Identifying directed therapies for Adamantinomatous Craniopharyngioma (ACP)

Adamantinomatous Craniopharyngioma (ACP) is a devastating brain tumour occurring in children. Due to the location of the tumour – near the pituitary gland, optic nerve and hypothalamus – it is associated with the worst quality of life scores of any childhood brain tumour. Almost all children with ACP suffer from a life-altering side effect or injury from either the tumour itself or treatment. Side effects and injuries include hormone imbalances, blindness, and morbid obesity. The current standard of treatment includes surgical removal of the tumour followed by radiation therapy – there are currently no targeted therapies to treat ACP. It is vital to better understand the biology of these tumours in order to develop better therapies that can improve the quality of life for these patients.

Revolutionising treatment for ACP

ACP tumours are heterogeneous, which means they are made up of different types of cells. The aim of the research programme, led by Dr Todd Hankinson, is to understand the behaviour of the different types of cells and identify targets for treatment.

Previous research has shown that the different cells communicate with each other using cytokines (chemical messenger signals released from cells that allow them to 'talk' to each other) to promote tumour survival and growth. The research team will use various cutting edge laboratory techniques to determine which cytokines are involved and if they can be blocked using drugs. The researchers will then test these drugs in mice to determine which ones are the most effective at treating the tumour.

The two most promising therapies will then be tested in five children with newly diagnosed ACP. The treatments will be given prior to a biopsy or surgical removal of the tumour to evaluate if the drugs are able to cross the blood–brain barrier and reach the tumour. With this information, the team will begin a clinical trial of a novel treatment for children with ACP.

Collaborating to find a cure

This research programme is bringing together researchers from University of Colorado Denver and Institute of Child Health, University College London, with expertise in basic and clinical research. The researchers will be part of a consortium called Advancing Treatment for Paediatric Craniopharyngoioma that is dedicated to identifying and developing targeted treatments that will improve the quality of life of children with ACP.

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