Development of novel chemical therapies against childhood craniopharyngioma

01 June 2012

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

Childhood craniopharyngioma is a rare but devastating brain tumour. Whilst it can usually be ‘successfully’ treated in that most children do not die, these young patients are left with a range of problems that result in a very poor quality of life. The team aims to identify new drugs that could offer cure whilst reducing or avoiding the need for damaging brain surgery and radiotherapy.

Amount of grant: £39,576*  |  Date of award: June 2012

The team
Dr Juan Pedro Martinez-Barbera, Prof Mehul Dattani & Dr Thomas Jacques, UCL Institute of Child Health; Dr Helen Spoudeas, University College London Hospitals; Mr Dominic Thompson & Dr Darren Hargrave, Great Ormond Street Hospital.

Background

Childhood craniopharyngioma (adamantinomatous craniopharyngioma, ACP) is a rare but devastating childhood brain tumour. It accounts for 5 to 10% of all childhood brain tumours, affecting around 20 children a year in the UK, most commonly children between the ages of 5 and 10 years.  

ACP usually develops near the pituitary gland, at the base of the brain. It is a benign tumour, meaning that it cannot spread to other parts of the body, but it behaves aggressively and can invade surrounding parts of the brain if not treated. It affects the function of the pituitary gland, causing hormonal imbalances, and it also damages the optic nerve, causing vision problems.

Treatment involves surgery to remove as much of the tumour as possible, followed by radiotherapy and/or chemotherapy to try to ensure that the tumour has been completely removed and prevent recurrence.

Following such treatment, most children with ACP will survive. However the problems caused by the tumour are often permanent and may get worse, rather than better, after treatment. Long-term consequences include severe obesity, diabetes, learning difficulties, visual impairment and hormone deficiencies.

Project description

Great Ormond Street Hospital is the leading treatment centre for ACP in the UK, with 10-12 new referrals every year, around half of the UK cases.

Dr Juan Pedro Martinez-Barbera, a leading expert in pituitary development, has put together an impressive team that includes clinical colleagues specialising in endocrinology, neurosurgery, histopathology and paediatric oncology. This collaboration between scientists and clinical specialists enhances the likelihood of the project achieving success.

The team will use a laboratory model of childhood ACP to assess the effectiveness of seven different drugs in slowing or stopping tumour growth. These seven drugs are at various different stages of testing in human trials for treatment of other tumours which share key characteristics with ACP.

If successful, the team will demonstrate proof-of-principle that a particular drug is efficient in the treatment of ACP and pave the way for a clinical trial in affected children.

What difference will this project make?

Although most children diagnosed with ACP survive, they are left with a poor quality of life as a result of the effects of the tumour and the treatment that is used to get rid of it.

New approaches to treatment are desperately needed. Dr Martinez-Barbera and colleagues are working towards the development of new drug therapies that they hope may be used in place of the devastating surgical approaches currently used. If their work is successful, they expect to proceed to clinical trial within one to two years.

Ultimately, this work offers the hope of an improved quality of life for the survivors of childhood ACP.

There is no doubt that any improvement in the treatment of ACP would be significant. The current surgical treatments are almost as bad as the diseases, and effective pharmacological interventions would be a tremendous improvement over the current treatment protocols.

* this project is being funded in collaboration with Great Ormond Street Hospital Children’s Charity, with each charity contributing 50% of the total cost of £79,152.

Read more: About childhood brain tumours | Other brain tumour research | Brain tumour initiative

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