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美国食品药品监督管理局(FDA)批准用于血液系统恶性肿瘤的药物——过去十年回顾

FDA-Approved Drugs for Hematological Malignancies-The Last Decade Review.

作者信息

Sochacka-Ćwikła Aleksandra, Mączyński Marcin, Regiec Andrzej

机构信息

Department of Organic Chemistry and Drug Technology, Faculty of Pharmacy, Wroclaw Medical University, 211A Borowska Street, 50-556 Wroclaw, Poland.

出版信息

Cancers (Basel). 2021 Dec 24;14(1):87. doi: 10.3390/cancers14010087.

Abstract

Hematological malignancies, also referred to as blood cancers, are a group of diseases involving abnormal cell growth and persisting in the blood, lymph nodes, or bone marrow. The development of new targeted therapies including small molecule inhibitors, monoclonal antibodies, bispecific T cell engagers, antibody-drug conjugates, recombinant immunotoxins, and, finally, Chimeric Antigen Receptor T (CAR-T) cells has improved the clinical outcomes for blood cancers. In this review, we summarized 52 drugs that were divided into small molecule and macromolecule agents, approved by the Food and Drug Administration (FDA) in the period between 2011 and 2021 for the treatment of hematological malignancies. Forty of them have also been approved by the European Medicines Agency (EMA). We analyzed the FDA-approved drugs by investigating both their structures and mechanisms of action. It should be emphasized that the number of targeted drugs was significantly higher (46 drugs) than chemotherapy agents (6 drugs). We highlight recent advances in the design of drugs that are used to treat hematological malignancies, which make them more effective and less toxic.

摘要

血液系统恶性肿瘤,也被称为血癌,是一组涉及异常细胞生长且在血液、淋巴结或骨髓中持续存在的疾病。包括小分子抑制剂、单克隆抗体、双特异性T细胞衔接器、抗体药物偶联物、重组免疫毒素,以及最后出现的嵌合抗原受体T(CAR-T)细胞在内的新型靶向疗法的发展,改善了血癌的临床治疗效果。在本综述中,我们总结了2011年至2021年间美国食品药品监督管理局(FDA)批准用于治疗血液系统恶性肿瘤的52种药物,这些药物分为小分子和大分子药物。其中40种也已获得欧洲药品管理局(EMA)的批准。我们通过研究FDA批准药物的结构和作用机制对其进行了分析。应该强调的是,靶向药物的数量(46种药物)显著高于化疗药物(6种药物)。我们重点介绍了用于治疗血液系统恶性肿瘤的药物设计方面的最新进展,这些进展使药物更有效且毒性更低。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e783/8750348/f46793813112/cancers-14-00087-g001.jpg

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