NYC Gazette

Researchers at the Icahn School of Medicine at Mount Sinai have conducted a phase 1 trial on a personalized cancer vaccine, PGV001. Led by Dr. Nina Bhardwaj, the study involved a small group of patients and aimed to enhance cancer treatment by leveraging the immune system. The findings were published in Cancer Discovery.

The PGV001 vaccine is designed to be personalized for each patient’s unique cancer by targeting neoantigens, which are specific mutations in cancer cells not present in healthy cells. This approach seeks to improve outcomes for patients who do not respond well to existing immune-based therapies or develop resistance over time.

Dr. Bhardwaj stated, “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.” The trial included 13 patients with various cancers, such as non-small cell lung cancer and breast cancer, who had already undergone standard treatments but were still at high risk of recurrence.

The vaccine was developed using advanced tools to identify promising neoantigens for each patient, optimizing immune activation without causing serious side effects. At a five-year follow-up, six out of 13 treated patients survived, with three remaining tumor-free.

Mount Sinai scientists plan further studies on PGV001 in larger patient groups and its efficacy when combined with other treatments. New trials include those for glioblastoma and prostate cancer.

This research received support from several organizations including the National Cancer Institute and the Parker Institute of Cancer Immunotherapy. Details about contributors and industry connections are available in the full research report.