Aggeletopoulou Ioanna, Pantzios Spyridon, Triantos Christos
Division of Gastroenterology, Department of Internal Medicine, University Hospital of Patras, 26504 Patras, Greece.
Hepatogastroenterology Unit, Academic Department of Internal Medicine, General Oncology Hospital of Kifissia "Agioi Anargyroi", National and Kapodistrian University of Athens, 14564 Athens, Greece.
Cancers (Basel). 2025 Jan 23;17(3):376. doi: 10.3390/cancers17030376.
Hepatocellular carcinoma (HCC), the most prevalent form of primary liver cancer, presents significant therapeutic challenges due to its molecular complexity, late-stage diagnosis, and inherent resistance to conventional treatments. The intermediate to low mutational burden in HCC and its ability to evade the immune system through multiple mechanisms complicate the development of effective therapies. Recent advancements in immunotherapy, particularly neoantigen-based vaccines, offer a promising, personalized approach to HCC treatment. Neoantigens are tumor-specific peptides derived from somatic mutations in tumor cells. Unlike normal cellular antigens, neoantigens are foreign to the immune system, making them highly specific targets for immunotherapy. Neoantigens arise from genetic alterations such as point mutations, insertions, deletions, and gene fusions, which are expressed as neoepitopes that are not present in healthy tissues, thus evading the immune tolerance mechanisms that typically protect normal cells. Preclinical and early-phase clinical studies of neoantigen-based vaccines have shown promising results, demonstrating the ability of these vaccines to elicit robust T cell responses against HCC. The aim of the current review is to provide an in-depth exploration of the therapeutic potential of neoantigen-based vaccines in HCC, focusing on neoantigen identification, vaccine platforms, and their integration with immune checkpoint inhibitors to enhance immunogenicity. It also evaluates preclinical and clinical data on efficacy and safety while addressing challenges in clinical translation. By taking advantage of the unique antigenic profile of each patient's tumor, neoantigen-based vaccines represent a promising approach in the treatment of HCC, offering the potential for improved patient outcomes, long-term remission, and a shift towards personalized, precision medicine in liver cancer therapy.
肝细胞癌(HCC)是原发性肝癌最常见的形式,由于其分子复杂性、晚期诊断以及对传统治疗的固有抗性,带来了重大的治疗挑战。HCC中低到中等的突变负荷及其通过多种机制逃避免疫系统的能力,使有效疗法的开发变得复杂。免疫疗法的最新进展,特别是基于新抗原的疫苗,为HCC治疗提供了一种有前景的个性化方法。新抗原是源自肿瘤细胞体细胞突变的肿瘤特异性肽。与正常细胞抗原不同,新抗原对于免疫系统来说是外来的,这使其成为免疫疗法的高度特异性靶点。新抗原由点突变、插入、缺失和基因融合等基因改变产生,这些改变表达为健康组织中不存在的新表位,从而逃避通常保护正常细胞的免疫耐受机制。基于新抗原的疫苗的临床前和早期临床研究已显示出有前景的结果,证明了这些疫苗引发针对HCC的强大T细胞反应的能力。本综述的目的是深入探讨基于新抗原的疫苗在HCC中的治疗潜力,重点关注新抗原鉴定、疫苗平台及其与免疫检查点抑制剂的整合以增强免疫原性。它还评估了疗效和安全性的临床前和临床数据,同时解决临床转化中的挑战。通过利用每个患者肿瘤独特的抗原特征,基于新抗原的疫苗代表了一种有前景的HCC治疗方法,有望改善患者预后、实现长期缓解,并推动肝癌治疗向个性化、精准医学转变。