药物开发 药物开发
1University of Queensland, Brisbane, Australia.
Alzheimer's & dementia : the journal of the Alzheimer's Association
|December 26, 2025
概括
新的阿尔茨海默病 (AD) 疗法结合了新的抗tau抗体和聚焦超声波. 这种方法在恢复蛋白质稳定和改善小鼠模型内存方面表现有希望,早期的人体试验证明了安全性.
科学领域:
- 神经科学是一个神经科学.
- 免疫治疗是一种免疫疗法.
- 医疗器械 医疗器械
背景情况:
- 目前的阿尔茨海默氏病 (AD) 疗法提供有限的症状缓解.
- 已批准的抗粉样β抗体需要进一步验证现实世界的有效性.
- 对于阿尔茨海默病,迫切需要替代治疗策略.
研究的目的:
- 探索阿尔茨海默病 (AD) 的补充治疗策略.
- 针对粉样蛋白和沉积,以及相关的认知障碍.
- 开发新的抗tau免疫疗法,并利用低强度超声波用于药物输送和神经调节.
主要方法:
- 开发并测试了一种新的抗陶抗体 (RNJ1) 与基准抗体 (HJ8.5) 相对应.
- 在粉样蛋白沉积小鼠模型 (APP23) 中评估扫描超声波 (SUS) 的疗效.
- 开发了针对纳米体的JAM-1,以促进血液-大脑屏障 (BBB) 开放药物输送.
- 制造了一种经过ISO13485认证的研究设备 (UltraThera Pilot),并对阿尔茨海默病患者进行了安全试验.
主要成果:
- 新型抗陶抗体RNJ1在体外和体内表现优于HJ8.5.
- 蛋白质稳定性恢复被确定为tau免疫治疗的新型疗效指标.
- 在没有BBB开放的扫描超声波 (SUS) 改善了APP23小鼠的记忆缺陷,但没有降低粉样β负担.
- 在12名阿尔茨海默症患者的安全性试验中,证明了试验装置的安全性,耐受性和可行性.
结论:
- 阿尔茨海默病 (AD) 的认知改善可能与粉样蛋白β (Aβ) 清除脱离.
- 这些发现对设计未来基于超声波的AD治疗试验有影响.
- 确定了一种评估针对大脑的免疫疗法的新指标.
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