This information is for educational purposes only. Off-label drug use carries risks. Always consult a qualified physician before using any drug outside its approved indication.
Itraconazole
Itraconazole
Brand names: Sporanox, Onmel, Tolsura
ORIGINALLY APPROVED FOR
Fungal Infections (Aspergillosis, Histoplasmosis, Candidiasis)
Itraconazole was discovered to have potent anti-cancer activity through a mechanism entirely different from its antifungal properties. It is a powerful inhibitor of the Hedgehog (Hh) signalling pathway — which drives growth of basal cell carcinoma, medulloblastoma, and prostate cancer. Separately, it inhibits angiogenesis by blocking VEGFR2 trafficking and mTOR signalling. Phase II clinical trials have shown significant activity in prostate cancer and basal cell carcinoma.
Molecular Pathways Targeted
Mechanism of Action in Cancer
Inhibits Hedgehog pathway by trapping Smoothened (SMO) protein in an inactive state → suppresses Gli transcription factors → blocks tumour proliferation in Hh-driven cancers. Also blocks VEGFR2 glycosylation and trafficking to cell surface → impairs tumour angiogenesis. Inhibits mTOR independently.
Cancers Studied
Typical Off-Label Dosing
200 mg twice daily (same as antifungal dosing). Best absorbed with full meal or acidic beverage. Must use oral solution formulation for predictable absorption. Requires therapeutic drug monitoring in some protocols.
* Dosing information from research literature only. Not a prescription. Requires physician supervision.
Cautions & Drug Interactions
- Strong CYP3A4 inhibitor — major drug-drug interactions with many chemotherapy agents
- Cardiac: avoid in ventricular dysfunction — negative inotropic effect
- Liver toxicity — monitor LFTs, especially with long-term use
- Absorption very erratic with capsules — oral solution preferred for consistent levels
- Can elevate levels of statins, benzodiazepines, many other drugs
- Requires physician monitoring — drug level testing recommended