Fang Yu-Ting, Yang Wen-Wei, Niu Ya-Ru, Sun Yong-Kun
Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing 100021, China.
Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Hebei Cancer Hospital, Chinese Academy of Medical Sciences, Langfang 065001, Hebei Province, China.
World J Gastrointest Oncol. 2023 Apr 15;15(4):571-595. doi: 10.4251/wjgo.v15.i4.571.
Pancreatic adenocarcinoma (PDAC) is a fatal disease with a 5-year survival rate of 8% and a median survival of 6 mo. In PDAC, several mutations in the genes are involved, with Kirsten rat sarcoma oncogene (90%), cyclin-dependent kinase inhibitor 2A (90%), and tumor suppressor 53 (75%-90%) being the most common. Mothers against decapentaplegic homolog 4 represents 50%. In addition, the self-preserving cancer stem cells, dense tumor microenvironment (fibrous accounting for 90% of the tumor volume), and suppressive and relatively depleted immune niche of PDAC are also constitutive and relevant elements of PDAC. Molecular targeted therapy is widely utilized and effective in several solid tumors. In PDAC, targeted therapy has been extensively evaluated; however, survival improvement of this aggressive disease using a targeted strategy has been minimal. There is currently only one United States Food and Drug Administration-approved targeted therapy for PDAC - erlotinib, but the absolute benefit of erlotinib in combination with gemcitabine is also minimal (2 wk). In this review, we summarize current targeted therapies and clinical trials targeting dysregulated signaling pathways and components of the PDAC oncogenic process, analyze possible reasons for the lack of positive results in clinical trials, and suggest ways to improve them. We also discuss emerging trends in targeted therapies for PDAC: combining targeted inhibitors of multiple pathways. The PubMed database and National Center for Biotechnology Information clinical trial website (www.clinicaltrials.gov) were queried to identify completed and published (PubMed) and ongoing (clinicaltrials.gov) clinical trials (from 2003-2022) using the keywords pancreatic cancer and targeted therapy. The PubMed database was also queried to search for information about the pathogenesis and molecular pathways of pancreatic cancer using the keywords pancreatic cancer and molecular pathways.
胰腺腺癌(PDAC)是一种致命疾病,5年生存率为8%,中位生存期为6个月。在PDAC中,多个基因发生突变,其中最常见的是 Kirsten 大鼠肉瘤癌基因(90%)、细胞周期蛋白依赖性激酶抑制剂2A(90%)和肿瘤抑制因子53(75%-90%)。母亲对果蝇同源基因4的缺失占50%。此外,PDAC中自我保护的癌症干细胞、致密的肿瘤微环境(纤维组织占肿瘤体积的90%)以及抑制性和相对耗竭的免疫微环境也是PDAC的构成要素和相关因素。分子靶向治疗在多种实体瘤中得到广泛应用且效果显著。在PDAC中,靶向治疗已得到广泛评估;然而,采用靶向策略改善这种侵袭性疾病的生存率收效甚微。目前美国食品药品监督管理局仅批准了一种用于PDAC的靶向治疗药物——厄洛替尼,但厄洛替尼联合吉西他滨的绝对获益也很小(2周)。在本综述中,我们总结了目前针对PDAC致癌过程中失调的信号通路和成分的靶向治疗及临床试验,分析了临床试验缺乏阳性结果的可能原因,并提出改进方法。我们还讨论了PDAC靶向治疗的新趋势:联合多种途径的靶向抑制剂。通过查询PubMed数据库和美国国立生物技术信息中心临床试验网站(www.clinicaltrials.gov),使用关键词“胰腺癌”和“靶向治疗”来识别已完成并发表(PubMed)以及正在进行(clinicaltrials.gov)的临床试验(2003年至2022年)。还通过查询PubMed数据库,使用关键词“胰腺癌”和“分子途径”来搜索有关胰腺癌发病机制和分子途径的信息。