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Repurposing a multiple sclerosis drug to treat glioblastomas

Researchers at The University of California, San Diego, have found that a drug called teriflunomide has the ability to boost the effectiveness of chemotherapy drugs to treat glioblastomas.

Please note: We’ve had a lot of interest in this research and we would like to highlight two things: 

1) Before this drug could become available to patients with glioblastoma, human clinical trials would be necessary to support its safety and efficacy. 

2) This is not research funded by The Brain Tumour Charity, though we are very interested to see where it leads.

Glioblastoma is the most common primary, high-grade brain tumour occurring in adults. It affects approximately 2,200 people in the United Kingdom each year.

Despite undergoing rigorous treatment regimens, patients with glioblastoma have an extremely poor prognosis due to tumour recurrence. While the combinations of surgery, radiotherapy, and chemotherapy can target the majority of glioblastoma cells, there is a small population of cancer stem cells that remain. These cancer stems cells can replicate indefinitely and eventually cause tumour re-growth.

In order to continue to replicate, cancer stem cells need to keep producing DNA. To produce DNA, these cells must make a substance called pyrimidine, an important component of DNA. However, teriflunomide, a drug already being used to treat multiple sclerosis (MS), blocks the production of this substance. And without pyrimidine, cancer stem cells cannot continue to replicate.

In a study published in Science Translational Medicine, researchers from University of California, San Diego, were able to demonstrate that teriflunomide was able to shrink glioblastomas and improve survival in pre-clinical models.

The research team also found that when teriflunomide was used in combination with another anti-cancer drug called BKM-120, it was able to boost the effectiveness of BKM-120, further improving survival.

These findings are especially exciting because teriflunomide is already being used in people affected by MS. This means that the safety of the drug doesn’t have to be tested again, allowing the researchers to accelerate the findings of this study to clinic trials quicker.

If you have any questions about this, or any of the research projects we fund, please contact us at research@thebraintumourcharity.org