News & Clinical Trials

Registry results posted Oct 21, 2026 for a small Phase 2, non-randomized trial at UT Health San Antonio (2017 to 2025). 32 people with advanced hepatocellular carcinoma (liver cancer) who had not had prior systemic treatment started sorafenib, with hydroxychloroquine 400 mg daily added from day 15. Their median time to tumor progression was 8 months (95% CI 4 to 19 months). Only 25 finished the first cycle and 2 completed a full year of treatment. A second group of 5 patients who had already progressed on sorafenib had HCQ added, but none were evaluable. There was no sorafenib-only comparison group, so these results cannot show whether HCQ added any benefit. Results are posted on the registry only; no journal paper yet.

Lab study (Tantawy et al., British University in Egypt): in HN9 oral squamous cell carcinoma cells, adding ivermectin to the chemotherapy drug cisplatin killed fewer cancer cells than cisplatin alone. The combination reduced cisplatin's S-phase cell-cycle arrest and lowered pro-apoptotic markers (BAX, caspase-3); the authors suggest ivermectin's antioxidant and anti-inflammatory effects may interfere with cisplatin. A negative (antagonistic) result in one lab cell line only; not tested in animals or people.

Preclinical study (El-Tanani et al.): in gefitinib-resistant EGFR-mutant NSCLC cells (HCC827 GR5), gefitinib alone barely reduced viability (92.7%), while mebendazole 0.1 µM + gefitinib 1 µM cut viability to 37.7%, with increased DNA damage and γ-H2AX replication-stress signaling. In a mouse model the combination slowed tumor growth (doubling time 6.2 to 25 days) without obvious systemic toxicity. Cell and animal data only; not tested in patients.

Preclinical study (Mohammed et al.): researchers packed hydroxychloroquine and resveratrol together into coated nanoparticles. In HepG2 liver cancer cells the nanoparticles were more toxic than either free drug, triggered apoptosis and affected autophagy, and were less toxic to normal skin cells. A 30-day oral study in mice looked only at safety and found no deaths and mild liver changes. No tumor-shrinkage testing in animals or patients yet.

The June 2026 Anticancer Research self-reported telemedicine cohort of ivermectin plus mebendazole (Hulscher et al.; Anticancer Res 2026 Jun;46(6):3243-3255; original PMID 42203321; DOI 10.21873/anticanres.18194; n=197 enrolled / 122 follow-up) claimed a high rate of clinical benefit. Fully retracted October 2026 (Anticancer Res 2026 Oct;46(10):5877; PMID 42823188; DOI 10.21873/anticanres.18425). Timeline: Expression of Concern 9 Jun 2026 (PMID 42300708), update Sep 2026 (PMID 42703869), full retraction Oct 2026. Grounds were methodological and analytical problems after post-publication review, independent expert assessment, and the authors' appeal — not fabrication. Do not cite the unretracted framing as supporting evidence. Retraction: https://pubmed.ncbi.nlm.nih.gov/42823188/. Evidence level: retraction notice.

As of this curation scan, ClinicalTrials.gov queries for fenbendazole return parasitology/oxfendazole and unrelated studies — not registered human cancer treatment trials of fenbendazole. Evidence level: registry observation; human oncology evidence remains preclinical/case-level.

Animal study (Wistar rats, six per group, no tumors): rats given the checkpoint inhibitor atezolizumab showed oxidative stress, inflammation and ovary damage. Ivermectin 2 mg/kg by mouth for 3 days beforehand lessened these changes and preserved the fertility hormone AMH, but a 6-day course did not help much. This tests side-effect protection, not cancer treatment; small rat study only; not tested in people.

Systematic review and meta-analysis (Al-Talqani et al.) of 7 studies, 383 adults with cancer, comparing chemotherapy plus hydroxychloroquine against chemotherapy alone or placebo. Adding HCQ did not significantly improve objective response (OR 0.67; 95% CI 0.30–1.46), complete or partial response, or stable disease. Side effects such as headache and nausea were similar between groups. The authors call for larger randomized trials. Evidence level: meta-analysis of small studies; negative result.

Preclinical study (Fan et al.): researchers built a DNA-based nanorobot carrying methylene blue as a light-activated (photodynamic) drug plus platinum particles that make oxygen inside tumors. The oxygen bubbles helped it push deeper into tumor tissue and eased low oxygen, which made the light treatment work better; the authors report 85% tumor regression in mice. An engineered nanodevice with light treatment, not methylene blue taken by mouth; mice only; not tested in people.

