Improving treatment options for children diagnosed with rare tumours of the central nervous system
Fast facts
- Title: Improving treatments for patients with rare embryonal and sarcomatous CNS tumours
- Lead researcher: Dr Katja von Hoff and Prof Barry Pizer
- Where: Aarhus University Hospital, Denmark / Alder Hey, Liverpool, UK
- When: June 2024 – May 2029
- Cost: £1.5 million over 5 years
- Research type: CNS tumours, Rare tumours, REST, Clinical
- Award type: Quest for cures
Embryonal and sarcomatous tumours of the central nervous system are extremely rare, which makes diagnosing and treating them difficult. Embryonal tumours develop from cells that are left over from the early stages of development. Embryonic cells are normally harmless but can sometimes become cancerous. There are many different types of embryonal brain tumour, and work to understand the diversity of these tumour types is ongoing. This unique group of brain tumours need different treatment compared to other brain tumours to ensure the best patient care. However, because there isn’t much clinical data available, current treatment recommendations are based on small studies of historic patient cases.
What is it?
Professor Katja von Hoff, from Denmark and Professor Barry Pizer, UK, are teaming up to improve treatments for young people diagnosed with this very rare group of brain tumours, known as rare embryonal and sarcomatous tumours of the central nervous system, or REST. Since they are very rare, this collaborative project seeks to build an international European network of researchers and healthcare professionals, aiming to work together to shape new clinical trials, and define best practice for the treatment of REST patients. The team will look at the biology of these CNS tumours, how they behave and how they are currently treated. They will also create new tumour models to test different drugs in the lab. Using all this information, they hope to be able to recommend the best treatments for each child diagnosed with one of these tumours. With our funding, this project hopes to drive forward the research and development needed and find better treatments for families facing this challenging diagnosis.
Why it’s important
The current treatments available for children diagnosed with REST involve harsh radiotherapy and chemotherapy drugs that have been developed for different tumour types, so might not be the best therapy for this specific group of tumours. These tumours need more effective and targeted treatments to ensure that children get the best care possible.
Research is just one other way your regular gift can make a difference
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.
In this section
Dr Katja von Hoff
Dr Katja von Hoff is a researcher in Aarhus University Hospital in Denmark. She specialises in brain tumours such as medulloblastoma, ependymoma and paediatric tumours.
Prof Barry Pizer
Professor Barry Pizer is a researcher at Alder Hey, Liverpool who focuses on paediatrics and paediatric oncology. He also has history as a Consultant Paediatric Oncologist.