Funded by The Little Princess Trust and administered by CCLG
Lead investigator: Dr Jake Mann, University of Birmingham
Award: £39,490.54
Awarded February 2024
Hepatocellular carcinoma (HCC) is a devastating childhood cancer that develops in the liver. Very little is known about how it develops in children or why it is not killed by the immune system. HCC has few treatment options - less than half of patients survive, and those that do usually need a liver transplant.
It’s also not clear what DNA mutations cause the tumours to develop. Doctors know that some genetic conditions, like bile salt export pump (BSEP) deficiency, make children are at higher risk of HCC. However, they don’t understand why.
The three key research goals for HCC are to:
- Uncover what makes children with certain genetic conditions susceptible to this cancer.
- Find which genetic errors make HCC develop.
- Understand how it evades the immune system.
Dr Jake Mann is leading this project from the University of Birmingham. He aims to address these key goals by using cutting-edge gene sequencing. He will use tumour samples from patients with BSEP deficiency who have also had a liver transplant for HCC.
Dr Mann’s team want to prove they can get high quality genetic information from these tumour samples. This will allow them to look at the genetic code inside cancer cells to see which DNA mutations are driving the tumour. They will also look at how BSEP deficiency makes a child more at risk of developing HCC. Finally, the team will look at which immune cells are in the tumours, to see whether medicines could help the immune system to fight the cancer.
Overall, Dr Mann hopes to improve our understanding of liver tumours in children and to find new targets for treatment. This could reduce the number of liver transplants that are required, and significantly improve the lives of children with HCC.