What cancer types were most commonly treated with ivermectin in oncology trials?

Checked on December 7, 2025
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Executive summary

Clinical research testing ivermectin in human oncology so far has focused mainly on triple‑negative breast cancer (TNBC) in combination with immune checkpoint inhibitors; preclinical and lab studies have targeted multiple cancers including pancreatic and breast cell lines [1] [2] [3]. Large, conclusive randomized trials in humans are absent and most evidence remains preclinical or early phase [4] [3].

1. What trials actually exist — and which cancer was most commonly studied?

The clearest, repeatedly cited clinical program is a phase I/II trial evaluating oral ivermectin combined with balstilimab (an anti‑PD‑1 checkpoint inhibitor) for metastatic triple‑negative breast cancer (TNBC), NCT05318469, listed in ASCO abstracts and trial registries [5] [1]. Multiple patient‑facing writeups and trial summaries cite TNBC as the principal human cancer indication being actively tested with ivermectin in combination with immunotherapy [1] [6].

2. Preclinical breadth — many cancer types in the lab, few in people

Laboratory and animal experiments report ivermectin activity across a spectrum of cancer cell lines — breast, glioblastoma, pancreatic, colorectal and others — showing antiproliferative and pro‑apoptotic effects in vitro and in vivo models [3] [7] [2]. Notably, a 2025 Anticancer Research paper described ivermectin synergizing with recombinant methioninase to eradicate MiaPaCa‑2 pancreatic cancer cells in vitro [2]. These preclinical signals have driven interest but are not equivalent to proven clinical benefit [3].

3. Why TNBC? A mechanistic and pragmatic explanation

Investigators emphasize TNBC because it is an aggressive, immunologically “cold” tumor where drugs that stimulate T‑cell infiltration could matter; some preclinical work suggests ivermectin can increase immune infiltration and sensitize tumors to checkpoint blockade, making TNBC an attractive early clinical target [1] [6]. Trial designers also favor repurposing an inexpensive, well‑characterized drug as a partner to immunotherapy — a practical rationale cited in trial documents and commentary [1] [6].

4. Scale and quality of human evidence — still early and limited

Reviews and clinical‑trial overviews stress that human evidence is scarce: there are no large randomized controlled trials showing benefit of ivermectin for cancer, and most clinical activity is early phase (phase I/II) or confined to single‑site studies and abstracts [4] [5]. Fact‑checking and major health‑news outlets echo this caution: researchers note promising lab data but no proof of efficacy in humans to date [8] [9].

5. Competing perspectives and the risk of misinformation

Some patient‑oriented and integrative sites champion broad off‑label use and anecdotal protocols, citing case reports and non‑peer‑reviewed compilations; these sources often extrapolate from lab work or small case series and promote usage beyond controlled trials [10] [11]. Mainstream cancer centers and public health fact‑checks warn that such promotion risks patient harm, therapy abandonment, and toxicity misattribution; experts cited by AP and NYT say ivermectin is not a proven cancer cure and must be tested rigorously [8] [9].

6. What remains unaddressed in available reporting

Available sources do not mention large‑scale randomized phase III trials of ivermectin for any cancer, nor do they provide evidence that ivermectin monotherapy cured or meaningfully prolonged survival in humans [4] [8]. The literature also does not document consistent, replicated clinical efficacy signals across cancer types beyond the ongoing TNBC combination trials [4] [5].

7. How to interpret the landscape — pragmatic advice for clinicians and patients

The scientific case for further testing is based on plausible mechanisms and preclinical activity across multiple cancers; however, the only significant human trial activity identified targets metastatic TNBC in combination with checkpoint inhibitors [1] [2]. Clinicians and patients should view ivermectin today as an investigational repurposing candidate with encouraging lab data but unproven clinical benefit; using it outside trials for cancer lacks support from randomized evidence and is discouraged by mainstream experts [4] [8].

Limitations: this synopsis uses the provided reporting and trial abstracts; other unpublished or later trials may exist but are not cited in the current sources (not found in current reporting).

Want to dive deeper?
Which oncology trials tested ivermectin and what were their cancer types and phases?
What dosing regimens and routes were used for ivermectin in cancer clinical studies?
What clinical outcomes and safety results have ivermectin oncology trials reported?
Is there a proposed anticancer mechanism of action for ivermectin and which tumor types express relevant targets?
Are any ongoing or planned randomized trials evaluating ivermectin as a cancer therapy?