• Vaccine Name: MUC1 Peptide-Poly-ICLC Vaccine
• Target Pathogen: Cancer
• Target Disease: Cancer
• Vaccine Ontology ID: VO_0007698
• Type: Peptide vaccine
• Status: Clinical trial
• Host Species for Licensed Use: None
• Antigen: 5 MUC1 VNTRs (100 aa; 5×GVTSAPDTRPAPGSTAPPAH (Kimura et al., 2013)
• MUC1 gene engineering:
  • Type: Recombinant protein preparation
  • Description: This vaccine contains mucin 1 (MUC1) peptide. (NCIT_C82417; NCT00986609; NCT03300817)
  • Detailed Gene Information: Click Here.
• Immunization Route: subcutaneous injection
• Description: A vaccine preparation containing MUC1 100-mer peptide (100µg) and the adjuvant Poly-ICLC (Hiltonol®, 500µg; TLR3 agonist) with potential immunopreventive and antineoplastic activities, evaluated in four clinical settings: colorectal cancer prevention (two trials), triple-negative breast cancer (TNBC), and lung cancer prevention (NCIT_C82417; NCT00986609; NCT03300817). In the first colorectal trial (Phase I/II; n=39; (Kimura et al., 2013)), 43.6% (17/39) responded with anti-MUC1 IgG ↑2.2–36.3-fold at week 12; 75% (12/16) mounted booster memory at week 52. In the second colorectal trial (Phase II RCT; n=103; (Schoen et al., 2023)), 25% (13/52) vaccine vs 0/50 placebo responded (p<0.0001); 84.6% (11/13) mounted booster memory; immune responders showed 38% absolute reduction in adenoma recurrence vs placebo (27.3% vs 66.0%, aRR=0.41, P=0.08). In the lung cancer prevention trial (Phase I; n=45 completers; (Pennathur et al., 2025)), 13.3% (6/45) responded; high circulating MDSCs found in both current and former smokers. TNBC trial (Early Phase I; n=29); results not posted. Across colorectal trials, MDSCs were elevated pre-vaccination in non-responders (Kimura et al., 2013; Schoen et al., 2023); PMN-MDSC and e-MDSC subtypes and elevated IL-6 and IL-8 were specifically associated with non-response in the second trial (Schoen et al., 2023). Antibodies cloned from one high-responding vaccinee from the pilot trial (Lohmueller et al., 2016) using proteomics/NGS identified 13 fully human anti-MUC1 IgGs binding tumor-associated hypoglycosylated MUC1 on tumor but not normal tissues, with nanomolar-picomolar Fab affinities; H15K6 and H16K6 mediated CDC in the original complement assay; experimental CAR-T cells constructed from antibody variable regions achieved 9–33% specific lysis of MUC1+ tumor cells with IFN-γ production. Follow-up functional characterization (McKeague et al., 2024) demonstrated that vaccine-elicited anti-MUC1 mAbs mediated ADCC, ADCP, ADCR, and ADCT but not CDC under the experimental conditions tested; membrane-proximal epitopes enhanced ADCP/ADCT efficiency. Pre-vaccination transcriptomics ((Cameron et al., 2024); n=69) identified higher CD4+ frequencies, enriched iCOSL/PI3K/AKT/mTOR and B-cell signaling, elevated CD40L and NFkB phosphorylation in responders; week 2 transcriptomic model achieved AUROC 0.741 with six transcripts directly linked to week-12 antibody titer (HLA-DQA2, DDX12P, C22orf29 positive; CEP55, TNFSF14, RP11-81H14.2 negative). Safety (Schoen et al., 2023): grade 2 injection site reactions in 69.8% (37/53); no grade ≥3 treatment-related adverse events. No evidence of adverse effects or autoimmunity from vaccine-elicited antibodies was observed during >5 years of follow-up in the pilot-derived antibody study (Lohmueller et al., 2016).

Human Response

• Immune Response: 4/27 current smokers and 2/18 former smokers responded to the vaccine; overall MUC1 vaccine immunogenicity was 13.3% (6/45) (NCT03300817).