New tools to improve treatment and care in paediatric craniopharyngioma

18 May 2015

Dr Juan Pedro Martinez-Barbera, UCL Institute of Child Health

Paediatric craniopharyngioma is a brain tumour that has high survival but can leave survivors with very poor quality of life. New, targeted treatments are urgently needed but progress is hampered by our poor understanding of the biology of these tumours. This collaborative project brings together teams from the UK and Germany to help profile these tumours and work towards the development of improved treatments.

Amount of grant: £458,728 | Date of award: March 2015

Overview

Childhood craniopharyngioma (ACP) is an aggressive tumour of the pituitary gland that is associated with high survival rates but a very poor quality of life for survivors.

Its difficult location and invasive tendencies make complete surgical removal exceptionally difficult. Remaining tumour tends to regrow and needs further management by surgery or radiotherapy.

The effects of the tumour and the treatments on this critical part of the brain can be drastic. Almost every ACP survivor becomes a chronic patient, suffering severe, lifelong problems that can include severe obesity, diabetes, seizures, visual disturbances, behavioural and learning difficulties.

The behaviour of ACPs can vary enormously; some grow very slowly, and remain localised in a small area of the brain, whereas others grow fast and infiltrate surrounding brain tissues.

It would be helpful to be able to predict how a particular tumour is going to evolve, in order to tailor treatment appropriately, but we do not currently have the means to do this. Current treatments are non-specific and are associated with serious consequences.  

The need to improve on the current treatment strategy for these tumours is hampered by the lack of knowledge of their biology. As a rare tumour, previous studies have been limited by low numbers of tumour samples. To address this, international collaboration is required.

This project brings together world-class scientists and clinicians working on ACP to address fundamental questions about ACP and translate the findings to improve management and therapy of the disease.

The Childhood Craniopharyngioma Research Consortium is comprised of basic scientists and clinicians from six institutions in the UK and Germany.

The team is working on the development of a set of tools that will help predict how a particular tumour may change with time. They will apply state of the art molecular technology to profile a large set of ACP samples before moving on to correlate these profiles with tumour behaviour.

The final step of this work is the identification of potential new drugs that could act on the genes identified during the earlier phases of this work.

About the research team

The lead applicant has assembled a top-notch team comprised of a formidable group of talented clinical, translational and basic science collaborators.
External reviewer
Dr Juan Pedro Martinez-Barbera, of the UCL Institute of Child Health, is one of the world’s leading authorities on the biology of ACP, having developed unique laboratory models for the study of these tumours.

Under the Childhood Craniopharyngioma Research Consortium, he is working with colleagues from centres across the UK and Germany in a collaboration that will help ensure availability of sufficient samples and associated clinical data.

The multi-disciplinary team includes basic scientists as well as endocrinologists, neuropathologists and oncologists, bringing a wealth of expertise and experience in the research and clinical management of ACP in children.

What difference will this project make?

Despite their benign nature, ACPs are devastating tumours that leave young survivors facing lifelong problems and a poor quality of life.

The proposed research will clearly lead to an improved understanding of the molecular pathogenesis of ACP and the strong track record of the investigator and the team ensures that the research will be productive.External reviewerThe nature and location of ACPs means that tumour tissue often cannot be completely removed without affecting brain tissue. Surgeons therefore leave tumour tissue behind, which tends to re-grow after a period of time. Radiotherapy can be used to alleviate tumour regrowth but is often not performed in the developing brains of young children.

Other tools are desperately needed to complement these current treatments and offer a brighter future to affected children.

Through this project, the Childhood Craniopharyngioma Research Consortium aims to provide tools to improve clinical care. The use of specific drugs capable of slowing down tumour growth would allow clinicians to postpone or avoid radiotherapy in young children, avoiding brain damage. New mechanisms of delivering the drugs to the tumour site could also reduce potential side effects of the drugs.

Finally, the research will give an insight into the biology of ACP, and the differences underlying its variable growth and infiltration properties, potentially leading to more personalised management of patients in the future.

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

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