Combination treatments for neuroblastoma

Overview

Neuroblastoma – a nerve tumour – is one of the most common childhood cancers, with around 100 children diagnosed every year in the UK. Most of these children are under the age of five years. Although many young neuroblastoma patients initially respond favourably to therapy, around 50% of patients have a high-risk form of the disease that will relapse and require further, aggressive treatment. These treatments are associated with major long-term toxicities including infertility, kidney and hearing impairment and increased risk of second cancers. There is a pressing need for improved, lower toxicity treatment to reduce relapse, increase survival rates and boost quality of life.

Project description

Dr Andrew Stoker is an expert in developmental neuroscience. He has a background in the study of a type of protein known as phosphatases that are involved in nerve growth and regeneration. He has recently shifted his focus onto phosphatase function in neural cancers and has shown that certain drugs that inhibit production of phosphatases are effective in killing neuroblastoma cells in laboratory conditions. Importantly, these drugs appear not to affect other cells. Dr Stoker has put together an expert team from across the UCL Institute of Child Health to take this work forward. The team will examine the effect of a phosphatase-inhibiting drug known as BMOV in combination with an existing neuroblastoma treatment called 13-cis-retinoic acid (RA). They want to confirm the ability of BMOV to enhance the effect of RA and they also aim to define the genes and pathway changes that are driven by BMOV/RA treatment, identifying further therapeutic targets for the treatment of neuroblastoma.

What difference will this project make?

Children with high-risk neuroblastoma have a poor outlook, with low survival and a high risk of treatment-related complications. This work aims to deliver a better understanding of ways to treat this devastating cancer, focusing in particular on the treatment of residual disease and the reduction of relapse. This project is being funded in collaboration with Great Ormond Street Hospital Children’s Charity, with each charity contributing 50% of the total cost of £138,488.

Neuroblastoma.