Last updated: February 2026
Ivermectin, an FDA-approved antiparasitic drug, shows promising anticancer effects against glioblastoma multiforme (GBM) in preclinical studies. Research demonstrates multiple mechanisms including Akt/mTOR pathway inhibition, mitochondrial dysfunction induction, and anti-angiogenic activity â all targeting the most aggressive primary brain cancer with a median survival of just 12-15 months.
Multiple preclinical studies have investigated ivermectin's effects on glioblastoma cells. The following are the major findings from peer-reviewed research:
Research has identified several molecular pathways through which ivermectin exerts anti-tumor effects in glioblastoma cells:
Ivermectin suppresses phosphorylation of Akt, mTOR, and ribosomal S6 in glioblastoma cells. The Akt/mTOR pathway is a central regulator of cell survival, growth, and proliferation â its inhibition leads to reduced tumor cell viability and increased autophagy [1] [3].
Ivermectin decreases mitochondrial respiration, membrane potential, and ATP levels while increasing mitochondrial superoxide production. This creates lethal oxidative stress in tumor cells. The effects are reversed in mitochondria-deficient cells or with antioxidant treatment, confirming the mitochondrial mechanism [1].
By blocking the AKT/mTOR signaling axis, ivermectin triggers autophagy â a cellular self-digestion process. In glioma cells, this autophagy is cytotoxic rather than protective, leading to autophagic cell death [3].
P21-activated kinase 1 (PAK1) is an oncogenic kinase involved in proliferation, metastasis, and angiogenesis. Ivermectin inhibits PAK1, disrupting downstream signaling cascades that promote tumor growth. This mechanism has been demonstrated across multiple cancer types [4].
Ivermectin blocks GLUT4-mediated glucose uptake and inhibits the JAK/STAT signaling pathway, starving glioma cells of their glycolytic fuel and accelerating autophagic death [5].
GBM is one of the most vascularized brain tumors. Ivermectin inhibits capillary network formation and suppresses proliferation and survival of human brain microvascular endothelial cells (HBMECs), targeting the tumor's blood supply [1].
Mitochondrial dysfunction and anti-angiogenesis in GBM
Liu et al. demonstrated that ivermectin inhibits glioblastoma growth in vitro and in two xenograft mouse models by inducing mitochondrial dysfunction, oxidative stress, and suppressing the Akt/mTOR pathway. Also showed anti-angiogenic effects on brain endothelial cells.
Liu Y, et al. Biochem Biophys Res Commun. 2016;480(3):415â421. PMID: 27771251
Cell cycle arrest and apoptosis in glioma
Song et al. showed ivermectin induces dose-dependent growth inhibition, G0/G1 cell cycle arrest (via CDK2/4/6 downregulation), and caspase-dependent apoptosis in glioma cells both in vitro and in vivo.
Song D, et al. J Cell Biochem. 2019;120(1):622â633. PMID: 30596403
Autophagy via AKT/mTOR in glioma
Ivermectin was shown to induce autophagy-mediated cell death in glioma cells through inhibition of the AKT/mTOR signaling pathway, paralleling PAK1/Akt/mTOR axis disruption seen in breast cancer.
Liu J, et al. Biosci Rep. 2019;39(12):BSR20192489. PMC6900471
Broad anticancer review including GBM
Comprehensive review of ivermectin's anticancer properties across tumor types. Confirmed dose-dependent inhibition in U87 and T98G GBM cells and caspase-dependent apoptosis. Highlighted multiple mechanisms including PAK1 inhibition, WNT-TCF pathway modulation, and mitochondrial respiration disruption.
Tang M, et al. Pharmacol Res. 2021;163:105207. PMC7505114
Glycolysis inhibition via GLUT4/JAK/STAT
Feng et al. discovered that ivermectin accelerates autophagic glioma cell death by inhibiting glycolysis through blocking GLUT4-mediated JAK/STAT signaling pathway activation, revealing a metabolic vulnerability.
Feng M, et al. Environ Toxicol. 2022;37(4):754â765. DOI: 10.1002/tox.23440
Comprehensive review: ivermectin as glioma therapeutic strategy
Hu et al. published a comprehensive review consolidating all known anti-glioma mechanisms of ivermectin, including selective targeting of tumor-specific proteins, programmed cell death induction, and modulation of tumor-related signaling pathways.
Hu X, et al. J Neurosci Res. 2024;102(1):e25254. PMID: 37814994
Ivermectin + ATRA repurposing for GBM
Preprint study combining ivermectin with all-trans retinoic acid (ATRA) showed promising results against glioblastoma multiforme, providing evidence for drug combination approaches.
medRxiv preprint. 2024. DOI: 10.1101/2024.08.26.24312575
Intranasal nanoparticle delivery for brain targeting
First study exploring direct ivermectin delivery to the brain. Nanoencapsulated ivermectin administered intranasally showed targeted glioblastoma suppression, addressing the blood-brain barrier challenge.
Published 2025. PMID: 40497800
Review: Repurposing ivermectin for GBM
Latest review article consolidating the case for ivermectin repurposing in glioblastoma, addressing critical issues around bioavailability, dosing, and translational challenges.
Med Chem Res. 2025. DOI: 10.1007/s00044-025-03435-z
| Context | Dose | Notes |
|---|---|---|
| FDA-approved antiparasitic | 0.15â0.2 mg/kg (single dose) | Standard human dose for parasitic infections |
| In vitro GBM studies | 5â40 ΞM | Concentration range showing anti-tumor effects in cell culture |
| In vivo mouse xenografts | 10â40 mg/kg | Doses used in mouse tumor models (not directly translatable to humans) |
| Off-label cancer protocols (anecdotal) | 0.5â2 mg/kg | Reported in alternative medicine contexts; no clinical trial data for GBM |
Blood-brain barrier challenge: A major limitation is whether oral ivermectin achieves therapeutic concentrations in brain tissue. Ivermectin is a substrate of P-glycoprotein efflux pumps at the BBB, which may limit brain penetration. The 2025 intranasal nanoparticle study [7] represents an attempt to address this challenge.
Mouse-to-human dose scaling: Doses effective in mouse xenograft models (10â40 mg/kg) cannot be directly translated to human doses. Allometric scaling and species-specific pharmacokinetics must be considered. The effective in vitro concentrations (5â40 ΞM) may be difficult to achieve with standard oral dosing.
Standard of care for GBM: The current standard treatment for glioblastoma is maximal surgical resection followed by radiation with concurrent and adjuvant temozolomide (the Stupp protocol). Median survival is approximately 14â17 months. Patients should not delay or replace proven treatments with experimental approaches.
Related: For general ivermectin dosing and protocol information, see our Ivermectin Protocol Guide.