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NEW YORK, NY – A groundbreaking personalized cancer vaccine, PGV001, has demonstrated promising results in a phase 1 clinical trial, offering hope for improved cancer recurrence prevention. Researchers at the Icahn School of Medicine at Mount Sinai, under the leadership of Nina Bhardwaj, MD, Ph.D., have developed this multi-peptide neoantigen vaccine, designed to harness the body’s immune system to combat cancer.
The early-stage study, published in Cancer Discovery, involved 13 patients with various cancers, including non-small cell lung cancer, head and neck cancer, urothelial cancer, breast cancer, and multiple myeloma. The vaccine utilizes personalized neoantigens—unique mutations found in cancer cells but not healthy cells—to stimulate a targeted immune response.
“We wanted to develop cancer vaccines that can stop cancer from coming back in patients who are at high risk of recurrence,” said Dr. Bhardwaj. “This study shows that making personalized cancer vaccines is possible and safe. While this is a phase 1 study with a small group of patients, it’s an exciting step toward using the immune system to help people live cancer-free, longer.”
The PGV001 vaccine is tailored to each patient’s specific cancer, utilizing advanced computational tools to identify the most effective neoantigens. This personalized approach aims to overcome the limitations of existing immune-based therapies, such as CAR T cells and checkpoint inhibitors, which may not be effective for all patients or can lead to resistance.
The trial focused on patients who had already undergone standard cancer treatments but remained at high risk of recurrence. The vaccine was well-tolerated, with no serious side effects reported. At the five-year follow-up, six of the 13 patients were still alive, and three of those six were tumor-free. The vaccine also demonstrated the ability to enhance the immune system’s response to cancer.
“Unlike tumor-associated antigens, neoantigens are not subject to central tolerance, meaning they can trigger a robust immune attack against cancer cells,” explained Dr. Bhardwaj.
The Mount Sinai team plans to conduct further studies with larger patient groups and explore the vaccine’s efficacy in combination with other cancer treatments. Based on the promising phase 1 results, three additional trials of PGV001 have been initiated, targeting glioblastoma, urothelial cancer (in combination with an immune checkpoint inhibitor), and prostate cancer.
The research underscores the potential of personalized cancer vaccines to revolutionize cancer treatment by leveraging the immune system to target individual tumor characteristics.
More information: Mansi Saxena et al, PGV001, a multi-peptide personalized neoantigen vaccine platform: Phase I study in patients with solid and hematological malignancies in the adjuvant setting, Cancer Discovery (2025). DOI: 10.1158/2159-8290.CD-24-0934
Disclaimer: This news article is based on information from a phase 1 clinical trial involving a small number of patients. Phase 1 trials primarily assess safety and dosage, and while the results are promising, they do not establish definitive efficacy. Larger, more comprehensive clinical trials are necessary to validate these findings and determine the long-term effectiveness of the PGV001 vaccine. The information provided should not be interpreted as medical advice. Individuals with cancer should consult with their healthcare providers to discuss appropriate treatment options.
