Mebendazole + Chemotherapy for Brain Tumor

This trial tests whether the drug mebendazole can enhance the effectiveness of chemotherapy for brain tumors in children and young adults. It targets two types of brain tumors: low-grade gliomas, which grow slowly, and more aggressive high-grade gliomas. Participants must have a confirmed diagnosis of one of these tumors, and for those with low-grade gliomas, the tumors should be growing or causing symptoms. The trial seeks to determine if adding mebendazole to existing treatments can improve outcomes, such as slowing tumor growth. As a Phase 1/Phase 2 trial, this research aims to understand how the treatment works in people and measure its effectiveness in an initial, smaller group, offering participants a chance to contribute to groundbreaking advancements in brain tumor treatment.

Previous studies found that combining mebendazole with bevacizumab and irinotecan is safe and well-tolerated, even at high doses, indicating a good safety record for treating high-grade gliomas. For low-grade gliomas, research on carboplatin, vincristine, and temozolomide has shown these drugs are generally safe, though some side effects may occur. Adding mebendazole to this treatment has been considered safe and might improve outcomes compared to using temozolomide alone. Overall, while some side effects may occur, past research has shown these drug combinations to be well-tolerated.¹²³⁴⁵

Researchers are excited about these treatments for brain tumors because they explore the unique potential of combining mebendazole, a drug traditionally used for parasitic infections, with chemotherapy agents like carboplatin, temozolomide, and vincristine. Unlike standard treatments that primarily focus on killing cancer cells, mebendazole may disrupt the tumor's ability to grow by targeting its microtubules, a critical component of cell division. This novel approach could enhance the effectiveness of existing chemotherapy regimens, offering hope for improved outcomes, especially in challenging conditions like gliomas. Additionally, this multi-drug regimen might provide a more comprehensive attack on tumor cells, potentially overcoming resistance seen in conventional therapies.

This trial will evaluate the effectiveness of mebendazole combined with different chemotherapy regimens for brain tumors. Participants in the low-grade glioma arm will receive vincristine, carboplatin, temozolomide, and mebendazole. Studies have shown that this combination can be effective against low-grade gliomas, with a pilot study reporting an 87% survival rate over five years. Participants in the high-grade glioma/pontine glioma arm will receive bevacizumab, irinotecan, and mebendazole. Mebendazole shows promise for high-grade gliomas, with some studies reporting a 33% overall response rate when combined with bevacizumab and irinotecan. Early research in lab settings and mice suggests that mebendazole can slow the growth of brain tumor cells. Overall, these studies indicate that adding mebendazole to existing treatments may improve outcomes for patients with certain brain tumors.¹³⁵⁶⁷