New Delhi: A drug originally developed for pancreatic cancer has demonstrated promising results in treating medulloblastoma, which is the most common malignant brain tumor found in children, offering new hope for treatment options.
In the study, published in the Journal of Clinical Investigation, the drug triptolide, which is extracted from a vine used in traditional Chinese medicine, and its water-soluble prodrug version Minnelide -- was found to increase symptom-free survival in preclinical medulloblastoma models -- all without showing signs of toxicity. A prodrug is an inactive medication that the body converts into an active drug through enzymatic or chemical reactions.
Survival rates of medulloblastoma vary according to which one of the four subtypes a patient has, but the worst survival rates, historically at about 40 percent, are for Group 3. And the research focused on Group 3.
The research by Jezabel Rodriguez Blanco, Assistant Professor at the Medical University of South Carolina, focused on the drug triptolide and Minnelide and discovered triptolide's ability to target MYC -- an oncogene, or gene that has the potential to cause cancer.
While MYC is dysregulated, or out of control, in about 70 percent of human cancers, it shows up in much higher levels in Group 3 medulloblastoma.
The study showed that the more copies of MYC that a tumor has, the better that triptolide works. Its efficacy was 100 times higher in the Group 3 tumors with extra MYC copies, Blanco said.
Further, she found that Minnelide could reduce tumor growth and the spread of cancer cells to the thin tissues that cover the brain and spinal cord, called leptomeninges.
In addition, the prodrug also increased the efficacy of the chemotherapy drug cyclophosphamide -- currently used in treatment.
Minnelide is currently being tested in clinical trials of adults with different types of cancer, including pancreatic cancer.