在前列素相关的前性痴呆症中对latozinemab的第一阶段研究
Michael Ward1, Lawrence P Carter1, Julie Y Huang1
1Alector Inc. South San Francisco California USA.
Alzheimer's & dementia (New York, N. Y.)
|February 15, 2024
概括
新型疗法拉托津涅马布有效增加了GRN相关的前性痴呆症 (FTD) 患者的大脑和血液中的progranulin (PGRN) 水平. 该药在最初的人类试验中显示出有前途的安全性,支持FTD-GRN的进一步开发.
科学领域:
- 神经科学是一个神经科学.
- 遗传学 遗传学 是一个
- 药理学 药理学是指药理学的学科.
背景情况:
- 在GRN基因的异合突变降低了progranulin (PGRN) 水平,导致高透率的前性痴呆症 (FTD).
- 拉托齐内马布是一种单克隆抗体,旨在提高具有GRN突变的FTD患者的PGRN水平.
研究的目的:
- 评估Latozinemab在FTD-GRN患者中的安全性,耐受性,药理动力学 (PK) 和药理动力学 (PD).
- 评估拉托齐内马布在血和脑脊液 (CSF) 中增加PGRN水平的疗效.
主要方法:
- 一项首次在人身上进行的第一阶段研究涉及多次静脉注射的拉托齐尼马布.
- 招募了8名患有异构性功能丧失GRN突变 (FTD-GRN) 引起的FTD的症状参与者.
- 评估了安全性,耐受性,PK和PD参数.
主要成果:
- 拉托齐内马布表现出良好的安全性和PK/PD概况.
- 多次剂量拉托齐内马布成功增加了血和脑液中的PGRN水平.
- 参与者的PGRN水平接近健康志愿者观察到的水平.
结论:
- 第1阶段数据表明,拉托齐内马布是FTD-GRN的潜在治疗剂.
- 需要对FTD-GRN进行进一步的临床开发.
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