Researchers make “promising” medulloblastoma breakthrough

2 min read

Scientists at the Brain Tumour Research Centre of Excellence at Queen Mary University of London have made a significant finding which could mean more effective treatment for children with a specific type of brain tumour.

Research focused on medulloblastoma – the most common high-grade brain tumour in under 16s – showed that those with a specific sub-type of the disease could benefit from a new combination of treatment. If further studies are positive, it could see children with this tumour subtype treated with a new combination of drugs which it is hoped would prove less harmful and lead to a better quality of life.

Professor Silvia Marino (pictured), who leads the research team and worked on the study, said: “There are four groups of medulloblastoma, each of them composed of different subtypes but, at the moment, all patients are treated with the same plan of surgical resection followed by chemotherapy and radiotherapy. Our research has shown that there could be important benefits for one of the subtypes of group four, by treatment with a combination of pre-existing drugs.”

Having more targeted therapies according to subtype is needed to improve outcome and quality of life; many patients who survive can live with life-long deficits caused by treatment. High levels of a protein called BMI1 are found in group four medulloblastoma patients and when this is combined with low levels of another protein called CHD7 the prognosis is poor as the tumour regrows. The new research shows that in these cases the tumour is vulnerable to a combination treatment of BMI1 (PTC-209) and MAPK/ERK (PD32590) inhibitors resulting in significantly extended survival. Initial results in the laboratory indicate that the non-cancer cells in the brain are not affected by treatment.

Our Director of Research, Policy and Innovation, Dr Karen Noble, said: “The results of this study are very promising. A kinder, and more effective treatment for patients with medulloblastoma is much needed. Personalised medicine, tailoring treatments to the individual’s tumour, is the way forward, and this research shows that this could be possible for one sub-group of this disease.”

Amongst those welcoming the news is the family of Rayhan Majid, who was just four when he died within months of a medulloblastoma diagnosis.

Rayhan's family marked the fourth anniversary of his death yesterday by launching a Fundraising Group called Remembering Rayhan to raise vital funds in his memory.

His mum Nadia said: “Rayhan endured four operations, one of which left him unable to speak or walk, and six weeks of radiotherapy. He was on his first round of chemotherapy when he died. We were helpless as we watched our boy change from being full of laughter and energy to being unable to communicate or move. The treatment was almost as cruel as the disease itself and this cannot be right so any improvement for patients diagnosed in the future is long overdue.” 

The study is published by the journal Neuro-oncology and can be read online here.


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