Personalized Cancer Vaccine Generates Responses Across Cancer Types

PGV001 induced immune responses across various cancer types like breast and urothelial cancer, as well as multiple myeloma: © stock.adobe.com

PGV001, a personalized multi-peptide neoantigen cancer vaccine, induced a strong immune response in patients with non-small cell lung cancer, head and neck cancer, urothelial cancer, breast cancer and multiple myeloma, according to early findings from a phase 1 trial. Data from the trial were simultaneously shared in a news release and published in Cancer Discovery, a journal of the American Association for Cancer Research.

Among the 13 patients treated with PGV001 across five different cancers, positive outcomes were observed, and 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 them remained tumor-free, providing early evidence of PGV001’s potential long-term benefits.

“We wanted to develop cancer vaccines that can stop cancer from coming back in patients who are at high risk of recurrence. This study shows that making personalized cancer vaccines is possible and safe,” Dr. Nina Bhardwaj said in the news release. “This is a phase 1 study with a small group of patients [13 patients] with a variety of cancers [non-small cell lung cancer, head and neck cancer, urothelial cancer, breast cancer and multiple myeloma], but it’s an exciting step toward using the immune system to help people live cancer-free, longer.”

Bhardwaj currently serves as the lead researcher of the investigation, as well as the Ward-Coleman Chair in Cancer Research and director of the Vaccine and Cell Therapy Laboratory at the Icahn School of Medicine at Mount Sinai, in New York.

This study investigates personalized cancer vaccines that target patient-specific neoantigens — proteins capable of triggering an immune response against cancer. Results indicate that these tailored vaccines can generate a strong immune response, with early data indicating a possible link to improved long-term survival. PGV001 — a tailored vaccine — was designed using a relatively small number of antigens compared with other recent trials and stimulated a robust immune response across multiple cancer types.

How Does PGV001 Work? More on the Phase 1 Study

PGV001 helps the body’s immune system recognize and attack cancer cells in order to stop the disease from coming back, and can be made to fit each patient’s unique cancer. Scientists use advanced technology to identify neoantigens — tiny changes in cancer cells — that are not found in healthy cells. Unlike tumor-associated antigens, neoantigens are more likely to trigger a strong immune response because they are not subject to central tolerance, meaning the immune system sees them as threats.

In recent years, immune-based therapies have changed cancer care with treatment like CAR T-cell therapy, bi-specific antibodies, antibody-drug conjugates and immune checkpoint inhibitors. While these approaches have improved outcomes, some patients do not respond or develop resistance. Personalized cancer vaccines like PGV001 aim to address these challenges by teaching the immune system to target cancer-specific mutations more effectively.

This study evaluated patients who had already undergone standard cancer treatments but remained at high risk of recurrence. Using a specialized platform, researchers analyzed tumor and normal DNA to identify neoantigens for each patient. The PGV001 vaccine was then designed with selected peptides to enhance immune activation.

PGV001 will continue to be evaluated in larger groups of patients by Mount Sinai scientists, who will also test how the vaccine works with other cancer treatments, the news release explained. Additionally, these data from the phase 1 study have prompted three additional PGV001 trials: one in newly diagnosed glioblastoma, one in urothelial cancer in combination with an immune checkpoint inhibitor and one in prostate cancer.

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