Preclinical study (Fang et al.): a nanoparticle loaded with hydroxychloroquine was designed to jam the autophagy recycling system in liver cancer cells. It increased immune-alarm (cGAS-STING) signaling and cell damage, and with radiotherapy it improved tumor control in mouse models, including distant tumors, with a further benefit when a checkpoint inhibitor was added. A complex engineered nanoparticle, not plain HCQ; cells and mice only; not tested in people.

Preclinical study (Xu et al.): researchers solved cryo-EM structures of the human OCTN1 transporter, which has been linked to cancer cell growth and invasion. Structure-based virtual screening then identified mebendazole as a potent OCTN1 inhibitor, and mebendazole suppressed the proliferation of cervical and lung cancer cells. Structural and cell-culture data only; not tested in animals or patients in this report.

Preclinical study (Chu et al., McGill University): in human neuroblastoma cells, ivermectin delayed the clearance of stress granules and reduced new production of the protective chaperone Hsp70, by blocking its translation rather than its gene expression. The Hsp70 effect was not seen in cervical carcinoma cells. Cell-culture mechanism data only; no animal or patient results.

Case report (Cheng and Araujo, University of Florida): a 74-year-old man with metastatic melanoma declined standard therapy and took ivermectin and fenbendazole on his own. His tumors and ctDNA (Signatera, 2.04 to 0.18 MTM/mL) initially fell, then both worsened (ctDNA 0.93 MTM/mL, growing axillary node). Because the tumor was extremely high-mutation and immunogenic, the authors note spontaneous immune regression is equally plausible as a drug effect, note there is no clinical evidence of efficacy, and urge safety counseling. Single patient; cannot show cause and effect.

Indiana University Phase II pilot of hydroxychloroquine for joint pain from aromatase-inhibitor–associated musculoskeletal symptoms in breast cancer patients (n≈35). Supportive-care focus. Evidence level: planned pilot; no results yet.

Rutgers Phase I dose-finding study of hydroxychloroquine added to oral decitabine/cedazuridine and venetoclax in acute myeloid leukemia (n≈18). Evidence level: planned early Phase I; no results yet.

University of Florida Phase II randomized study comparing intermediate- vs high-dose ivermectin with standard immune checkpoint inhibitors in adults with solid tumors. Evidence level: planned interventional trial; no results yet.

Preclinical study (Fattahi et al., Tehran University of Medical Sciences): in three prostate cancer cell lines (LNCaP, DU145, PC3), combining mebendazole with the anti-androgen flutamide reduced cell viability, colony formation and migration more than either drug alone, and increased apoptosis. Mebendazole IC50 was about 55 µM. Authors suggest the combination could allow lower flutamide doses. Cell-culture data only; not tested in animals or patients.

Lab chemistry study (Degirmenci et al.): researchers built polymers that carry both the chemotherapy drug gemcitabine and hydroxychloroquine and tested different chemical links for releasing each drug. The best version released both drugs well, was more toxic to pancreatic cancer cells and was taken up by BxPC-3 cells and 3D tumor spheroids. Lab cells only; no animal or human data.

Mouse study reporting that nutritional ketosis may enhance therapeutic effects of mebendazole combined with CPI-613 (devimistat) in juvenile high-grade glioma models. Evidence level: preclinical animal only.

Preclinical evaluation of mebendazole in paediatric low-grade glioma models showing antiproliferative and antimigratory effects. Evidence level: preclinical — complements existing pediatric glioma trial listings.

Systematic review synthesizing preclinical and clinical evidence for mebendazole in primary brain tumours, including CNS penetrance and multimodal anticancer mechanisms. Evidence level: systematic review — summarizes, does not replace, controlled trials.

Meeting abstract (Reynolds and Fly, J Biol Chem vol. 302 suppl., Abstract 4275): reports lab tests of the antiparasitic drugs ivermectin, mebendazole and albendazole in colorectal cancer cells, with the authors describing selective and synergistic anticancer activity. This is a short conference abstract, not a full peer-reviewed paper, and results are in lab cells only; not tested in people.

