Ricklin Daniel, Lambris John D
Department of Pathology and Laboratory Medicine, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA.
Nat Biotechnol. 2007 Nov;25(11):1265-75. doi: 10.1038/nbt1342.
The complement system is a central component of innate immunity and bridges the innate to the adaptive immune response. However, it can also turn its destructive capabilities against host cells and is involved in numerous diseases and pathological conditions. Modulation of the complement system has been recognized as a promising strategy in drug discovery, and a large number of therapeutic modalities have been developed. However, successful marketing of complement-targeted drugs has proved to be more difficult than initially expected, and many strategies have been discontinued. The US Food and Drug Administration's approval of the first complement-specific drug, an antibody against complement component C5 (eculizumab; Soliris), in March 2007, was a long-awaited breakthrough in the field. Approval of eculizumab validates the complement system as therapeutic target and might facilitate clinical development of other promising drug candidates.
补体系统是固有免疫的核心组成部分,它在固有免疫和适应性免疫反应之间起桥梁作用。然而,它也可能将其破坏能力转向宿主细胞,并参与多种疾病和病理状况。补体系统的调节已被公认为药物研发中的一种有前景的策略,并且已经开发出大量治疗方法。然而,事实证明,以补体为靶点的药物成功上市比最初预期的要困难,许多策略已被终止。2007年3月,美国食品药品监督管理局批准了首个补体特异性药物,一种针对补体成分C5的抗体(依库珠单抗;Soliris),这是该领域期待已久的突破。依库珠单抗的获批证实了补体系统作为治疗靶点的地位,并可能促进其他有前景的候选药物的临床开发。