Investigating a new therapy for a problematic childhood cancer
Background
Neuroblastoma is the most common type of cancer in infants, with most new cases occurring in children under the age of two. In those affected, solid tumours form as a result of abnormal growth of immature nerve cells called neuroblasts. Normally, these cells grow into functioning nerve cells. But in neuroblastoma, they become cancer cells instead. Neuroblastoma is a very complex cancer and there are many types that behave very differently.
One subtype with a particularly poor prognosis is neuroblastoma caused by rearrangements in a gene called TERT that result in uncontrolled cancer growth. Only 20 per cent of children with this type of neuroblastoma survive.
The research
This project aims to work out how a particular protein called BRD4 leads to the development and growth of tumours in children with TERT-rearranged neuroblastoma. It also aims to explore how best to target and inhibit the action of this protein to stop cancer cell growth.
The impact
With a new understanding of the processes that lead to this type of neuroblastoma, and how best to target it, the project will provide vital evidence that could lead to much needed clinical trials and potentially a cure for this devastating children’s cancer.