MUC1 Vaccine for Lung Cancer

Not currently recruiting at 1 trial location

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: National Cancer Institute (NCI)

Must not be taking: Steroids, Anti-immune therapy

Disqualifiers: Malignancy, Hepatitis, HIV, Autoimmune, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 4

Duration: 10 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new vaccine to determine if it can help prevent lung cancer in current or former smokers. The MUC1 Peptide-Poly-ICLC Vaccine aims to boost the immune system to combat cellular changes before they become cancerous. It may be suitable for those with a history of smoking at least 30 packs a year, particularly if they quit within the last 15 years. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new vaccine.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you have used oral or systemic steroids or other systemic anti-immune therapy within 90 days before joining. Inhaled or nasal steroids and local steroid injections for pain are allowed.

Is there any evidence suggesting that the MUC1 peptide-Poly-ICLC vaccine is likely to be safe for humans?

Research shows that MUC1-based vaccines could help prevent cancer from worsening. In earlier studies, the MUC1 Peptide-Poly-ICLC vaccine did not cause harmful side effects, indicating safety for participants. It also effectively activated the immune system, a positive sign for its potential effectiveness.

These results suggest the vaccine was well-tolerated in previous studies, although it remains in the early stages of testing. So far, no major negative effects have been reported, which may reassure those considering participation in a clinical trial with this vaccine. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatment options for lung cancer, which often include chemotherapy, radiation, and surgery, the MUC1 Vaccine is unique because it uses a novel approach to target cancer cells. This vaccine is designed to stimulate the immune system by introducing the MUC1 peptide combined with Poly-ICLC, a compound that boosts the immune response. Researchers are excited about this treatment because it aims to train the immune system to recognize and attack cancer cells specifically, potentially leading to fewer side effects and a more targeted attack compared to conventional therapies. This approach could represent a significant advancement in how lung cancer is treated by harnessing the body's natural defense mechanisms.

What evidence suggests that the MUC1 vaccine might be an effective treatment for lung cancer?

Research has shown that the MUC1 peptide-Poly-ICLC vaccine, which participants in this trial will receive, might help prevent lung cancer. It boosts the immune system to recognize and fight cancer cells. Studies have found that MUC1-based vaccines can trigger the body's defenses against tumors, potentially stopping cancer from starting and spreading. Some early tests showed encouraging results, indicating that this vaccine might slow down or stop the transformation from normal cells to cancerous ones. While these findings are promising, further research is needed to fully understand the vaccine's potential in preventing lung cancer. ^{1 2 3 6 7}

Who Is on the Research Team?

Arjun Pennathur

Principal Investigator

Mayo Clinic in Rochester

Are You a Good Fit for This Trial?

This trial is for current or former smokers with a high risk of lung cancer, who have good physical health and normal organ function. Participants should not be pregnant, must agree to use contraception if necessary, and cannot have had any other cancers (with some exceptions), hepatitis B or C, HIV, certain liver diseases, strong allergic reactions to similar vaccines, uncontrolled illnesses that could affect the study's outcome, or autoimmune diseases.

Inclusion Criteria

Creatinine =< institutional upper limit of normal (ULN)

Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT]) =< 1.5 x institutional upper limit of normal (ULN)

Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria

Receiving any other investigational agents

Known autoimmune disease

I am HIV positive.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive MUC1 peptide-Poly-ICLC vaccine subcutaneously at weeks 0, 2, and 10

10 weeks

3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

18 weeks

1 visit (in-person) at week 28

What Are the Treatments Tested in This Trial?

Interventions

• MUC1 Peptide-Poly-ICLC Vaccine

Trial Overview The trial is testing a MUC1 peptide-Poly-ICLC vaccine designed to prevent lung cancer by helping the immune system fight off potential cancer cells. It's in an early phase to see how well it works and what side effects it might cause when given to people at high risk due to their smoking history.

How Is the Trial Designed?

Group I: Prevention (MUC1 peptide-Poly-ICLC vaccine) Experimental Treatment 2 Interventions

Patients receive MUC1 peptide-Poly-ICLC vaccine SC at weeks 0, 2, and 10.

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials

14,080

Recruited

41,180,000+

Published Research Related to This Trial

L-BLP25, a peptide vaccine targeting the MUC1 protein, has shown a strong safety profile and induced a significant immune response in preclinical studies, leading to T-cell proliferation and IFN-gamma production.

In a randomized phase II trial for patients with stage IIIB/IV non-small cell lung cancer, L-BLP25 demonstrated a median survival of 30.6 months compared to 13.3 months for best supportive care, particularly benefiting those with locoregional stage IIIB disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

L-BLP25: a peptide vaccine strategy in non small cell lung cancer. Sangha, R., Butts, C.[2020]

Three different vaccines targeting the MUC1 tumor-associated antigen were tested for their ability to boost immunity and reject tumors in mice, revealing that only the dendritic cell (DC) vaccine effectively induced T cell immunity and led to tumor rejection.

