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针对亨廷顿病的静脉注射人类牙纸干细胞治疗的II期试验:一种随机,双盲,安慰剂控制的随机研究

Joyce Macedo Sanches Fernandes^1,2, Eduardo Pagani^3,4, Cristiane Valverde Wenceslau⁵

¹Faculdade de Medicina, Programa de Pós-Graduação Hospital das Clínicas, Universidade Estadual de Campinas (UNICAMP), Campinas, SP, 13083-872, Brazil. joycemacedosilva@gmail.com.

Stem cell research & therapy

|August 7, 2025

概括

人类牙纸干细胞疗法NestaCell®显著改善了亨廷顿病患者的运动和功能结果. 这种有前途的疗法显示出良好的安全性,因此需要进一步进行III期试验.

科学领域:

神经科学是一个神经科学.
再生医学是一种再生医学.
临床试验临床试验

背景情况:

亨廷顿病 (HD) 是一种致命的神经退行性疾病,目前没有疾病修饰治疗方法.
人类牙纸干细胞 (hDPSCs) 显示出神经保护和调节神经炎症的潜力.
一项II期试验评估了NestaCell®,一种全源的hDPSC疗法,用于HD患者.

研究的目的:

评估NestaCell®在亨廷顿病患者的安全性和有效性.
为了确定HDPSCs治疗效益的最佳剂量.
为了探索NestaCell®的潜在神经保护作用.

主要方法:

一个随机,双盲,安慰剂对照试验,涉及35名HD患者.
患者在11个月内接受了hDPSCs (1或200万细胞/公斤) 或安慰剂的静脉注射.
主要终点:统一亨廷顿病评分表 (UHDRS) 总动力得分 (TMS) 的变化;次要结果包括功能能力,胆和基于MRI的白质量定量.

主要成果:

与安慰剂相比,NestaCell®表现出良好的安全性,没有增加不良事件.
这两种hDPSC剂量都显著改善了UHDRS-TMS (p=0.005).
高剂量 (200万细胞/千克) 显著改善了UHDRS总功能能力 (TFC) (p=0.011),在胆和功能检查清单得分方面还有额外的好处. 核磁共振扫描显示了神经保护的趋势.

结论:

尼斯塔塞尔 (NestaCell®) 耐受性良好,并且在HD患者的运动和功能结果中显示出统计学上显著的改善.
预先的MRI数据表明潜在的神经保护作用,值得进一步调查.
这些发现支持将NestaCell®推进到III期试验,以更大规模的有效性和安全性确认.

关键词:

亨廷顿病就是亨廷顿病.干细胞是一种干细胞.统一的亨廷顿病评级表.

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