Investigating a new targeted treatment for neuroblastoma

Associate Professor Tao Liu

Children’s Cancer Institute Cancer Council NSW Funding: $450,000Funding duration: 2019–2021


Cancer is the most common cause of childhood death from disease. Neuroblastoma is the most common solid tumour in early childhood. It is a particularly complex cancer, with many subtypes that each behave very differently.

The majority of patients with advanced neuroblastoma die of the disease, and survivors suffer from life-long adverse effects due to high-dose chemotherapy which is the current standard treatment. 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.

Targeted treatments for children with this type of neuroblastoma are desperately needed to improve outcomes for these children.

The research

Professor Liu and his team are investigating how particular proteins affect the development and growth of tumours in children with TERT-rearranged neuroblastoma. In this study, the team will examine the role of a protein called CDK7. They also aim to explore how best to target and inhibit the action of this protein to stop cancer cell growth.

The impact

Understanding the processes that lead to cancer is critical to developing targeted treatments that have the best chance of curing disease while minimising side effects. This project will provide vital evidence on the development of TERT-rearranged neuroblastoma and how best to target it, which they hope will lead to much needed clinical trials for this devastating children’s cancer.

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