A drug that was developed to treat pancreatic cancer has shown promise in treating medulloblastoma which is the most common malignant brain tumour in children. Survival rates vary depending on which one of the four subtypes a patient has, but the worst survival rates, historically at about 40%, are for Group 3 and this research focused on that type. The study was led by Jezabel Rodriguez Blanco, Ph.D., an assistant professor who holds dual appointments at MUSC Hollings Cancer Center and the Darby Children’s Research Institute at MUSC and published in the Journal of Clinical Investigation.

For the study, Rodriguez Blanco focused on the drug triptolide which is extracted from a vine used in traditional Chinese medicine, and its water-soluble prodrug version, Minnelide. A prodrug is an inactive medication that the body converts into an active drug through enzymatic or chemical reactions.

MYC is an oncogene or gene that has the potential to cause cancer. This is present in high levels in Group 3 medulloblastoma than in the other medulloblastoma subgroups. Historically, MYC has not been impacted by drugs. However, a presentation by the research team showed that the more copies of MYC that a tumour has, the better that triptolide works. This led Rodriguez Blanco to get cell lines and animal models and work on propagating them, get the drug and use it.

A report in Science Daily says that determining the mechanism of action has been the most challenging part of the project, she noted, due to the drug’s multiple effects and there could still be additional mechanisms beyond those that Blanco identified.

She said, “It was affecting MYC gene expression by affecting the RNA pol II activity, and then it was affecting how long the protein lasts. So, the fact that it’s working through two different mechanisms on this oncogene may explain why it’s so effective in tumors that have extra copies of MYC.”

Despite the challenges of narrowing down the mechanism of action specific to the cancer, it was quite clear that however it worked, it did work, she said.

The efficacy was 100 times higher in the Group 3 tumours with extra MYC copies than in the Sonic Hedgehog tumours with normal levels of MYC, she said. She found that Minnelide reduced tumour growth and the spread of cancer cells to the thin tissues that cover the brain and spinal cord, called leptomeninges. It also increased the efficacy of the chemotherapy drug cyclophosphamide, which is currently used in treatment.

Blanco is hopeful that, with this new research on Group 3 medulloblastoma, a clinical trial for children with this disease can be launched.

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