Hepatocellular carcinoma (HCC) is the most common form of liver cancer, accounting for more than 80% of cases. Immunotherapy is a new treatment option for HCC, but not everyone responds to it. Researchers from Johns Hopkins Kimmel Cancer Center found that combining immunotherapy with a personalized anti-tumor vaccine increased tumor shrinkage compared to immunotherapy alone. Liver cancer is the sixth most common cancer in the world, with an estimated 905,700 people diagnosed in 2020. This number is expected to rise to 1.4 million by 2040. HCC, being the most common type of liver cancer, is difficult to treat, and only a small percentage of patients respond to immunotherapy.

The preliminary clinical trial for the personalized DNA vaccine GNOS-PV02 aimed to educate the immune system to recognize antigens present in the cancer, so the immune system can better attack cancer cells. The vaccine is personalized for each individual patient based on their unique cancer DNA mutations. GNOS-PV02 was used in combination with the immunotherapy drug pembrolizumab, and participants in the study experienced twice the rate of tumor shrinkage compared to those receiving immunotherapy alone. The FDA approved pembrolizumab for HCC treatment in 2018. Mark Yarchoan, MD, lead study author, noted that past cancer vaccines failed due to targeting non-specific antigens and using them against advanced cancers without other immunotherapy.

Researchers recruited 36 participants for the clinical trial, and almost one-third of them experienced tumor shrinkage, twice as many as seen in studies of immunotherapy alone for HCC. Additionally, about 8% had no evidence of a tumor after receiving the combination treatment. Larger randomized studies are needed to confirm the efficacy of personalized cancer vaccines and define the optimal treatment sequence for their use. Anton Bilchik, MD, PhD, chief of medicine at Providence Saint John’s Cancer Institute, was impressed by the results of the study. He highlighted the significance of the personalized vaccine in doubling the response to immunotherapy, especially for patients who have not responded to first-line treatments or are not candidates for surgery or transplantation.

Martin Gutierrez, MD, director of Phase I research at the John Theurer Cancer Center at Hackensack University Medical Center, also found the results of the study encouraging. He suggested conducting a larger Phase II trial in first-line therapy to further evaluate the effectiveness of personalized cancer vaccines. Personalized cancer vaccines are seen as the future of cancer treatment, as they use the patient’s own tumor biopsy cells to identify mutations and predict which genes can be recognized by the immune system. This approach combines advanced technology and artificial intelligence to enhance treatment outcomes for patients with difficult-to-treat cancers like HCC. The integration of personalized vaccines with existing treatment options like immunotherapy holds promise for improving patient responses and outcomes in the fight against liver cancer.

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