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Developing an innovative new approach to treat rare and incurable childhood brain tumours

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Diffuse midline gliomas are fast-growing, highly aggressive brain tumours. This type of cancer most often affects children between five to nine years of age and there is currently no effective treatment. Dr Antonis Pouliopoulos at King’s College London is carrying out laboratory research to investigate using focused ultrasound* to deliver cutting-edge gene-based therapies directly into the tumour, improving their effectiveness and reducing the risk of side effects. This targeted approach has the potential to become a powerful and adaptable platform for treating these incurable brain tumours – offering the hope of more effective treatments that can help improve children’s survival and quality of life.

This project is jointly funded by Action Medical Research and LifeArc.

How are children’s lives affected now?

Diffuse midline gliomas (previously called diffuse intrinsic pontine gliomas, or DIPGs) grow between the two halves of the brain. Unfortunately, these tumours can’t be removed with surgery and although treatment with radiotherapy can temporarily slow down their growth, it is not curative.

“Unfortunately, the outlook for children is very poor,” says Dr Pouliopoulos. “Sadly, their lives will be cut short – with an average survival of less than a year1.”

Although the causes of this type of cancer are unknown, scientists have uncovered certain faulty genes often involved in driving tumour growth – opening new opportunities to develop targeted treatments that can switch off their activity. But a critical challenge is that these medicines must be able to cross the blood-brain-barrier (BBB) to reach the tumour.

New treatments are desperately needed that can help improve survival for children with diffuse midline gliomas – especially those that can cross the BBB.

Dr Pouliopoulos

How could this research help?

“Our long-term goal is to develop a safe and effective targeted gene-based therapy for children with these brain tumours,” says Dr Pouliopoulos.

The team is investigating using focused ultrasound* to temporarily open the BBB, allowing cutting-edge gene-based therapies to enter the tumour.

“We will wrap these gene-based therapies into tiny packages and then direct ultrasound precisely on the part of the brain where the cancer is located,” says Dr Pouliopoulos. “As the rest of the body will not be exposed to treatment, this should also reduce the risk of side effects.”

The researchers will carry out a series of laboratory experiments to test the safety and effectiveness of this approach in a brain tumour model.

We hope our results will help pave the way for a future clinical trial in children with diffuse midline gliomas – offering new hope for these desperately ill children and their families.

Dr Pouliopoulos

*Focused ultrasound is a technology that focuses beams of ultrasonic energy (high frequency sound) precisely and accurately on targets deep inside the brain without damaging surrounding tissues.


  1. The Brain Tumour Charity, DIPG prognosis: [website accessed 23 November 2023]

Research table

Project details

Project Leader Dr Antonis N Pouliopoulos, PhD
Location Department of Surgical and Interventional Engineering, King’s College London
Project Team Professor Maria Thanou, PhD
Other Locations School of Cancer and Pharmaceutical Sciences, King’s College London
Grant Awarded
Grant Amount £246,000
Start Date
End Date
Duration 36 months
Grant Code (GN number) GN3017


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