全球肝癌新药研发临床试验进展总结  

Progress summary clinical trials of new drugs for liver cancer

作　　者：孙嘉阳 胡威 唐玉 Sun Jiayang;Hu Wei;Tang Yu(Zunyi Medical University,Zunyi 563006,Guizhou,China;Department of Oncology,Hospital of Zunyi Medical University,Early Clinical Research Ward,Hospital of Zunyi Medical University,Zunyi 563000,Guizhou,China;National Cancer Center/National Clinical Research Center for Cancer/Drug Clinical Trial Research Center of Cancer Hospital,Peking Union Medical College,Chinese Academy of Medical Sciences,Beijing 100021,China)

机构地区：[1]遵义医科大学,贵州遵义563006 [2]遵义医科大学附属医院肿瘤科,遵义医科大学附属医院早期临床研究病房,贵州遵义563000 [3]国家癌症中心/国家肿瘤临床医学研究中心/中国医学科学院北京协和医学院肿瘤医院药物临床试验研究中心,北京100021

出　　处：《肝癌电子杂志》2024年第4期1-5,I0001,共6页Electronic Journal of Liver Tumor

摘　　要：目的:总结2020—2024年6月全球肝癌药物注册临床试验开展情况及发展趋势。方法:基于英国Informa公司旗下的Pharma Intelligence开发的Trialtrove数据库,提取2020—2024年全球范围启动的肝癌药物临床试验及涉及试验药物的相关信息,分析肝癌临床研究的年度增长情况、试验、药物特征及其相关因素。结果:研究统计时间段内,全球共开展了356个肝癌药物注册临床试验,占全部肿瘤治疗药物临床试验的4.3%,其中由国内企业发起189个(53.1%)。肝癌临床试验启动数量在2022年达到顶峰(101个)。在试验分期方面,Ⅰ期临床试验(含Ⅰ、Ⅱ期)是最常见的临床试验分期类型(266个,74.7%);在试验适应证方面,针对晚期肝癌的药物研发所占的比例最高(324个,91.0%)。在所有试验药物中,所涉及的药物类型主要包括抗体类药物(48.0%)、小分子靶向药物(25.4%)、细胞基因类药物(15.4%)和抗体耦联药物(5.0%)。32个临床试验针对初治的不可手术或局部治疗的晚期肝癌的临床试验中,探索的主要试验性治疗方式是以免疫检查点抑制剂治疗为基础的药物联合治疗;针对晚期≥2线治疗患者开展的药物临床试验共262个,其中32个(12.2%)在免疫治疗失败的晚期肝癌患者中开展,目前均处于Ⅰ、Ⅱ期试验阶段,涉及的试验药物类型较为多样。结论:全球肝癌药物临床开发较为活跃,国内企业研发药物在其中发挥重要作用。在肝癌中,免疫治疗是最为活跃的药物研发领域;在后线肝癌临床试验中,细胞基因治疗也是重要的临床研究领域。建议国内企业加大针对肝癌新药研发投入,挖掘更多新靶点和新机制作用药物,拓宽肝癌治疗的可能方向。Objective:To summarize the development trend of global clinical trials of primary hepatic cancer(HCC)from 2020 to June 2024.Methods:Based on the Trialtrove database developed by Informa Pharma Intelligence(London,UK),the clinical trials of liver cancer and the related medicines launched from 2020 to 2024 were extracted.The annual growth rate,the characteristics of trials and tested products were analyzed.Results:During the statistical period,a total of 356 clinical trials for HCC were carried out worldwide,accounting for 4.3%of all anti-cancer clinical trials.Among them,189(53.1%)were initiated by domestic companies.The number of clinical trials for HCC peaked in 2022(101 trials).Phase I(including phaseⅠ/Ⅱ)clinical trials were the most common trial phase type(266 trials,74.7%).In terms of trial indications,drug development for advanced HCC accounted for the highest proportion(324 trials,91.0%).Among all the investigational drugs,the types of drugs involved mainly included antibody drugs(48.0%),small molecule targeted drugs(25.4%),cell and gene therapy(15.4%)and antibody-drug conjugate(5.0%).Thirty-two clinical trials focused on first line treatment for late-stage HCC.Among them,the main investigational treatment modality was combined immunotherapy based on immune checkpoint inhibitors.A total of 262 clinical trials were conducted for patients with advanced HCC progressed after previous treatment,of which 32 trials(12.2%)were carried out in patients failed immunotherapy.All the 32 trials were in phaseⅠ/Ⅱtrials,involving a variety of experimental drug types.Conclusions:The clinical development of liver cancer drugs is active in the world,and domestic enterprises play an important role.In liver cancer,immunotherapy is the most active field of drug development.While in the clinical trials including heavily treated HCC patients,cell and gene therapy is also an important clinical research area.It is suggested that domestic enterprises should increase investment in the development of novel drugs for liver cancer

关 键 词：肝癌 药物 临床试验 免疫治疗 细胞基因治疗 

分 类 号：R73[医药卫生—肿瘤]