Exploratory lab study (Onsoti et al., Kenya): in HeLa cervical cancer cells, ivermectin (IC50 about 9.65 µM) and ascorbic acid (vitamin C, IC50 about 4.58 mM) each slowed cell growth. Combined, the effect was additive near those doses and synergistic only at higher concentrations; the authors call the synergy weak. Gene tests showed TP53 up and STAT3, BCL2, HIF1α and TNF down. Lab cells only; not tested in people.

Cell-line study reporting that ivermectin modulates ER, HER2, and TGF-β signaling in ER-positive and endocrine-resistant breast cancer models. Evidence level: preclinical in vitro only.

In vitro study reporting tumor-growth suppression by ivermectin in gastric cancer cell lines and patient-derived organoids. Evidence level: preclinical cell/organoid work — not human clinical efficacy.

Computational-experimental study reporting synergistic responses to sorafenib plus hydroxychloroquine in KRAS-mutant breast cancer models. Evidence level: preclinical — not a clinical trial result.

VA-sponsored Phase I/II randomized study (estimated n≈93; start 2026-04-01) of intra-arterial hydroxychloroquine in lipiodol with transarterial embolization (TAE), then oral HCQ maintenance, versus lipiodol/TAE plus oral placebo, for intermediate-stage unresectable HCC. Evidence level: ongoing Phase I/II; results pending.

Recruiting Phase II combining MEK inhibitor tunlametinib, PD-1 antibody pucotenlimab, and hydroxychloroquine in metastatic NRAS-mutant melanoma. Evidence level: planned/ongoing Phase II; no results yet.

Intravesical nanoformulation combining fenbendazole with photothermal therapy reported to trigger ferroptosis and dual immunotherapy signals in bladder cancer models. Evidence level: preclinical nanomedicine — not a registered human cancer trial of fenbendazole.

Published results (Haldar et al., MD Anderson): 34 patients with KRAS-mutant metastatic pancreatic cancer who had already had at least one treatment got the MEK inhibitor binimetinib plus hydroxychloroquine. The starting dose was poorly tolerated (one grade 3 muscle-enzyme rise with kidney impairment, one grade 3 QT prolongation), so the dose was lowered to binimetinib 30 mg + HCQ 600 mg twice daily. Of 31 patients who could be assessed, 2 had a partial response and 9 had stable disease (response rate 6.5%, disease control 35.5%); median progression-free survival was 1.9 months and median overall survival 5.3 months. The authors conclude the combination had a challenging toxicity profile and limited activity. Honest negative early-phase result.

Cancer Research Communications Phase I (n=11): MTD/RP2D HCQ 600 mg BID with suba-itraconazole 150 mg BID; common AEs hypertension, QTc prolongation, diarrhea, nausea; no PSA declines ≥50%; median PSA-PFS 5.5 months. Planned Phase II expansion not pursued (limited activity + changing BCR landscape).

Lab study (da Silva et al., Brazil): in gastric cancer cell lines, mebendazole reduced cell growth about as much as the chemotherapy drug 5-FU, with less toxicity to non-cancer cells. It lowered expression of nucleotide-metabolism genes (TYMS, MTHFD1, HPRT1, PRPS1), stopped cells in the G0/G1 phase, and computer docking suggested it binds PRPS1 and HPRT1. Patient tumor data linked higher levels of these genes to worse survival. Results are in lab cells and computer models only; not tested in people.

Veterinary trial (Soltanali et al.): 20 pet dogs with canine transmissible venereal tumor were randomly assigned, double-blind, to standard weekly vincristine alone or vincristine plus two ivermectin injections (0.5 mg/kg). Dogs on the combination reached confirmed remission sooner, about 4.3 vs 5.9 weeks for aggressive tumors and 3.8 vs 5.5 weeks for less aggressive ones, with no added blood or kidney toxicity seen. Small study in dogs with a tumor type that responds well to chemotherapy; not tested in people.

The July 2025 Case Reports in Oncology 3-patient fenbendazole self-administration series (originally PMC12215191) was retracted ~Jan 21, 2026 for undisclosed conflict of interest — not for fabrication of outcomes. Do not cite the unretracted framing as supporting evidence. Retraction: PMC12823103; Karger statement: https://karger.com/cro/article/19/1/169/941881/Retraction-StatementPaper-by-William-Makis-Ilyes. Evidence level: retraction notice.

