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JCO：淀粉样变性新药NEOD001初步临床试验结果

2016-02-09 MedSci MedSci原创

淀粉样变性单抗
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 系统性淀粉样变性是由于组织器官内变性的、错误折叠的蛋白质积累所导致的。淀粉样蛋白聚集可以影响到多种器官，包括心脏、肾脏、肝脏、软组织、神经系统和胃肠道。这严重影响到了器官结构和功能，但由于症状轻微往往导致诊断不够及时。NEOD001是一种以这些错误折叠的蛋白质为靶点的单克隆抗体。目前，NEOD001正在进行临床I/II期试验，用于治疗轻链淀粉样变性。 受试者在参与试验时已经至

系统性淀粉样变性是由于组织器官内变性的、错误折叠的蛋白质积累所导致的。淀粉样蛋白聚集可以影响到多种器官，包括心脏、肾脏、肝脏、软组织、神经系统和胃肠道。这严重影响到了器官结构和功能，但由于症状轻微往往导致诊断不够及时。NEOD001是一种以这些错误折叠的蛋白质为靶点的单克隆抗体。目前，NEOD001正在进行临床I/II期试验，用于治疗轻链淀粉样变性。

受试者在参与试验时已经至少完成过一场抗血清的细胞导向性治疗。受试者有部分或者更好的血液学反应并且有持续性的器官功能失调。受试者每28天接受一次NEOD001静脉注射。剂量分别是0.5、1、2、4、8、16和24mg/kg。主要目的是确定最大的耐受剂量和推荐剂量用于进一步研究，并且还要评价药物的安全性和耐受性。次要的目的包括了药代动力学性质、免疫原性和器官反应。27名受试者被分为7个队列。试验中没有出现严重的药物相关的不良反应，也没有出现由于药物相关的不良反应导致的试验终止。试验中也没有剂量限制性毒性或者抗药物抗体。最为常见的不良反应有疲劳、上呼吸道感染、咳嗽和呼吸困难。14个心脏可评估的患者中，有八人达到了心脏反应的条件，六人病情稳定。

这项研究表明，每月一次给药的NEOD001是安全的，且良好耐受。未来推荐的剂量为24mg/kg。目前二期临床扩大试验正在进行中。全球范围内的三期临床也已经发起。

原始出处：
Morie A. Gertz et al. First-in-Human Phase I/II Study of NEOD001 in Patients With Light Chain Amyloidosis and Persistent Organ Dysfunction. JCO. Published Ahead of Print on February 8, 2016 as 10.1200/JCO.2015.63.6530

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    2016-05-21 lidong40

    #JCO#

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    2016-02-11 xjy03

    #淀粉样变#

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    2016-02-11 zhouqu_8

    #淀粉样变性#

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    2016-02-11 huangdf

    #变性#

    0

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