Drug for pancreatic cancer shows promise against brain tumour in kids

New Delhi: A drug that was developed to treat pancreatic cancer has shown promise to treat medulloblastoma — the most common malignant brain tumour in children. 

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 per cent, 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 per cent 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 tumour has, the better that triptolide works. Its efficacy was 100 times higher in the Group 3 tumours with extra MYC copies, Blanco said.

Further, she found that Minnelide could reduce tumour 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.