Projects we fund

Overview

Modern treatments enable doctors to cure more than three quarters of children diagnosed with cancer. Despite this, cancer remains one of the main causes of death in children. Most of these deaths result from a group of high risk cancers. To try to improve survival for high-risk patients, the team at Great Ormond Street Hospital is developing a new Experimental Cancer Therapeutics programme to aid the introduction of new therapies.

Background

Improvements in the survival rates for childhood cancers have been brought about over recent decades as a result of sequential clinical trials, which have sought to optimise the use of chemotherapy, radiotherapy and surgery in the treatment of childhood cancers. The rate of improvement in survival is now slowing and there remains a core of high risk childhood cancers (including high risk leukaemia, brain tumours and metastatic neuroblastoma and sarcoma) in which recent progress has been disappointingly low. There is a desperate need for new therapies to save the lives of children with these high risk cancers.

Targeted therapies for childhood cancer

Advancing knowledge of the underlying biology of different childhood cancers has enabled the development of a new generation of ‘targeted’ therapies. These therapies, which target specific biological abnormalities present in tumour cells, now need to be tested in childhood cancer patients. Advancing knowledge of the underlying biology of different childhood cancers has enabled the development of a new generation of ‘targeted’ therapies. Great Ormond Street Hospital and University College London Hospital, as the UK’s largest paediatric oncology centre are at the forefront of development of new treatments for childhood cancer. Dr Darren Hargrave and Professor Kathy Pritchard-Jones are part of an established team of consultants at GOSH who are leaders in clinical trials in their respective fields. Dr Hargrave is a leading expert in paediatric oncology drug development with a specific interest in neuro-oncology. Professor Pritchard-Jones is an internationally-recognised expert in paediatric oncology with a particular interest in Wilms’ tumour. Together with colleagues, Dr Hargrave and Professor Pritchard-Jones are developing a new Experimental Cancer Therapeutics programme to facilitate the introduction of new targeted treatments for children with cancer. Children with Cancer UK is funding a dedicated Senior Clinical Research Fellow who will play a key role in this programme. The Fellow will drive forward the implementation of early clinical trials of new targeted agents in children with cancer. Early phase clinical trials test new treatments on people for the first time. They help doctors to ascertain the correct doses of new drugs and identify any possible side effects. These trials are carried out in a very small number of patients, usually with advanced cancer and who have had all available standard treatment.

New treatment for children with high-risk neuroblastoma

The initial trial the Fellow will lead on will be an early trial of a new ‘anti-GD2’ antibody in children with high-risk neuroblastoma. Encouraging results have been reported from the USA and there is a high level of demand for this therapy from parent groups in the UK as it offers hope to children who have been failed by existing therapies. This therapy can have significant side-effects and the trial will explore alternative dose and schedules, to optimise its beneficial effects whilst minimising distress to the child. Neuroblastoma Treatments