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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

What is it?

This grant is for a large programme of work into two different childhood brain tumours: choroid plexus carcinoma (CPC) and supratentorial C11orf95-RELA ependymoma (ST-EP-RELA).

CPCs occur within the ventricles (fluid filled spaces) of the brain and can block cerebrospinal fluid, the liquid around the brain and spinal column, from circulating and draining normally.

ST-EP-RELA tumours are found near the top of the head and are distinguished by a particular DNA change – where DNA for “C11orf95” is joined abnormally to DNA for “RELA”. This DNA change is thought to cause the tumour to grow.

To study these tumours Professor Gilbertson will forge ahead with three main areas of work:

  1. Making better models to use in the lab.
  2. Discovering and testing new drugs.
  3. Trialling the best way to deliver new drugs to tumours.

Aim 1: Making better models

This grant builds upon skills this group already has, such as making new lab models of these tumours. The group have previously begun work on new models of CPC and ST-EP-RELA and this grant allows them to have the dedicated time and expertise to see this through to fruition.

This is the first milestone in their grant because they’ll then use the models (together known as a pre-clinical pathway) to help achieve the other milestones.

Aim 2: Discovering new drugs

Professor Gilbertson and colleagues have previously tested over 1.2 million molecules against brain tumours and this grant enables them to take this work even further. These ‘early-stage’ molecules include drugs used for other diseases, natural products and chemically synthesised compounds. The models created by the team in Aim 1 will be used to see if the most promising candidates work against CPC and ST-EP-RELA tumours. This will be the next step in the search for new drug treatments and, for the first time, the treatments will be specific to these rare tumours.

Aim 3: Testing new drugs

A previous study by the team showed that two existing ‘late-stage’ drug candidates showed exceptional promise. This grant will allow them to drive the work forward into the clinic, and the researchers hope to have new clinical trials set up during the grant’s five-year-term.

Aim 4: Drug delivery

The fourth area that this programme of work will focus on is how we can get drugs to the tumour. The blood brain barrier often stops potential therapies from entering the brain, so it’s an important aspect of treatment that urgently needs more attention.

Gilbertson and his colleagues will look at whether drugs can be mixed with special gels and used to fill the cavity left when a tumour is removed through surgery. The gels will be formed by a constant supply of the drug that sits directly next to any tumour cells that might be left after surgery.

Another approach they’ll trial is whether the use of tiny tubes, called catheters, can be used with special pumps to deliver regular doses of the drugs directly into the cerebrospinal fluid around the brain. This means that drugs that would normally be used up in minutes could have access to the tumour for much longer.

Why is it important?

While only about 20 children a year are diagnosed with these tumours, they don’t have a good survival outcome, yet.

Compared to other cancers, childhood brain tumours are very rare, and the tumours Professor Gilbertson is studying are rare, even within this group. This means that until now they haven’t been given the attention we know they so desperately deserve. This research programme will change this.

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.

This grant will fund the collaboration of some of the best minds from the University of Cambridge, The Institute of Cancer Research and the University of Kentucky (USA) with added clinical input from St. Jude Children’s Hospital (USA). The expertise and resources behind this research proposal shows great promise for a future where there’s a precision treatment for CPC and ST-EP-RELA tumours.

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.


  • 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

Research is just one other way your regular gift can make a difference

Research is the only way we will discover kinder, more effective treatments and, ultimately, stamp out brain tumours – for good! However, brain tumours are complex and research in to them takes a great deal of time and money.

Across the UK, over 100,000 families are facing the overwhelming diagnosis of a brain tumour and it is only through the generosity of people like you can we continue to help them.

But, by setting up a regular gift – as little as £2 per month – you can ensure that families no longer face this destructive disease.

Donate today

Professor Richard Gilbertson is a paediatric oncology clinician and a researcher and is currently the Director of the Cambridge Cancer Centre at Cambridge University.