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Treatments for a type of childhood brain tumour called ependymoma have improved very little in 20 years. In half of children, ependymoma comes back after the first treatment, following this outcomes are dismal: only 1 in 4 patients survive for more than 5 years.
Biological Markers of Ependymoma in Childhood and Adolescence (BIOMECA) 2
Dr Timothy Ritzmann
University of Nottingham
Nottingham, NG7 2RD
1 March 2025
36 months
£350,000
Treatments for a type of childhood brain tumour called ependymoma have improved very little in 20 years. In half of children, ependymoma comes back after the first treatment, following this outcomes are dismal: only 1 in 4 patients survive for more than 5 years.
In the last 10 years, we have seen an amazing improvement in our knowledge of the way cancers grow, including a better understanding of tiny components within tumour (or cancer) cells called molecules. These molecules include DNA (the building blocks of life), and proteins. The study of these molecules is known as molecular biology. We want to apply this emerging knowledge to ependymoma.
Understanding the molecular biology of ependymoma will give us information about how a particular tumour might behave, for example do we need to give more intensive treatment for a tumour at higher risk of coming back, or could we give less treatment to reduce side effects whilst still effectively treating the tumour?
To deliver better treatments for ependymoma, we need to make links between molecular biology and patient survival, so that we can recommend which patients should have which treatments, and to design better research led clinical trials in the future.
We are therefore aiming to use advanced scientific techniques for analysing the molecular biology of ependymoma and linking these results with the treatment and survival data collected as part of a large, European study (called a clinical trial) which is currently testing the best ways of treating children and young people with ependymoma. The full name of the clinical trial is SIOP Ependymoma II.
Through linking the molecular biology with the clinical trial, we will:
– Answer questions posed by researchers, in a bigger and better understood group of patients (the clinical trial patients);
– Answer important questions about what makes ependymoma come back again after treatment (relapse);
– Identify tumours at higher risk of relapse and treat them more intensively as well as identifying tumours for which less intense, but curative treatment might be possible.
– Investigate better ways to detect ependymoma by examining the fluid that surrounds the brain and spine.
In the last 9 years nearly one thousand children have been diagnosed with ependymoma across Europe. According to USA data, a similar number can also be expected there over this time. Multiplying these numbers up across the world highlights the scale of the problem we face. The treatment of ependymoma remains a significant challenge and a major reason why brain tumours are the major cause of death in childhood.
The impact of a brain tumour on a child or young person and their family is huge. Besides intensive treatment, long hospital stays and the risk of death, families are also impacted by the financial costs related to treatment, loss of employment, loss of educational opportunities and the impact on the health and wellbeing of the wider family unit.
The goal of this project is to learn through applying our scientific knowledge of ependymoma molecular biology to the day-to-day experiences of the children and young people with this disease. By developing better trials of the future, we can deliver better treatments based on the risks faced by individual patients. Over the longer term, this will contribute to a reduction in the number of children who relapse and die from ependymoma, whilst at the same time reducing the side effects of the treatments we develop.
In addition to developing better treatments and research studies, our project will help us find better ways of detecting ependymomas earlier by analysing fluid from around the brain and spine (called CSF), a key step in reducing the impact of identifying a first tumour or relapse at a late stage, when it is harder to treat.
Finally, by learning how to best apply our molecular tests to a clinical trial, we will develop ourselves as researchers and doctors. We will learn the best ways to bring our new ideas to improve treatment plans for children. As many of our researchers also investigate other types of childhood cancer, this knowledge will also benefit children with other tumours.
The BIOMECA consortium is a group of the key leaders involved in European ependymoma research. Our aim is to take molecular biology findings and use them in designing ependymoma treatment. This proposal describes an ongoing project combining the resources and expertise of our laboratories. Together we have built an unprecedented task force, none of the countries on their own would be able to undertake this work due to the rarity and complexity of the disease.
Our team includes children’s cancer doctors and specialist pathologists (experts in analysing tumours to make diagnoses and guide treatments) alongside research scientists. Our laboratories are based in the UK, Italy, Germany, France and Austria and our leaders are involved in both the BIOMECA2 study and in the SIOP Ependymoma II trial. This puts us in a unique position to lead ependymoma research across Europe and use our findings to develop better treatments for patients.
Our laboratories have a track record, with numerous publications in major science journals. We have contributed to, and continue to develop, milestone discoveries in ependymoma research.
Through the first BIOMECA study we have developed close working relationships and experience of sharing samples, data and analyses to create a genuine pan-European approach to delivering better outcomes. Collectively, we have the infrastructure to complete the scientific tests we have proposed and have demonstrated this through the publication of the first BIOMECA study. The questions that we have proposed in all three sections of our research plan have arisen from earlier work by our researchers, and we therefore have the background knowledge and access to patient samples to address them.
Our proposal will permit us to effectively combine clinical and molecular results to support our longer-term objectives which include future combined efforts with other researchers in North America, ultimately delivering better outcomes and better clinical trials for children and young people with ependymoma into the decades to come.
