Ground-breaking research discovers secrets behind hard-to-treat childhood brain tumours

  • Researchers at the University of Edinburgh have made a major breakthrough in understanding how childhood glioblastoma brain tumours develop;
  • Research funded by Children with Cancer UK has found that the specific location of the cell that gives rise to the tumour profoundly influences how the tumour will subsequently develop and the level of its severity;
  • It is hoped that mapping the origins of these cancers and the earliest events driving their growth will lead to the development of new treatments;
  • The discovery coincides with the start of Brain Tumour Awareness Month.
The research funded by Children with Cancer UK – the UK’s leading charity dedicated to research into childhood cancer – has found that the specific brain region of the originating cell that gives rise to the tumour has a profound impact on tumour formation. Mapping the cancer’s origins helps shed light on the different types of childhood glioblastoma and identifies new vulnerabilities that may be explored in the future search for treatments. These findings have been released at the start of Brain Tumour Awareness Month, a yearly event which aims to raise awareness of the devastating impact of brain tumours and to raise funds and help to support people suffering from this disease. Glioblastoma is the most aggressive form of brain cancer. While rare in children, making up just 8%(1) of childhood brain and spinal cord tumours in the UK, it can be particularly devastating, as their location makes them hard to treat. Sadly, just a quarter of glioblastoma patients survive more than one year. Tumours can arise in different parts of the brain and can appear very similar, however they have discreet mutations depending on their location. This has so far been a puzzle for scientists, who have been unable to explain why different mutations and cancers arise in different regions of the brain. The study, led by Professor Steven Pollard, set out to understand the origins of these aggressive childhood brain tumours and why certain mutations give rise to different forms of the disease. Working in the laboratory culture dish they were able to test the responses of normal brain stem cells obtained from forebrain or hindbrain to different mutations and see how they responded. It gave Professor Pollard and his team the opportunity to explore the very earliest stages of brain tumour formation, which is otherwise impossible using patient derived tumour cells. They found that the location of the cells – termed their regional identity – impacts how they will respond to different mutations. A powerful mutation can be dangerous in one region of the brain, however, similar cells from another region of the brain can detect that it is dangerous and have a ‘safety switch’ stopping any further development. For example, a mutation that arises in the forebrain can cause tumours in the region, while the same mutation will not have the same impact on cells from the hindbrain. These results can help scientists understand why different mutations and cancers arise in different anatomical regions of the brain, with varying degrees of aggressiveness and help us understand the origins of childhood brain cancer and how different tumours work. Ultimately, this new insight may help future development of more effective treatments for the disease. Professor Steven Pollard, research lead and CRUK Senior Research Fellow at the University of Edinburgh commented:
For a long time, how these childhood forms of glioblastoma develop has been a bit of a puzzle. We have been unsure as to why certain gene mutations appear in certain regions and with different levels of aggressiveness. Our research answers this question and has revealed the importance of particular class of protein termed transcription factors, which control the cell identity and have such a dramatic impact on how these mutations operate. Our work adds to the growing evidence that paediatric cancer cells have become ‘stuck’ in their immature stem cell-like state. We are also excited by the possibilities the new cellular models we have built can be used in development and testing of new treatments.
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One child who was diagnosed with glioblastoma is Dominic Ballard, 11, who was diagnosed at just four months old. For two years Dominic underwent surgery and chemotherapy to reduce the tumour. Happily, his tumour was treated successfully and he is now monitored annually, however, he suffers from a number of side effects as a result of the treatment. Dominic’s mental health, hearing and sight have all been impacted by the initial treatment for his tumour. Researchers hope that this latest development in understanding glioblastoma will lead to the discovery of less invasive and kinder treatments, resulting in fewer debilitating side effects. Commenting on the findings, Dominic’s mum Ann said:
Dominic has undergone so much since his diagnosis in 2010, but he has turned into a lovely boy who is an inspiration to us all. He does struggle as a result of the side effects from his treatment, so we are pleased to hear that scientists are getting closer to making better treatments that could reduce the likelihood of children suffering from long term side effects.
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Dr Nick Goulden from Children with Cancer UK, the main funder of the research said:
In Brain Tumour Awareness Month we aim to highlight the ongoing major challenges faced by children, adolescents and young adults who develop brain cancer. Brain tumours in this age group arise in developing brains. Unfortunately, treatments such as surgery and chemotherapy are not cancer specific and can cause damage to normal cells leading to significant long term side effects such as those experienced by Dominic.  This Children with Cancer UK funded research project has employed state of the art technology to advance our knowledge of how and why brain tumours develop in different areas of the growing brain. We now hope that Professor Pollard’s findings will be harnessed to develop new cancer specific treatments that will be both more effective and cause fewer side effects. This is another excellent example of how charitably funded research can directly impact on survival and quality of survival for patients.


(1)According to figures from the Royal Marsden NHS Foundation Trust –
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Notes to the editor About Children with Cancer UK Children with Cancer UK is the leading national charity dedicated to research into childhood cancer. We fund research into the causes and treatment of childhood cancers and provide funding for support for families affected by childhood cancer. We have accelerated breakthroughs to improve childhood cancer survival rates and find kinder, more effective treatments with fewer toxic side effects. This ground-breaking research, which would otherwise go unfunded, saves the lives of children with cancer. About childhood cancer and Children with Cancer UK’s impact Every day in the UK, 12 children and young people are diagnosed with cancer. Fifty years ago, only 30% of children with leukaemia survived, and for most other forms of childhood cancer survival rates were even lower. Today, thanks to our supporters and the dedication of visionary researchers like those we fund, more than 80% of young patients can be successfully treated. More vital research is needed though as there are still a number of cancers affecting children and young people with low survival rates and life-limiting side effects. Cancer remains the single largest cause of death from disease in children and young people in the UK. About the research The research was funded by a grant from Children with Cancer UK and undertaken by The University of Edinburgh, starting in April 2016 and lasting for four years. The funding amount was £357,589. The research was led by Professor Steven Pollard, a CRUK Senior Research Fellow at the University of Edinburgh and his world-class laboratory at the Centre for Regenerative Medicine. About the Centre for Regenerative Medicine at the University of Edinburgh’s Institute for Regeneration and Repair. The Centre for Regenerative Medicine (CRM) is a research centre based at the University of Edinburgh’s Institute for Regeneration and Repair. Scientists and clinicians study stem cells, disease and tissue repair to advance human health. By better understanding how stem cells are controlled and how diseases develop in a lab environment, they hope to find new ways to treat patients. Research at the Centre is aimed at developing new treatments for major diseases including cancer, heart disease, liver failure, and degenerative diseases such as multiple sclerosis and Parkinson’s. The Centre is located in the Edinburgh BioQuarter, a site shared by the Royal Infirmary Hospital and the University’s Clinical Research facilities. This makes it ideally placed to translate basic science into clinical therapies. Brain Tumour Awareness Month Brain Tumour Awareness Month, taking place this March, is a month to raise awareness of the devastating impact of brain tumours and to raise funds and help to support people suffering from this disease. Brain tumours claim more lives than any other childhood cancer. For those that survive, the outlook is discouraging, with up to two thirds of survivors left with moderate to severe disabilities. This Brain Tumour Awareness Month Children with Cancer UK wants to raise awareness of the devastating impact of brain cancer on children and highlight the research we’re funding to improve survival rates and develop less harmful treatments. For further details please visit the following link.
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