Combination therapy to treat medulloblastoma

Friday 13 April 2018

Recent research has demonstrated that combination therapy to target a specific protein, called MYC, in tumour cells could prove to be effective in treating medulloblastoma

Medulloblastoma is the most common high grade brain tumour found in children, accounting for 15-20% of all childhood brain tumours. Research has shown that this tumour can be divided into four subgroups with varying prognoses based on clinical, genetic, and demographic differences.

Previous research has established that increased amounts of a protein called MYC is found in medulloblastoma, particularly in the group 3 subgroup. MYC controls various processes involved in cell growth and division.

Furthermore, elevated levels of MYC are associated with poor prognoses and tumour recurrence, making it is essential to target this protein. However, currently there are no effective drugs to target and block MYC activity.

The research team at Uppsala University in collaboration with various researchers from the United States of America tested two drugs, called JQ1 and Milciclib, which would block the production of this protein and its activity, respectively.

The results of the study, published in the Oncogene journal, demonstrated that JQ1 prevented the production of the MYC protein in tumour samples collected from medulloblastoma patients.

This resulted in a decreased amount of available MYC protein and prevented tumour growth. In addition, they demonstrated that the drug, Milciclib, blocked MYC activity in tumour cells, suppressing tumour growth.

The researchers then tested the combination of the two drugs to block MYC. They found the combination to be extremely effective, relative to using the drugs individually, in suppressing tumour growth.

“We saw that the substances used in the treatment were able to cross the blood brain barrier, which means they can actually make their way in and attack the tumour cells in the brain,” said Fredrik Swartling, principle investigator of the study.

In addition to crossing the blood brain barrier, one of the drugs, Milciclib, is currently in clinical trials to treat a different type of cancer.

If the drug successfully clears the trials, it can be quickly translated to treat medulloblastoma.

While this study does provide an effective combination therapy to treat medulloblastoma, further research is required before this drug combination can be given to people.

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