DIPG is a devastating and aggressive brain tumour that occurs in children. Researchers now know the genetic mistake that leads to the condition and Dr Jasper de Boer wants to examine thousands of existing drugs to see if they could help put the brakes on this hard-to-treat childhood cancer.
Why this research is vital
Diffuse intrinsic pontine glioma (DIPG) is a devastating and aggressive brain tumour found almost exclusively in children. The disease affects about 40 children a year in the UK and spreads so aggressively that after diagnosis, median survival is less than a year.
The disease is caused by a faulty gene in the child’s DNA – their body’s in-built instruction manual. The fault removes a vital instruction that normally prevents cells from multiplying out of control.
Although the faulty gene has been identified, current treatments are not effective. It is therefore critical to identify new treatments that could help improve survival and quality of life for children affected by DIPG.
In this study, Dr Jasper de Boer and his team will explore using existing drugs to either block the mutation directly or restore the cell’s ‘stop signal’ that normally prevents out-of-control growth. His team will test lots of drugs already approved for other purposes and analyse how they affect DIPG tumour growth.
During the screening process, Dr de Boer and his team will be looking at specific cell processes that are critical for DIPG tumour growth, observing whether certain drugs or combination of drugs disrupt the gene’s cancer-causing ability. These screens will hopefully identify the most promising drug/drugs that could be taken forward into clinical trials.
Impact of this project
The great advantage of redirecting “old” drugs to “new” uses (drug repurposing) is that a great deal is already known about the drugs, including their side effects and dosing requirements. This makes it much cheaper and faster to get them into the clinic than if they were brand new drugs.
If Dr Jasper de Boer’s team successfully identify new drugs that could improve survival and quality of life for children affected by DIPG, Professor Darren Hargrave (co-applicant on the grant) and his team at Great Ormond Street Hospital will take these discoveries forward into clinical trials.
This project is jointly funded by Sparks and charity partner Great Ormond Street Hospital Children’s Charity through their national research funding call.