Hepatocellular carcinoma (HCC) is the most common form of primary liver cancer and is a leading cause of cancer-related death worldwide. Despite recent advancements in systemic therapy for advanced HCC, the 5-year survival rate remains <10%. One of the newest treatment options for HCC is immunotherapy — a treatment using a person’s own immune system to fight the cancer. However, past studies show that only 15–20% of HCC diagnoses respond to immunotherapy and about 30% may be resistant.
In the study, researchers recruited 36 participants for the clinical trial. All participants received the combination of a personalized DNA vaccine, GNOS-PV02, and pembrolizumab.
At the end of the study, researchers found that almost one-third of participants experienced tumor shrinkage, which is about twice as many people seen in studies of immunotherapy alone for HCC. Additionally, about 8% of the study participants had no evidence of a tumor after receiving the combination treatment.
“The response rate on this study is high enough that I think it’s unlikely that the pembrolizumab alone did this and it supports the idea that the vaccine contributed to the efficacy observed. I think it’s also notable that the response rate was higher than pembrolizumab alone without a major increase in toxicity” said Mark Yarchoan, lead study author.
While further research is needed to validate these findings and optimize treatment protocols, the results of this phase 1/2 trial represent a significant step forward in the quest for more effective therapies for advanced hepatocellular carcinoma. With continued advancements in personalized immunotherapy, there is hope for transforming the landscape of cancer care and improving outcomes for patients facing this devastating disease.
Reference: Yarchoan, M., Gane, E.J., Marron, T.U. et al. Personalized neoantigen vaccine and pembrolizumab in advanced hepatocellular carcinoma: a phase 1/2 trial. Nat Med (2024). https://doi.org/10.1038/s41591-024-02894-y
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