Projects we fund

Overview

Neuroblastoma is one of the most common solid tumours to occur in children, predominantly affecting children under the age of five years. Around 40% of children diagnosed with neuroblastoma have a high-risk form that is essentially incurable using conventional treatments (chemotherapy, radiotherapy, surgery). Immunotherapy approaches, turning the power of the immune system against the tumour, have already shown considerable promise in the treatment of neuroblastoma. The immune system consists of a variety of different cells that protect us against pathogens like bacteria and viruses. Certain immune cells called T cells have the potential to kill tumour cells, but unfortunately tumours have an armoury of mechanisms that enable them to avoid recognition and eradication. Gene therapy has been used to modify T cells, adding proteins called T cell receptors that can overcome some of these tumour defence systems. Early phase clinical studies of this T cell therapy have shown promise but, on their own, these T cell receptors lack the ability to fully activate the T cell. In this project, Dr Gilham and colleagues will generate and test additional proteins, called “chimeric antigen receptors” (“CAR”), that can fully activate T cells thereby enhancing their anti-tumour potential.

What difference will this project make?

The obvious benefit of this work is the potential to improve survival in children with neuroblastoma. If the technology functions optimally, it is strongly possible that the T cells may fight the local tumour and then provide a long-lived population of cells that could potentially fight recurring tumour cells at later times, thus guarding against relapse. Neuroblastoma