Pancreatic cancer Drug Offers Hope Against Aggressive Medulloblastoma, Study Finds

A recently published study in the Journal of Clinical Investigation finds a rug that was developed to treat pancreatic cancer has now been shown to increase symptom-free survival in preclinical medulloblastoma models – all without showing signs of toxicity. Medulloblastoma is the most common malignant brain tumor in children. Survival rates vary according to which one of the four subtypes a patient has, but the worst survival rates, historically at about 40%, are for Group 3, which this research focused on. 

The research 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.

To evaluate the efficacy of triptolide in targeting medulloblastoma the researchers utilized an established bioinformatic portal.

MYC is an oncogene or gene that has the potential to cause cancer. MYC is dysregulated, or out of control, in about 70% of human cancers, and it shows up in much higher levels in Group 3 medulloblastoma than in the other medulloblastoma subgroups. Despite its well-known role in cancer, this oncogene historically has been considered impossible to target with drugs.

The study concludes that the drug, originally designed for treating pancreatic cancer, has the potential to be an effective treatment option for the most aggressive form of medulloblastoma. This finding opens up new avenues for targeted therapies and provides hope for improving outcomes in patients with this challenging brain cancer. Further research and clinical trials are needed to confirm its efficacy and safety in this new application.