Case report (Krishnan et al.): a 47-year-old woman with metastatic colon cancer on nivolumab/relatlimab developed severe liver injury after raising her own fenbendazole dose. A standard causality score (RUCAM 8, probable) pointed to fenbendazole; her liver tests improved quickly after she stopped it, and immunotherapy was restarted safely without the injury coming back. A later published letter argued the pattern could reflect tumor cell death instead, and the authors replied defending their view. Single patient; cannot prove cause.

Preclinical study (Sheir et al., Egypt): researchers made ivermectin into nanocrystals to get around its poor solubility, then coated them with platelet membranes to help them reach tumors. In lab cells the coated version was more toxic to triple-negative breast cancer cells (MDA-MB-231) than the uncoated one and gentler on normal fibroblasts. In mice with breast tumors it gathered more in the tumor, slowed tumor growth and increased immune T-cell infiltration. Lab cells and mice only; not tested in people.

Randomized double-blind placebo-controlled early Phase I proof-of-principle study of oral hydroxychloroquine before prostatectomy, measuring autophagy markers (p62, LC3-II, NBR1) in tumor tissue (n≈20). Evidence level: biomarker-focused pilot; not an efficacy trial.

Small single-arm pilot (enrolling by invitation) of topical methylene blue dressing for advanced/inoperable squamous cell carcinoma not suitable for radiotherapy. Evidence level: tiny pilot; preliminary only.

Recruiting Phase II focused on autophagy maintenance strategies including hydroxychloroquine in ovarian cancer. Evidence level: ongoing Phase II; efficacy unknown.

Lab study (El-Tanani et al.): in A549 lung cancer and MDA-MB-231 triple-negative breast cancer cells, gefitinib 1 µM plus mebendazole 0.5 µM reduced cell viability to about 8-10% and 15%, well below either drug alone. In the lung cells the combination lowered inflammatory cytokines (IL-1β, IL-6, TNF-α, IFN-γ) and changed genes tied to cell division and nuclear transport. Lab cells only; not tested in animals or people.

Laboratory investigation of ivermectin alone and with doxorubicin in OSCC models, reporting synergistic cytotoxicity signals. Evidence level: in vitro preclinical — not clinical trial data.

Lab study (Baroudi et al.): in oral squamous cell carcinoma cell lines, hydroxychloroquine clearly reduced growth and colony formation of gingival cancer cells (Ca9-22) but had only a moderate effect on tongue cancer cells (SCC-9); another tongue line and normal oral cells showed no response or only non-specific toxicity at high doses. The drug blocked autophagy and changed apoptosis, mitochondrial and DNA-damage markers. Lab cells only; not tested in people.

Pilot of topical methylene blue (0.5 mg/mL) every other day for one month in morpheaform, infiltrative, or noduloulcerative basal cell carcinoma not suitable for surgery/radiotherapy. Evidence level: small pilot; efficacy unproven.

Preclinical study (Bandi et al.): adding the vitamin D analog paricalcitol and hydroxychloroquine to 5-FU plus oxaliplatin increased cancer cell death in three pancreatic cancer cell lines. In mice the combination slowed tumor growth and activated T and NK immune cells. The authors link the effect to lower integrin beta-4 and other tumor-matrix proteins and say it should be tested in trials. Cells and mice only; not tested in people.

Early Phase I pilot of local methylene blue injection as a potential radiosensitizer with concurrent radiation in locoregional recurrent head and neck squamous cell carcinoma (n≈10). Evidence level: small feasibility/safety pilot; efficacy unknown.

Mouse study combining fenbendazole with diisopropylamine dichloroacetate (DADA); authors report synergistic tumor growth inhibition and call for clinical study. Evidence level: preclinical animal only.

Preclinical study linking fenbendazole to pyroptosis via HK2/caspase-3/GSDME signaling in breast cancer cell models. Evidence level: preclinical in vitro — not human trial evidence.

Peer-reviewed review summarizing limited human clinical evidence for ivermectin as cancer therapy, noting widespread off-label interest and the need for well-designed trials. Evidence level: narrative review; not new clinical efficacy data.

Large randomized Phase III trial (estimated n≈474) comparing standard palbociclib + endocrine therapy versus arms adding hydroxychloroquine (with standard- or high-dose palbociclib). Evidence level: confirmatory-design trial planned; no results yet.

