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神经影像学技术在神经退行性疾病基于细胞治疗的临床试验中的最新进展。

Recent Advances in Neuroimaging Techniques to Assist Clinical Trials on Cell-Based Therapies in Neurodegenerative Diseases.

机构信息

Neurodegeneration Imaging Group, University of Exeter Medical School, London, UK.

出版信息

Stem Cells. 2022 Aug 25;40(8):724-735. doi: 10.1093/stmcls/sxac039.

DOI:10.1093/stmcls/sxac039
PMID:35671344
Abstract

Neurodegenerative diseases, such as Alzheimer's disease (AD) and Parkinson's disease (PD), are progressive disorders for which curative therapy is still lacking. Cell-based therapy aims at replacing dysfunctional cellular populations by repairing damaged tissue and by enriching the microenvironment of selective brain areas, and thus constitutes a promising disease-modifying treatment of neurodegenerative diseases. Scientific research has engineered a wide range of human-derived cellular populations to help overcome some of the logistical, safety, and ethical issues associated with this approach. Open-label studies and clinical trials in human participants have used neuroimaging techniques, such as positron emission tomography (PET) and magnetic resonance imaging (MRI), to assess the success of the transplantation, to evaluate the functional integration of the implanted tissue into the host environment and to understand the pathophysiological changes associated with the therapy. Neuroimaging has constituted an outcome measure of large, randomized clinical trials, and has given answers to clarify the pathophysiology underlying some of the complications linked with this therapy. Novel PET radiotracers and MRI sequences for the staging of neurodegenerative diseases and to study alterations at the molecular level significantly expands the translational potential of neuroimaging to assist pre-clinical and clinical research on cell-based therapy in these disorders. This concise review summarizes the current use of neuroimaging in human studies of cell-based replacement therapy and focuses on the future applications of PET and MRI techniques to evaluate the pathophysiology and treatment efficacy, as well as to aid patient selection and as an outcome measure to improve treatment success.

摘要

神经退行性疾病,如阿尔茨海默病(AD)和帕金森病(PD),是一种进行性疾病,目前仍缺乏有效的治疗方法。基于细胞的治疗旨在通过修复受损组织和丰富选择性脑区的微环境来替代功能失调的细胞群体,因此是一种很有前途的神经退行性疾病的疾病修饰治疗方法。科学研究已经设计了多种人类来源的细胞群体,以帮助克服这种方法相关的一些后勤、安全和伦理问题。在人类参与者中进行的开放性研究和临床试验使用了神经影像学技术,如正电子发射断层扫描(PET)和磁共振成像(MRI),以评估移植的成功,评估植入组织与宿主环境的功能整合,并了解与治疗相关的病理生理变化。神经影像学已成为大型随机临床试验的结果衡量标准,并为阐明与该治疗相关的一些并发症的病理生理学提供了答案。新型 PET 放射性示踪剂和 MRI 序列用于神经退行性疾病的分期和研究分子水平的改变,极大地扩展了神经影像学在辅助这些疾病的基于细胞的治疗的临床前和临床研究中的转化潜力。这篇简明综述总结了神经影像学在人类基于细胞的替代治疗研究中的当前应用,并重点介绍了 PET 和 MRI 技术在评估病理生理学和治疗效果、辅助患者选择以及作为改善治疗效果的结果衡量标准方面的未来应用。

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Recent Advances in Neuroimaging Techniques to Assist Clinical Trials on Cell-Based Therapies in Neurodegenerative Diseases.神经影像学技术在神经退行性疾病基于细胞治疗的临床试验中的最新进展。
Stem Cells. 2022 Aug 25;40(8):724-735. doi: 10.1093/stmcls/sxac039.
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