new therapy for DIPG. The ambition of the research team is to bring the K27M TCR to phase I/II trial in paediatric high grade glioma within five years.