Gencer Baris, Laaksonen Reijo, Buhayer Aliki, Mach François
Cardiology Division, Geneva University Hospitals, Switzerland.
Tampere University Hospital, Finland.
Swiss Med Wkly. 2015 Nov 2;145:w14179. doi: 10.4414/smw.2015.14179. eCollection 2015.
Biotechnological advances now enable the design of fully human antibodies to target specific antigens in a growing number of diseases. Monoclonal antibodies (mAbs) differ from traditional small chemical molecules in several ways: (1) biological production ‒ they are grown in and extracted from cell cultures; (2) specificity ‒ they demonstrate high target specificity, with a low risk of drug-drug interactions; (3) administration ‒ they are delivered parenterally (intravenously or subcutaneously); (4) dosage interval ‒ their extended half-lives generally allow for spaced dosing (from weekly to monthly). In cardiology, fully human mAbs directed against proprotein convertase subtilisin / kexin type 9 (PCSK9) have shown to be effective in reducing low-density lipoprotein cholesterol (LDL-C) in phase II clinical trials among patients with familial hypercholesterolaemia (FH). PCSK9 inhibitors have just received approval for the treatment of FH and clinical atherosclerotic disease, and patients not at target under maximally tolerated statin therapy or intolerant to statins. Large-scale phase III trials are currently assessing the role of PCSK9 inhibitors in the secondary prevention setting for patients with acute coronary syndromes (ACS) and poorly controlled LDL-C under evidence-based therapies. Another area currently under investigation for fully human mAbs in secondary prevention is their potential ability to inhibit inflammatory pathways. In this context, canakinumab, a specific mAb inhibiting interleukin-1β (IL-1β), has already received approval for the treatment of systemic juvenile idiopathic arthritis. The canakinumab anti-inflammatory thrombosis outcomes trial (CANTOS) is an ongoing trial assessing whether inhibition of IL-1β could reduce the occurrence of cardiovascular adverse events in 17,200 patients with ACS and with defined persisting inflammation.
生物技术的进步如今使得设计出完全人源化抗体成为可能,这些抗体可针对越来越多疾病中的特定抗原。单克隆抗体(mAb)在几个方面不同于传统的小分子化学药物:(1)生物生产——它们在细胞培养物中生长并从中提取;(2)特异性——它们具有高度的靶点特异性,药物相互作用风险低;(3)给药方式——通过肠胃外途径(静脉内或皮下)给药;(4)给药间隔——它们较长的半衰期通常允许间隔给药(从每周一次到每月一次)。在心脏病学领域,针对前蛋白转化酶枯草杆菌蛋白酶/kexin 9型(PCSK9)的完全人源化单克隆抗体在家族性高胆固醇血症(FH)患者的II期临床试验中已显示出可有效降低低密度脂蛋白胆固醇(LDL-C)。PCSK9抑制剂刚刚获批用于治疗FH和临床动脉粥样硬化疾病,以及在最大耐受剂量他汀类药物治疗下未达目标或对他汀类药物不耐受的患者。大规模III期试验目前正在评估PCSK9抑制剂在急性冠状动脉综合征(ACS)患者二级预防中的作用,以及在循证治疗下低密度脂蛋白胆固醇控制不佳的情况。完全人源化单克隆抗体在二级预防中目前正在研究的另一个领域是其抑制炎症途径的潜在能力。在此背景下,卡那单抗,一种抑制白细胞介素-1β(IL-1β)的特异性单克隆抗体,已获批用于治疗全身型幼年特发性关节炎。卡那单抗抗炎血栓形成结局试验(CANTOS)是一项正在进行的试验,评估抑制IL-1β是否可减少17200例ACS且存在明确持续性炎症患者发生心血管不良事件的情况。