BIOMarkers of Ependymomas in Children and Adolescents

18 May 2015

Professor Richard Grundy, Children’s Brain Tumour Research Centre, University of Nottingham

Ependymoma is a devastating brain tumour that kills up to 40 per cent of young patients, with little improvement over recent years. BIOMarkers of Ependymomas in Children and Adolescents (BIOMECA) is a unique partnership between leading European ependymoma specialists who are now collecting tumour samples from young patients being treated under the international ependymoma clinical trial. They are aiming to develop new risk categories and work towards the future personalisation of therapy.

Amount of grant: £494,498 | Date of award: March 2015

Overview

Ependymoma is the second most common malignant brain tumour in children and adolescents. Despite advances in brain imaging and technology to improve the precision of surgery and radiotherapy, up to 40 per cent of patients will die from their disease. This outcome has not improved in recent decades.

There is now a global effort towards developing a better understanding of the underlying biological activity of these tumours in order to increase the effectiveness and accuracy of treatment.

Previous research has suggested that certain biological abnormalities may play a role in predicting the behaviour of ependymomas and consequently patient outcome. Because ependymomas are so rare, however, researchers have been hampered by the limited availability of tumour tissue. In addition, many of the patients involved in the analyses have been treated under different regimes, making it difficult to draw conclusions about the impact of any given biological abnormality versus the impact of different treatments.

In order to access sufficient numbers of young patients, international teamwork is required. The BIOMECA project is a unique partnership between the leading European ependymoma specialists. Participants are collecting and analysing tumour samples from children and adolescents receiving standardised treatment under the new international ependymoma clinical trial (known as SIOPE Ependymoma II).

The team will use medical expertise and state-of-the-art technology to identify biological abnormalities present in ependymoma tumour samples, removed from patients during surgery.

Any biological abnormality thought to influence tumour behaviour and patient outcome will be cross-checked against more than 600 childhood ependymomas housed in the key ependymoma laboratories.

All biological findings will be married up with patients’ corresponding medical data in order to assess the clinical influence of any given molecular abnormality.

Since all children will be receiving standardised therapy, as part of the international trial, the impact of abnormalities thought to be important can be verified. This will enable the development of new risk categories for the future personalisation of therapy.

The team also expect to identify new, previously undetected defects. Such defects can potentially be targeted with new or existing, but untried, drugs.

About the research team

At risk of sounding hackneyed, I would characterize the investigation group as a ‘dream team'.
External reviewer
BIOMECA is a collaborative network of childhood ependymoma specialists, including eminent clinicians and scientists from pioneering laboratories in the UK, France, Germany and Italy. The team has a history of successful collaboration, under the SIOP Europe Brain Tumour Consortium.

The UK leader, Professor Richard Grundy, is one of the UK’s foremost experts in childhood ependymoma research and treatment.

Collectively, the BIOMECA team is unparalleled across Europe for its expertise and depth of knowledge in the field, having published several hundred papers and book chapters on the clinical and biological aspects of childhood brain tumours.

The five participating laboratories contain state-of-the-art tumour analysis technology and are all linked to hospitals that represent many of Europe’s Centres of Excellence in the management of childhood brain tumours.

What difference will this project make?

Clearly, putting together such a complex project involving multiple centres in multiple countries carries particular challenges, but this is the only way that progress can be made in rare tumours, and this project would serve well as a model for others contemplating similar ways to approach childhood malignancies.
External reviewer
Treatment for childhood ependymomas has changed little in the last 30 years and survival has not improved. There is a recognised need for a unified approach in ependymoma if we are to advance the cure rate. Global experts agree that an increased understanding of the biology of childhood ependymoma and its influence on patient survival must be integrated into future clinical practice if we are to improve survival.

By bringing together collaborators from across Europe, BIOMECA provides a unique opportunity to identify molecular abnormalities and link them with tumour location, spread, treatment resistance and recurrence. This in turn may allow the team to develop new tumour- and patient- specific risk categories so that in the future therapy can be personalised to ensure best outcome for individual children.

The overriding aims are to improve childhood ependymoma survival and decrease the associated toxicity and side effects observed with current treatment regimes, thereby benefiting all children and adolescents diagnosed with an ependymoma in the future.

Read more: About childhood brain tumoursOther brain tumour research | Patient stories: brain tumours

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