Study finds pancreatic cancer drug effective against pediatric brain tumor, medulloblastoma
For the study, the researchers focused on the drug triptolide, which is extracted from a vine used in traditional Chinese medicine, and its water-soluble prodrug version, minelide. It was developed to treat pancreatic cancer, however, it has now shown promising results in treating medulloblastoma which is the most common malignant brain tumor in children. Read on to know more about the study.
Pancreatic cancer drug effective against pediatric brain tumors
A drug developed to treat pancreatic cancer has shown promising results in treating medulloblastoma, the most common malignant disease. brain Tumour in children. Survival rates depend on which of the four subtypes a patient has, but the worst survival rates, historically around 40%, are for group 3 and this research focuses on that type. The study was led by Jezabel Rodriguez Blanco, Ph.D., an assistant professor who holds dual appointments at the MUSC Hollings Cancer Center and the Darby Children’s Research Institute at MUSC, and was 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, minelide. The prodrug is an inactive drug that the body converts into the active drug through enzymatic or chemical reactions.
MYC is an oncogene, or gene that has the potential to cause cancer. It is present at higher levels in group 3 medulloblastoma than in other subgroups of medulloblastoma. Historically, drugs have had no effect on MYC. However, a presentation by the research team showed that the more copies of MYC a tumor has, the better triptolide works. This inspired Rodriguez Blanco to obtain and propagate cell lines and animal models, obtain the drug, and use it.
Determining the drug’s mechanism of action has been the most challenging part of the project, because the drug has multiple effects, and there may be other mechanisms besides the ones identified by Blanco, says a report published in Science Daily.
He added, “It was affecting MYC gene expression by affecting RNA Pol II activity, and then it was affecting how long the protein lived. So, the fact that it was working through two different mechanisms on this oncogene may explain why it is so effective in tumors that have extra copies of MYC.”
Despite the challenges of narrowing down the specific mechanism for cancer, it’s pretty clear that no matter how it works, it works, he said.
They said efficacy was 100 times higher in group 3 tumors with extra MYC copies compared with sonic hedgehog tumors with normal levels of MYC. They found that Minelide reduced tumor growth and the spread of cancer cells into the thin tissue covering the brain and spinal cord, called the leptomeninges. It also increased the efficacy of the chemotherapy drug cyclophosphamide, which is currently used in treatment.
Blanco hopes that with this new research on Group 3 medulloblastoma, clinical trials can be initiated for children with this disease.
(with inputs from agencies)
Get the latest news on Times Now as well as breaking news and top headlines from across health and the world.
