The glowing tumour trial for children with aggressive brain tumours

Project title: A Phase II multicentre trial for the use of 5-ALA in paediatric high grade brain tumours

Funded by The Little Princess Trust and administered by CCLG
Lead investigator: Dr Madhumita Dandapani, University of Nottingham
Award: £542,667.32
Awarded July 2022

Brain tumours are the most common cause of death from childhood cancer. Whether or not the whole tumour can be removed with surgery is the main factor that affects a child’s chance of survival. Unfortunately, tumours can often grow back even when MRI scans show that the whole tumour has been removed. This suggests that there are still some tumour cells left behind.

Over two decades ago, a natural dye called 5-ALA was  discovered, which makes brain tumour cells glow pink under a special blue light used during surgery. This shows the difference between tumour cells and healthy brain, helping the surgeon accurately identify the edges of the tumour and remove all tumour cells. The dye was shown to work well in adults for glioblastoma (a type of brain tumour) and has been approved for use since 2018. However, it is not currently approved for use in children. Laboratory studies have shown that that many different types of children’s brain tumours, including ependymoma and high grade glioma, glow pink with 5-ALA.

Dr Madhumita Dandapani and her multicentre team plan to conduct a clinical trial to prove that 5-ALA works for children as well as adults. They will examine the safety and feasibility of using it in children with aggressive brain tumours. The team will also see how much it can help surgeons distinguish between tumour and healthy brain so that they can remove as much tumour as possible. This will be done using MRI scans to see how much tumour has been removed, and the researchers will also look at tumour tissue to make sure that all glowing areas do contain tumour cells. Dr Madhumita Dandapani also plans to investigate the different areas of the tumour, using 5-ALA to accurately investigate the edges. She hopes that this will improve our understanding of what makes tumours invade into normal brain and help develop new treatments targeting these cell pathways.