Official title: Identifying directed therapies for adamantinomatous craniopharyngioma (ACP) using advanced biological techniques and genetically engineered pre-clinical models
Lead researcher: Dr Todd Hankinson
Where: University of Colorado, USA
When: September 2018 - August 2023
Cost: £1.58 million over five years
Research type: Paediatric, Craniopharyngioma (Low Grade), Academic
Adamantinomatous craniopharyngiomas (ACPs) are a devastating type of brain tumour occurring in children. These tumours are heterogeneous, which means they are made up of different types of cells. The aim of the research programme is to understand the behaviour of these different types of cells and identify targets for treatment.
Previous research has shown that the different types of cells that make up ACPs communicate with each other using chemical messengers called cytokines. Cytokines are molecules released from cells that allow them to ‘talk’ to each other and promote survival and growth.
The research team will use cutting edge laboratory techniques to determine which cytokines are involved and if their activity can be blocked using drugs. They will then test these drugs in pre-clinical models to determine which ones are the most effective at treating the tumour.
The two most promising therapies will be tested in five children with newly diagnosed ACP. The drugs will be given before a biopsy or surgical removal of the tumour to find out if the drugs are able to cross the blood–brain barrier.
With this information, the team will be able to start a clinical trial for new treatments for children with ACP.
Doing research on uncommon tumours is very difficult to do and having multi-national and multi-institutional support is very hard to find. The support provided by The Charity is critical to accelerate progress.
ACPs are associated with the worst quality of life due to the location of the tumour – near the pituitary gland, optic nerve and hypothalamus. Almost all children with ACP suffer from a life-altering side effect or injury from the tumour itself or its treatment. These 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, highlighting the need to better understand the biology of these tumours and develop improved therapies.
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.
This research will help develop targeted treatments that will improve the quality of life of children with ACP and their families.
Research into ACP biology has been overlooked and nearly no novel therapies have been introduced for over 50 years, resulting in patients having the lowest quality of life. This research has massive potential for patient benefit.
The research team have finished collecting the tumour samples to help them identify all the different types of cells in ACPs.
In the upcoming year, the research team will identify the different types of cytokines (chemical messengers) that are used by tumour cells.
Research is the only way we will discover kinder, more effective treatments and, ultimately, stamp out brain tumours – for good! However, brain tumours are complex and research in to them takes a great deal of time and money.
Across the UK, over 100,000 families are facing the overwhelming diagnosis of a brain tumour and it is only through the generosity of people like you can we continue to help them.
But, by setting up a regular gift – as little as £2 per month - you can ensure that families no longer face this destructive disease.