Phase I dose-escalation study of IV methylene blue for cytokine release syndrome (CRS) or ICANS after CAR-T or bispecific antibodies. Evidence level: early Phase I safety/preliminary efficacy; not an approved cancer treatment indication.

Peer-reviewed Phase 1 (n=17; Johns Hopkins / All Children’s): oral mebendazole up to 2500 mg/m²/day was tolerable with no dose-limiting toxicities; mean PFS 7.6 weeks (range 2–24). Authors conclude limited evidence of single-agent efficacy in refractory/progressive pediatric brain tumors. Complements the existing registry listing for NCT02644291.

ASCO policy/news item summarizing clinical guidance that ivermectin and fenbendazole should not be recommended for cancer treatment outside well-designed clinical trials, and advising routine conversations about supplements. Evidence level: professional society guidance — not a trial result.

ASCO abstract (meeting ID 250156 / JCO e13146): combination described as safe/well tolerated in early accrual; among 8 evaluable patients, 1 PR, 1 SD, 6 PD (median PFS 2.5 months; 4-month clinical benefit rate 37.5%). Evidence level: conference abstract, small n, preliminary — not peer-reviewed full results.

Study reporting fenbendazole activity against cervical cancer cells and cancer stem cell–like populations in preclinical models. Evidence level: preclinical — not approved human cancer therapy.

Preclinical work exploring mebendazole combined with radiation therapy in an IDH-mutant glioma context. Evidence level: preclinical — not human efficacy data.

Recruiting Phase II comparing a hydroxychloroquine-containing multi-drug regimen versus regorafenib in refractory RAS-mutant metastatic colorectal cancer. Evidence level: ongoing comparative Phase II; no results yet.

Randomized Phase II (n=59) adding hydroxychloroquine 200 mg BID to carboplatin-based chemotherapy in platinum-sensitive relapsed ovarian cancer. No benefit on ORR (85% vs 80%), PFS (12 vs 11 months), or OS (16 vs 21 months) versus chemo alone; HCQ was well tolerated. Honest negative RCT; pairs with ongoing AUTOMAIN. Evidence level: randomized Phase II — negative primary endpoint. Also PMC11839959.

Open-access Phase I report: HCQ was tolerable; RP2D 800 mg/day with continuous palbociclib 75 mg/day + letrozole; best responses included 2 partial responses and 11 stable disease among 14 evaluable patients. Evidence level: single-arm Phase I; larger randomized studies needed.

In vitro and xenograft mouse work reporting proliferation inhibition, apoptosis/mitotic catastrophe signals, and oral fenbendazole effects on tumor growth in ovarian cancer models. Evidence level: preclinical — not a human trial.

Phase Ib/II in 29 HR+/HER2− patients after first-line palbociclib failure. RP2D: HCQ 600 mg BID + palbociclib 200 mg daily. Reported ORR 41.4% in all enrolled; grade ≥3 neutropenia/leukopenia ~25%. Evidence level: single-arm Phase Ib/II; needs randomized confirmation.

Recruiting Phase I/II randomized study of hydroxychloroquine sulfate tablets versus placebo for prevention/treatment of radiation-induced oral mucositis and dysgeusia (n≈158). Supportive-care indication, not antitumor efficacy. Evidence level: ongoing supportive-care trial.

Review of preclinical anticancer activity, oral pharmacokinetics, and toxicity; emphasizes fenbendazole is not FDA/EMA-approved for human cancer and that clinical trials are needed. Evidence level: narrative review.

Recruiting study evaluating hydroxychloroquine pretreatment before radiotherapy and chemotherapy in advanced nasopharyngeal carcinoma. Evidence level: early interventional; not standard of care.

MD Anderson Phase I/II of autophagy inhibition with HCQ added to continuous low-dose palbociclib + letrozole. Completed; Phase I results published in npj Breast Cancer (see companion news entry). Evidence level: early-phase completed trial.

Completed Dana-Farber Phase II of ERK inhibitor LY3214996 alone or with hydroxychloroquine in advanced pancreatic cancer. Evidence level: completed Phase II; review registry/publications for outcomes before drawing conclusions.

Institutional patient-facing summary of the recruiting Phase I/II study pairing oral ivermectin with investigational anti–PD-1 balstilimab (NCT05318469). Notes experimental use in this setting. Evidence level: trial information page, not outcome data.

