Custom-Made Vaccine Combo Proves Effective in Bladder Cancer

Treatment with a custom-made vaccine and immune checkpoint inhibitor safely triggered a strong immune response in patients with bladder cancer, showing potential for wider use.

Treatment with an immune checkpoint inhibitor in combination with the PGV001 vaccine, a custom-made vaccine, created by researchers at the Icahn School of Medicine at Mount Sinai, was found to be safe and feasible for patients with bladder cancer, eliciting a strong immune response, according to a news release shared by Mount Sinai.

The PGV001 vaccine, when combined with immunotherapy, created only mild injection-site reactions, making it a safe and well tolerated approach. Importantly, a vaccine-specific T cell response not seen prior to treatment was observed in all patients who were included on the study. Based on these outcomes, investigators shared that this personalized vaccine approach demonstrated feasibility for both individuals with metastatic bladder cancer, as well as patients undergoing therapy after surgery.

This phase 1 study is launching as excitement continues to build around personalized cancer vaccines, adding to the growing evidence that personalized vaccines could make current cancer treatments that serve as the standard of care safer and more effective. This investigation is being led by Dr. Nina Bhardwaj and Dr. Matthew Galsky.

"Our findings move the field forward by showing how personalized vaccines like PGV001 can work in bladder cancer," Bhardwaj stated in the news release. "We’ve proven these custom vaccines can consistently activate the immune system in powerful, cancer-fighting ways."

Bhardwaj is a professor of Medicine (Hematology and Medical Oncology) and Urology; the director of Immunotherapy; medical director of the Vaccine and Cell Therapy Laboratory; co-director of the Cancer Immunology Program at The Tisch Cancer Institute; and a faculty member of the Icahn Genomics Institute, Mount Sinai Health.

Diving Deeper into this Investigation

Approximately 84,870 new cases of bladder cancer and 17,420 deaths due to the disease will occur in the United States this year, according to the American Cancer Society, highlighting the need for new therapies. Although patients with bladder cancer respond to immune-based treatments, there are some patients who do not benefit from an immunotherapy-alone approach, prompting researchers to investigate options to fill this gap in care.

PGV001 is engineered to target the mutations found within each patient’s tumor. By utilizing advanced tumor sequencing and a specialized platform, researchers identify specific tumor alterations that are likely to trigger an immune response. A personalized vaccine is then created using synthetic peptides that correspond to these markers. Administered alongside the immunotherapy drug Tecentriq (atezolizumab), PGV001 helps directly destroy cancer cells while sparing healthy cells.

The vaccine is showing encouraging outcomes across many cancer types, including in bladder cancer, and holds potential for wider use. Encouraged by these initial results, investigators are advancing development efforts, aiming to explore new combination strategies to benefit a larger patient population. This individualized immunotherapy treatment represents a new option for patients, particularly those with tumors unresponsive to existing therapies.

"This study adds to growing evidence that combining personalized vaccines with immunotherapy could help many more patients benefit," Galsky, added. "It also lays the groundwork for larger trials that are now underway."

Galsky is a professor of medicine (Hematology and Medical Oncology); director of Genitourinary Medical Oncology; co-director of the Center of Excellence for Bladder Cancer at The Tisch Cancer Institute; and associate director for Translational Research at The Tisch Cancer Institute.

Notably, the news release shared that this research brough a collaborative team of experts in immunology, oncology, genetics and pathology together at Mount Sinai. These contributors included Dr. Mansi Saxena, Dr. Jonathan Anker, Dr. Julia Kodysh, Dr. Anna Kaminska and others.

Supporters of the study includes The National Cancer Institute of the National Institutes of Health, the Cancer Research Institute, the Parker Institute of Cancer Immunotherapy, and industry partners.

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