Researchers at the Brain Tumour Research Centre of Excellence at Queen Mary University of London have, for the first time, identified the earliest steps in the development of medulloblastoma – the most common form of brain cancer in children.
The research, which could transform the way the disease is studied and treated, has been published in the journal Genes & Development.
Medulloblastoma can start to develop before a baby is even born, although we do not know why tumours take many years to develop. There are around 50 new diagnoses in children every year in the UK. Little is known about the biological mechanisms that lead to tumour initiation and current treatment options involve surgery along with harsh radiotherapy and chemotherapy, which are not always effective – particularly in the most aggressive forms of the disease.
Sadly, only 60% children survive a diagnosis of medulloblastoma beyond five years. When these treatments do succeed, they often lead to long-term side effects such as cognitive problems, as well as coordination, motor skill issues and endocrine dysregulation.
The scientific team, led by Professor Silvia Marino, has created 3D ‘mini brains’ in the laboratory – accurately recreating the environment in the developing embryonic brain where medulloblastoma originates.
The mini brain technique also provides a drug testing platform that replicates the human brain and is potentially better than existing animal models. By combining patient samples with mini brains grown in the lab, they can test drugs whilst monitoring toxicity to identify treatment options that specifically target cancer cells but spare healthy brain tissue, limiting the long-term consequences of therapy on children.
Prof Marino, Director of the Brain Tumour Research Centre of Excellence at Queen Mary University of London, said: “To develop effective but less toxic treatments for aggressive forms of medulloblastoma, we need to understand how these tumours start and grow within the developing embryonic brain. Our work not only advances our understanding of the disease but provides a platform for testing new therapies for this tumour.”
Welcoming the news was Helen Forbes-Low from Member Charity, The William Low Trust, who funded Thomas Willott, the PhD student who undertook this work. She said: “Our son, William, was just five when he was diagnosed with medulloblastoma and sadly didn’t see his 18th birthday. Since then, we have been committed to funding research to help make sure no one else has to go through what we did. We are proud of the role we have played in ensuring research like this at Queen Mary gets off the ground and can hopefully one day make a difference to children and families facing this devastating diagnosis.”
Also welcoming the news was Christina Dolding, who has just completed a 1,200-mile walk from Land’s End to John O’Groats in memory of Ollie Gardiner, who tragically died from medulloblastoma aged just 13. Christina has raised more than £11,000 for Brain Tumour Research through her epic challenge.
“Hearing of Ollie’s diagnosis was utterly heartbreaking. After Ollie’s funeral, his father told me he was worried Ollie might be forgotten. I promised myself then that if I ever had the chance to shine a light on his story again, I would. From the very start, I wanted to raise funds for present and future Ollies and to see the impact that fundraising efforts can have in terms of breakthroughs in the laboratory is a really special way to end this challenge,” Christina said.
Christina on her amazing challenge (left) and Ollie in his cricket gear (right)
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