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Craniopharyngiomas: developing new drug treatments for children with these devastating tumours

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Each year in the UK, around 30 children are diagnosed with a tumour called a craniopharyngioma.1 These tumours grow near the pituitary gland in the head, very close to the brain. Children normally undergo surgery, sometimes followed by radiotherapy. Both the tumour and its treatment can leave children with serious, long-term problems that badly affect quality of life, such as hormone problems, obesity, seizures and blindness. Unfortunately, current treatment is not always effective and tumours often regrow. Dr Carles Gaston-Massuet, of Barts and The London School of Medicine, aims to identify new drug treatments for children with these devastating tumours, in the hope of improving their quality of life.


How are children’s lives affected now?

When a child develops a craniopharyngioma, both the tumour and its treatment can cause problems. Although these tumours do not spread to other parts of the body, they can disrupt important parts of the brain that control essential body functions. Children can experience symptoms such as headaches, mood changes, blurred vision, slowed growth and early or delayed puberty.

Children are normally between five and 14 years old when their tumour is first diagnosed. They are typically offered surgery. Surgeons try to remove as much of the tumour as possible without damaging vital areas of the brain. Sometimes the entire tumour can be removed. If this isn’t possible and part of the tumour has to be left behind, children then need radiotherapy. Both surgery and radiotherapy can have secondary effects that reduce quality of life.

After treatment, children can be left with serious, long-term problems, including learning difficulties, obesity, seizures, blindness and hormone problems that require lifelong hormone replacement therapy. Tumours can also grow back. Better treatments are urgently needed.


How could this research help?

The ultimate aim of this research is to identify new drug treatments for children with craniopharyngiomas. So far, no medications have been approved for the treatment of these tumours. Recently though, this team has discovered that drugs called non-steroidal anti-inflammatory drugs, or NSAIDs, might have beneficial effects.

NSAIDs are a well-established group of medicines, which are already used to treat conditions such as arthritis and inflammation. The research team is investigating whether NSAIDs can slow down the growth of these tumours. They are also studying how they do this.

This research is in its early stages and is laboratory based, but the researchers hope that their work will eventually lead to clinical trials of a new drug treatment in children with craniopharyngiomas.  A new drug treatment that spares children from some of the devastating long-term health problems that they currently experience, is badly needed.





1. The Royal Marsden. Craniopharyngiomas. http://www.royalmarsden.nhs.uk/cancer-information/children/pages/craniop... Website accessed 14 September 2014.


Project Leader Dr Carles Gaston-Massuet PhD
Project Team Professor Tim Warner PhDProfessor Mehul T Dattani MBBS DCH FRCPCH FRCP MD
Project Location Centre For Endocrinology & Centre Translational Medicine William Harvey Research Institute, Barts and The London, Queen Mary's School of Medicine and Dentistry
Project Location Other Paediatric Endocrinology, Institute of Child Health, University College London
Project duration 3 years
Date awarded 25 July 2014
Project start date 1 March 2015
Project end date 28 February 2018
Grant amount £190,617
Grant code GN2272


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