Mebendazole in Glioma Brain Cancer Model - 2025 Johns Hopkins Study

HIGHLIGHTS:

In this preclinical study, scientists tested Mebendazole on various brain cancer (glioma) cells in lab dishes and in mice with tumors grown from patient-derived cells.

KEY FINDINGS: 1. MBZ kills or slows cancer cells on its own — It worked against many different types of malignant (aggressive) cancer cells at low concentrations. In patient-derived glioma cells, it was effective at even lower doses. 2. MBZ improves survival in mice — Using a special mouse model with a patient-derived grade 3 oligodendroglioma (a type of glioma), oral Mebendazole roughly doubled survival (from about 100 days untreated to 187 days treated). 3. How MBZ works (the mechanisms) — It causes DNA damage (shown by increased markers like pγH2AX), disrupts microtubules (the cell’s “skeleton” needed for division), and triggers apoptosis (programmed cell death) through caspases (enzymes that dismantle dying cells). It also affects proteins involved in cell cycle control and survival. 4. Even better when combined with radiation therapy (RT) — Radiation is a standard glioma treatment. In lab tests, MBZ + RT killed more cells than either alone, caused more cells to arrest in a vulnerable phase of division (G2/M). In the mouse model (tumors implanted in the brain): Untreated: short survival (109 days). RT alone: some improvement (median ~130 days). MBZ alone: better (median ~215 days). MBZ + RT: the best result — significantly longer survival than either alone, with over half (54%) of mice surviving long-term (>290 days, cured in many cases), and brain scans showing no tumor left in those long-term survivors. No major side effects (like weight loss) were seen with the combination. WHY THIS MATTERS: High-grade gliomas (aggressive brain cancers) usually have poor survival (often <2 years even with surgery, radiation, and chemo). Lower-grade ones (like this IDH-mutant type) get less attention and are often just watched after surgery. Mebendazole gets into the brain well, is already proven safe in humans (including glioma patients in earlier trials with temozolomide chemo), and is inexpensive. This study suggests Mebendazole could enhance radiation’s effect in IDH-mutant gliomas, potentially leading to better outcomes with fewer side effects. The researchers conclude that MBZ + radiation works better together than separately, supporting future human clinical trials to test this combination in specific patient groups (e.g., those with IDH mutations). My Take…

Look at Radiation only vs (MBZ + Radiation) Radiation only = 1/11 mice showed long term survival (more than 290 days) MBZ + Radiation = 6/11 mice showed long term survival (more than 290 days) The improvement that Mebendazole brings to Radiation Therapy is insane! Then look at this comparison: Untreated mice median survival 109 days Radiation Therapy only treated mice: 130 days (19% better) Mebendazole only treated mice: 215 days (97% better) MBZ + Radiation - we don’t get a “median survival” value because 6/11 mice survived more than 290 days. When to give Mebendazole? 1 day before Radiation Therapy had the best results, but giving it the same day as Radiation was almost as good.

Sources and References:

1. Repurposing the anthelmintic drug mebendazole in combination with radiation therapy in an isocitrate dehydrogenase mutant glioma model (PubMed 2025)
2. Dr William Makis. https://x.com/MakisMedicine/status/2010618651456815231?s=20.
3. Ivermectin and Mebendazole for Brain Cancer Success Stories: 115 Case Reports (OneDayMD 2025)