Paed 5-ALA
Research type
Research Study
Full title
A phase II multicentre trial for the use of 5-ALA in paediatric patients with high grade brain tumours
IRAS ID
1006815
Contact name
Madhumita Dandapani
Contact email
Sponsor organisation
University College London
Clinicaltrials.gov Identifier
Research summary
Brain tumours are the most common cause of death from childhood cancer. Complete tumour removal is the main determinant of long-term survival for most brain tumours. Often tumours grow back in the same area despite MRI scans showing complete removal, suggesting that some tumour cells have been left behind. Over two decades ago, a natural dye called 5-ALA (the pink drink) was identified, which made brain tumour cells glow pink under a special blue light used during surgery. Normal brain does not glow pink, thus helping the surgeon accurately identify the tumour edge and remove all areas of tumour. Based on proven benefit in glioblastoma (malignant brain tumour), the pink drink has been approved for use in adults since 2018. But it is not approved for those 18 years and under. We know from anecdotal use and laboratory studies that many different types of children’s brain tumours, including ependymoma and high grade glioma glow pink with 5-ALA.
Our pioneering clinical trial proposal will examine the safety and feasibility of using it in children with aggressive looking (on MRI scan) brain tumours. We will also study its efficacy in helping the surgeon achieve maximal safe tumour removal and discriminating normal brain from tumour. We have built in robust reviews of MRI scans to determine extent of tumour removal and studies in tumour tissue to determine if all glowing areas contain tumour cells. Additionally, we will conduct exploratory studies between the tumour edge and core, made possible due to the pink drink. This work will improve our understanding of what makes tumours invade into normal brain and help develop new treatments targeting these cell pathways, which could improve survival.
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
24/YH/0116
Date of REC Opinion
4 Jul 2024
REC opinion
Further Information Favourable Opinion