No brain tumour ignored

Fast facts

Official title: Curing the underserved – developing transformative therapies for rare childhood brain tumours
Lead researcher: Prof Richard Gilbertson
Where: University of Cambridge, UK
When: April 2019 – April 2025
Cost: £1.5 million over 5 years
Research type: Paediatric, Academic, Translational, Ependymoma and Choroid Plexus Carcinoma (High grade)
Grant round: Quest for Cures

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Choroid plexus carcinoma (CPC) and supratentorial C11orf95-RELA ependymoma (ST-EP-RELA) are aggressive paediatric brain tumours that can have a poor prognosis. No new treatments have been developed in the last 30 years, and even ‘standard’ therapy remains largely ineffective. New therapies have not yet been discovered due to the rarity of these two tumour types

What is it?

As current standard of care has not worked for these tumour types, this project will focus on testing new models and finding new treatments for both CPC and ST-EP-RELA. Professor Gilbertson and his team have previously tested over 1.2 million molecules against brain tumours to find promising molecules that can be used to create new drug treatments for CPC and ST-EP-RELA. They will continue doing so to find new drug treatments for these rare tumours.

Additionally, these researchers have already found that two drugs – which have entered the late stage of development where clinical trial testing is the next step before entering the market – have shown great promise to treat these tumours. The team will now develop a plan to bring these drugs to the clinic, with the goal of setting up clinical trials. To ensure that these drugs effectively target the tumour, the researchers will experiment with mixing these drugs with gels that will fill the space left by the tumour after surgery. They will also see if they can make use of tiny tubes in the brain, called catheters, that can be repurposed to transport the drugs throughout the brain. They will carry out these experiments and testing on preclinical models for CPC and ST-EP-RELA that have been newly developed in-house by the team.

Why is it important?

This grant will allow Professor Gilbertson’s team to concentrate their efforts on making new discoveries to help children with these tumours. The way the lab’s set up and the collaborations already in place will mean that any new discoveries are brought to the clinic as quickly as possible.

Who will it help?

Currently children with CPC and ST-EP-RELA are given the same treatment as children with other types of brain tumour. This is because there are no precision treatments for these rare tumours.

This research programme will give children affected by these diagnoses, and their families, hope for more effective treatments and, ultimately, increased survival.

Milestones

• Making new models to study these tumours in the lab.
• Test the best early-stage drug molecules in the newly developed pre-clinical pathway to find the best candidates for future clinical trials.
• Take previously discovered late-stage compounds through appropriate testing to prove that they should (or shouldn’t) be used in clinical trials in the near future.
• Investigate the usefulness of gels and catheters as drug delivery systems for CPC and ST-EP-RELA tumours.

If you have any questions about this, or any of our other research projects, please contact us on research@thebraintumourcharity.org