Cedars-Sinai Phase I/II study of oral ivermectin with immune checkpoint inhibitors in metastatic triple-negative breast cancer. Estimated primary completion Oct 2026. Evidence level: ongoing interventional trial; efficacy not established.

Phase II study comparing leflunomide versus MEK inhibitor plus hydroxychloroquine (± bevacizumab) in refractory RAS-mutant colorectal, pancreas, or lung cancer (n≈20). Evidence level: small ongoing Phase II; interpret cautiously. Registry update: ClinicalTrials.gov now lists this trial's status as unknown (last known: recruiting; not updated since Jan 2024).

Phase II study of hydroxychloroquine added to FOLFIRI and bevacizumab in metastatic colorectal cancer enriched for drug-tolerant persister (DTP) biology. Status: active, not recruiting. Evidence level: ongoing Phase II.

Registry record for the Gong et al. Phase Ib/II combination of hydroxychloroquine with escalated palbociclib doses in advanced HR+/HER2− disease after CDK4/6 inhibitor failure. Evidence level: see published Med paper for outcomes.

Mayo Clinic Phase III comparing methylene blue oral rinse versus standard-of-care mouthwash for oral mucositis pain in patients with solid or hematologic malignancies (n≈49). Supportive-care pain indication. Evidence level: completed Phase III supportive-care trial; not an antitumor study.

Preclinical work improving fenbendazole solubility via PLGA nanoparticles; intravenous nanoparticles reduced tumor weight in cell-line and patient-derived xenografts where oral fenbendazole did not. Evidence level: preclinical formulation study.

Northwestern Phase II of mitochondrial metabolism inhibitor CPI-613 (devimistat) plus hydroxychloroquine with 5-fluorouracil or gemcitabine in advanced chemorefractory solid tumors including biliary, colorectal, GEJ, and lung adenocarcinoma (n≈94). Evidence level: ongoing Phase II; efficacy not established.

Mouse study reporting that ivermectin reduced lung metastasis associated with neutrophil extracellular traps without reducing primary tumor growth. Evidence level: preclinical animal study — not human trial data.

Laboratory study of methylene blue photodynamic therapy reducing cell viability and MMP-9 in oral carcinoma and leukoplakia cell models. Evidence level: in vitro preclinical.

Completed Johns Hopkins Phase I safety study of mebendazole in children/young adults with recurrent medulloblastoma or high-grade glioma / DIPG. Evidence level: completed Phase I; registry results should be reviewed separately.

Randomized double-blind placebo-controlled report (n=40) claiming higher overall response (65% vs 10%) and longer PFS (9.25 vs 3 months) with mebendazole 500 mg BID added to bevacizumab/FOLFOX4. Evidence level: small single-center RCT; needs independent replication before practice change.

Medical University of South Carolina Phase I/II testing oral combinations of hydroxychloroquine with metformin, sirolimus, and optionally dasatinib/nelfinavir in advanced solid tumors and recurrent prostate cancer (n≈76). Evidence level: ongoing multi-drug early-phase study; efficacy unknown.

Phase I combination of hydroxychloroquine and chlorphenesin carbamate with mFOLFIRINOX in pancreatic cancer. Status: active, not recruiting. Evidence level: early Phase I safety study.

Phase II University of Pennsylvania trial testing abemaciclib alone vs abemaciclib + HCQ to target disseminated tumor cells / minimal residual disease after primary breast cancer therapy. Evidence level: ongoing randomized Phase II; results pending.

Fox Chase early Phase I neoadjuvant study of paricalcitol, hydroxychloroquine, and losartan (PHL) to modify stroma in resectable pancreatic ductal adenocarcinoma (n≈20). Status: active, not recruiting. Evidence level: small early-phase stroma-focused study.

In vitro study reporting dose-dependent growth inhibition, ROS-mediated mitochondrial apoptosis, and S-phase arrest in colorectal cancer cell lines. Evidence level: preclinical cell work only — not human clinical efficacy.

Phase II multi-arm study exploring avelumab or hydroxychloroquine with or without palbociclib to clear dormant breast cancer cells. Evidence level: ongoing interventional; efficacy not established.

Completed Johns Hopkins Phase I dose-finding study of mebendazole with temozolomide after chemoradiation. Protocol notes preclinical fenbendazole observations led investigators to select mebendazole for human testing. Evidence level: completed Phase I; not proof of clinical benefit.

