Combination therapy could be effective in treatment of medulloblastoma

The cancerous tumour known as medulloblastoma is the most common type of brain tumour found in children, accounting for between 15 and 20 per cent of all childhood brain tumours. It typically starts at the base of the skull and spreads to other parts of the brain and spinal cord.

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Research has shown that the tumour can be divided into four types, each with their own prognosis dependent on the genetics and demographics of the patient.

Research has also shown that a type of protein called MYC, which is responsible for cell growth and division, is found in particular types of medulloblastoma. The higher the levels of MYC, the poorer the prognosis and the more likely the tumour is to return.

There is currently no effective treatment to prevent the movement and production of MYC; however, researchers in the US have tested two drugs on tumour samples taken from patients suffering with medulloblastoma and found that the drugs – JQ1 and Milciclib – could potentially block production of the MYC protein.

The results of the study showed that JQ1 prevented the production of the MYC protein and Milciclib blocked MYC protein activity. The researchers also combined the two drugs and discovered that together they proved effective in suppressing tumour growth.

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Milciclib is currently being used in clinical trials to treat a different type of cancer; however, if it successfully passes these trials, it could potentially be used to treat medulloblastoma. Further research – perhaps paid clinical trials through a company such as – would be required before the combination with JQ1 could be prescribed.

Whilst medulloblastoma is more common in children, it can also affect adults, although it only accounts for one per cent of adult malignant brain tumours and is more prominent in adults aged 16 to 40. It typically affects balance, cognitive and fine motor skills, and is more commonly found in males.


Treatment in most cases currently includes surgery to remove as much of the tumour as possible whilst minimising the risk to the mental function of the patient. Most children will also be offered chemotherapy and radiotherapy if appropriate. Most adults will be offered a combination.

Less invasive treatments, such as proton therapy, provide the best chances of successful treatment with the least impact on quality of life.

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