While adjuvant-based vaccines generated antibody responses, they did not stimulate T cell responses or prevent tumor growth, highlighting the importance of T cell-mediated immunity for effective cancer treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Three different vaccines based on the 140-amino acid MUC1 peptide with seven tandemly repeated tumor-specific epitopes elicit distinct immune effector mechanisms in wild-type versus MUC1-transgenic mice with different potential for tumor rejection. Soares, MM., Mehta, V., Finn, OJ.[2022]

MUC1 is a protein that, when overexpressed and improperly modified, can trigger immune responses that may help improve survival rates in various cancers, including breast and pancreatic cancers.

Initial studies of MUC1-based vaccines have shown them to be safe and capable of inducing immune responses, suggesting they could be effective in preventing cancer recurrence and enhancing immune surveillance in high-risk individuals.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Natural and Induced Humoral Responses to MUC1. Von Mensdorff-Pouilly, S., Moreno, M., Verheijen, RH.[2021]

The MUC1-based tricomponent vaccine, when combined with the adjuvant fibroblast stimulating lipopeptide 1 (FSL-1), successfully triggered both humoral and cellular immune responses against cancer cells.

This vaccine not only generated antibodies that effectively targeted MCF-7 tumor cells but also significantly reduced tumor burden, indicating its potential efficacy as an antitumor treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Design of a MUC1-based tricomponent vaccine adjuvanted with FSL-1 for cancer immunotherapy. Li, M., Wang, Z., Yan, B., et al.[2020]

The study developed a Variable Epitope Library (VEL) vaccine platform targeting MUC1, an overexpressed antigen in tumors, which showed promising results in reducing tumor size compared to traditional wild-type peptide treatments.

Vaccination with the VEL immunogens not only reduced tumor area but also decreased lung metastasis and generated a strong CD8+ T cell response, indicating potential for effective cancer immunotherapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Antitumor efficacy of MUC1-derived variable epitope library treatments in a mouse model of breast cancer. Odales, J., Servín-Blanco, R., Martínez-Cortés, F., et al.[2022]

The BLP25 liposomal vaccine was well tolerated in patients with advanced non-small-cell lung cancer, with only mild and self-limiting side effects reported, indicating a favorable safety profile.

Immunological responses were observed, with 5 out of 12 patients generating cytotoxic T lymphocytes against MUC1-positive tumor cells, suggesting potential for further development despite no significant antitumor responses noted.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase I study of the BLP25 (MUC1 peptide) liposomal vaccine for active specific immunotherapy in stage IIIB/IV non-small-cell lung cancer. Palmer, M., Parker, J., Modi, S., et al.[2021]

Vaccines designed to enhance tumor antigen recognition in non-small cell lung cancer patients show promising results, including prolonged stable disease and tumor shrinkage, with no reported toxicity.

Specific vaccines such as LBLP25, TGF-beta2 antisense gene vaccine, and GVAX have demonstrated potential benefits that warrant further Phase III clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A review of vaccine clinical trials for non-small cell lung cancer. Nemunaitis, J., Nemunaitis, J.[2019]

Citations

1. medrxiv.org medrxiv.org/content/10.1101/2025.06.25.25328823v1

A Multicenter Pilot Study of MUC1 Vaccine in Current and ... The MUC1 vaccine was safe and elicited an immune response in 13%, which was lower than anticipated. Unexpectedly, we observed a high level of ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT03300817

NCT03300817 | MUC1 Vaccine in Preventing Lung Cancer ... MUC1 peptide-Poly-ICLC vaccine may stimulate the body's immune system and slow or stop the changes from normal to pre-cancer to cancer. Detailed Description.

3. prevention.cancer.gov prevention.cancer.gov/clinical-trials/clinical-trials-search/nct03300817

MUC1 Vaccine in Preventing Lung Cancer in Current and ... MUC1 peptide-Poly-ICLC vaccine may stimulate the body's immune system and slow or stop the changes from normal to pre-cancer to cancer. Intervention. Laboratory ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S0753332220310805

A review on development of MUC1-based cancer vaccine MUC1-based cancer vaccines are a promising strategy for preventing cancer progression and metastasis. This review summarizes the most significant milestones ...

5. nature.com nature.com/articles/s41698-025-01016-2

Mucin-1: a promising pan-cancer therapeutic target MUC1-based glycopeptide vaccines showed promising anti-tumoral immunity in earlier pre-clinical studies and some promising results in phase 2 ...

6. mycancergenome.org mycancergenome.org/content/clinical_trials/NCT01720836/

Clinical Trial: NCT01720836 There has been no toxicity observed and the vaccine is highly immunogenic in early disease. In the proposed NSCLC trial the anti-MUC1 immune ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12223159/

Mucin-1: a promising pan-cancer therapeutic target - PMC An ongoing phase 1/2 study is investigating the safety and efficacy of MUC1 Peptide-Poly-ICLC (Polyinosinic-polycytidylic Acid Stabilized With ...