Penn Phase II of MEK inhibitor binimetinib plus hydroxychloroquine in advanced KRAS-mutant non-small cell lung cancer (n=9 enrolled). Evidence level: completed small Phase II; review registry/publications before drawing conclusions.

Small uncontrolled case series (often cited as Stanford/Chiang) describing radiographic responses in genitourinary cancer patients who took fenbendazole with concurrent or recent standard therapies. Evidence level: anecdotal; concurrent treatments confound attribution.

HonorHealth Phase II neoadjuvant study adding hydroxychloroquine to nab-paclitaxel, gemcitabine, and cisplatin in untreated resectable, borderline, or locally advanced pancreatic adenocarcinoma (n≈19), focusing on CA19-9 normalization. Evidence level: completed small Phase II.

Phase I/II of checkpoint blockade plus HCQ in advanced melanoma. Status: active, not recruiting. Evidence level: early-phase combination; await published outcomes.

Emory Phase II of vitamin D analog paricalcitol and hydroxychloroquine combined with gemcitabine and nab-paclitaxel in advanced/metastatic pancreatic adenocarcinoma (n≈10). Evidence level: completed small Phase II.

Randomized Phase II preoperative study: HCQ arm improved pathologic and CA19-9 responses but did not improve overall or recurrence-free survival. Evidence level: randomized Phase II — mixed endpoints; no survival benefit shown.

Utah Phase I dose-escalation of ERK inhibitor ulixertinib (BVD-523) with fixed-dose hydroxychloroquine in advanced MAPK-mutated gastrointestinal adenocarcinomas (n≈16). Evidence level: completed Phase I; early safety/dose data only.

Pediatric Brain Tumor Consortium Phase I/II adding hydroxychloroquine to dabrafenib and/or trametinib in children with recurrent/progressive low- or high-grade glioma with BRAF aberration (n≈57). Status: active, not recruiting. Evidence level: ongoing early-phase pediatric combination; efficacy not established.

Norwegian Phase I of proteasome inhibitor carfilzomib plus hydroxychloroquine (with dexamethasone) in relapsed/refractory multiple myeloma (n≈19). Evidence level: completed Phase I; check publications for outcomes.

MD Anderson Phase I of binimetinib plus hydroxychloroquine in KRAS-mutant metastatic pancreatic adenocarcinoma (n≈34). Evidence level: completed Phase I dose-finding; not proof of clinical benefit.

Phase 2 randomized clinical trial in metastatic/advanced pancreatic cancer: higher response rate with HCQ but no overall survival benefit. Evidence level: randomized Phase II — response ≠ survival benefit.

Tanta University Phase III–labeled randomized study of mebendazole added to FOLFOX + bevacizumab in colorectal cancer. Evidence level: registry lists recruiting; interpret published companion results cautiously and independently.

MD Anderson Phase I/II of hydroxychloroquine with gemcitabine and docetaxel in recurrent or refractory osteosarcoma (n≈31). Status: active, not recruiting. Evidence level: early-phase sarcoma combination; await published outcomes.

Phase 2a TDM-guided study of mebendazole in advanced gastrointestinal cancer or cancer of unknown origin. Terminated for lack of effect (n=11). Evidence level: small terminated early-phase study — negative signal in this setting.

Phase II pilot of hydroxychloroquine, everolimus, or the combination for prevention of recurrent breast cancer (stage IIB+). Status: active, not recruiting. Evidence level: prevention-focused early Phase II.

Penn Phase I/II of hydroxychloroquine added to dabrafenib and trametinib in advanced BRAF-mutant melanoma (n≈50). Companion imaging study NCT05279027 exists. Evidence level: completed early-phase combination; review publications for final outcomes.

Completed early study of hydroxychloroquine alone in heavily pretreated metastatic pancreatic cancer; short median PFS/OS reported historically. Evidence level: older single-agent study — limited activity as monotherapy.

Phase I/II Northwell/Cohen Children's study of oral mebendazole plus standard regimens in pediatric low- and high-grade gliomas / DIPG. Status: active, not recruiting. Evidence level: early-phase combination safety/efficacy study.

Phase II of erlotinib with or without HCQ in chemo-naive advanced NSCLC with EGFR mutations. Status listed active, not recruiting on registry scans. Evidence level: older Phase II combination; check current publications for final outcomes.