Ivermectin-Mebendazole Combination Shows Favorable Patient-Reported Outcomes in Cancer Patients: Real-World Study
USA: A prospective observational study published in Anticancer Research has examined real-world experiences of cancer patients who received the antiparasitic drugs ivermectin and mebendazole as off-label therapy. The findings suggest high levels of treatment adherence, generally favorable tolerability, and substantial self-reported clinical benefit, although the researchers emphasize that randomized clinical trials are needed to determine efficacy.
The study was led by Nicolas Hulscher, Department of Epidemiology, McCullough Foundation, Dallas, TX, USA, and colleagues.
Drug repurposing has attracted growing interest in oncology because existing medications often have established safety profiles and lower costs than newly developed therapies. Both ivermectin and mebendazole have demonstrated anticancer activity in laboratory and preclinical studies, but evidence from clinical settings remains limited.
To investigate their real-world use, researchers analyzed data from a prospective observational cohort of 197 patients with cancer who were prescribed ivermectin and mebendazole through a U.S.-based telemedicine platform. Participants received compounded capsules containing 25 mg of ivermectin and 250 mg of mebendazole. Outcomes were assessed using standardized digital surveys completed at baseline and after six months of follow-up.
The study led to the following findings:
- Of the 197 enrolled patients, 122 (61.9%) completed the six-month follow-up assessment.
- The mean age of participants was 67 years, with a nearly equal distribution of men and women.
- The most common cancers were prostate cancer (27.9%) and breast cancer (18.3%).
- At baseline, 37.1% of participants reported active cancer progression.
- Treatment adherence was high, with 86.9% of participants completing their initial prescription and 66.4% remaining on therapy at six months.
- The overall clinical benefit ratio was 84.4%.
- At six months, 32.8% of participants reported no evidence of disease, while 15.6% reported tumor regression.
- Stable disease was reported by 36.1% of participants, and 15.6% reported disease progression.
- Many patients received concurrent cancer treatments, including chemotherapy (27.9%), radiation therapy (21.3%), surgery (19.7%), dietary modifications (37.7%), and nutritional supplements (49.2%).
- Side effects were reported by 25.4% of participants and were primarily mild, dose-dependent gastrointestinal symptoms.
- Despite adverse events, 93.6% of affected participants continued treatment after dose adjustments.
The authors noted that the observational design, self-reported outcomes, absence of a control group, incomplete follow-up, and concurrent use of other cancer therapies limit the interpretation of treatment effectiveness.
The researchers concluded that off-label ivermectin and mebendazole use was associated with high adherence, good tolerability, and self-reported clinical benefit in this real-world cohort. However, they emphasized that the findings are hypothesis-generating and warrant confirmation in randomized controlled trials.
Reference:
Hulscher N, Victory K, Thorp JA, Pinsky D, Diaz-Villalobos A, Gillooly P, Coulson F, Annazone M, Radesi C, Brooks J, McCULLOUGH PA, Risch H. Real-world Clinical Outcomes of Ivermectin and Mebendazole in Cancer Patients: Results from a Prospective Observational Cohort. Anticancer Res. 2026 Jun;46(6):3243-3255. doi: 10.21873/anticanres.18194. PMID: 42203321.
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