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新型帕金森病干细胞治疗方法。

Novel Approach to Stem Cell Therapy in Parkinson's Disease.

机构信息

1 International Stem Cell Corporation , Carlsbad, California.

2 Royal Melbourne Hospital , Parkville, Australia .

出版信息

Stem Cells Dev. 2018 Jul 15;27(14):951-957. doi: 10.1089/scd.2018.0001.


DOI:10.1089/scd.2018.0001
PMID:29882481
Abstract

In this commentary we discuss International Stem Cell Corporation's (ISCO's) approach to developing a pluripotent stem cell based treatment for Parkinson's disease (PD). In 2016, ISCO received approval to conduct the world's first clinical study of a pluripotent stem cell based therapy for PD. The Australian regulatory agency Therapeutic Goods Administration (TGA) and the Melbourne Health's Human Research Ethics Committee (HREC) independently reviewed ISCO's extensive preclinical data and granted approval for the evaluation of a novel human parthenogenetic derived neural stem cell (NSC) line, ISC-hpNSC, in a PD phase 1 clinical trial ( ClinicalTrials.gov NCT02452723). This is a single-center, open label, dose escalating 12-month study with a 5-year follow-up evaluating a number of objective and patient-reported safety and efficacy measures. A total of 6 years of safety and efficacy data will be collected from each patient. Twelve participants are recruited in this study with four participants per single dose cohort of 30, 50, and 70 million ISC-hpNSC. The grafts are placed bilaterally in the caudate nucleus, putamen, and substantia nigra by magnetic resonance imaging-guided stereotactic surgery. Participants are 30-70 years old with idiopathic PD ≤13 years duration and unified PD rating scale motor score (Part III) in the "OFF" state ≤49. This trial is fully funded by ISCO with no economic involvement from the patients. It is worth noting that ISCO underwent an exhaustive review process and successfully answered the very comprehensive, detailed, and specific questions posed by the TGA and HREC. The regulatory/ethic review process is based on applying scientific and clinical expertise to decision-making, to ensure that the benefits to consumers outweigh any risks associated with the use of medicines or novel therapies.

摘要

在这篇评论中,我们讨论了国际干细胞公司(ISCO)开发基于多能干细胞的帕金森病(PD)治疗方法的方法。2016 年,ISCO 获得批准进行世界上首例基于多能干细胞的 PD 治疗临床研究。澳大利亚监管机构治疗商品管理局(TGA)和墨尔本健康人类研究伦理委员会(HREC)分别审查了 ISCO 的广泛临床前数据,并批准评估一种新型人类孤雌生殖衍生神经干细胞(NSC)系,ISC-hpNSC,在 PD 阶段 1 临床试验(ClinicalTrials.gov NCT02452723)中。这是一项单中心、开放标签、剂量递增的 12 个月研究,具有 5 年随访期,评估了多项客观和患者报告的安全性和疗效指标。每个患者将收集 6 年的安全性和疗效数据。本研究共招募 12 名参与者,每个单剂量队列 30、50 和 7000 万 ISC-hpNSC 招募 4 名参与者。通过磁共振成像引导立体定向手术将移植物双侧放置在尾状核、壳核和黑质中。参与者年龄在 30-70 岁之间,患有特发性 PD ≤13 年,统一 PD 评定量表运动评分(第三部分)在“OFF”状态下≤49。该试验由 ISCO 全额资助,患者无经济利益。值得注意的是,ISCO 经历了详尽的审查过程,并成功回答了 TGA 和 HREC 提出的非常全面、详细和具体的问题。监管/伦理审查过程基于将科学和临床专业知识应用于决策,以确保消费者的利益超过与使用药物或新型疗法相关的任何风险。

相似文献

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[3]
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Mechanical Forces Guide Axon Growth through the Nigrostriatal Pathway in an Organotypic Model.

Adv Sci (Weinh). 2025-8

[2]
Characteristics of Parthenogenetic Stem Cells and Their Potential Treatment Strategy for Central Nervous System Diseases.

Neuropsychiatr Dis Treat. 2025-2-3

[3]
Promising biomarkers and therapeutic targets for the management of Parkinson's disease: recent advancements and contemporary research.

Metab Brain Dis. 2023-3

[4]
From 2D to 3D: Development of Monolayer Dopaminergic Neuronal and Midbrain Organoid Cultures for Parkinson's Disease Modeling and Regenerative Therapy.

Int J Mol Sci. 2023-1-28

[5]
Challenges in the clinical advancement of cell therapies for Parkinson's disease.

Nat Biomed Eng. 2023-4

[6]
Emerging Therapeutic Strategies for Parkinson's Disease and Future Prospects: A 2021 Update.

Biomedicines. 2022-2-3

[7]
Induced Cognitive Impairments Reversed by Grafts of Neural Precursors: A Longitudinal Study in a Macaque Model of Parkinson's Disease.

Adv Sci (Weinh). 2022-4

[8]
Preclinical Assessment of Mesenchymal-Stem-Cell-Based Therapies in Spinocerebellar Ataxia Type 3.

Biomedicines. 2021-11-24

[9]
Stem Cell-Based Therapies: What Interventional Radiologists Need to Know.

Semin Intervent Radiol. 2021-11-24

[10]
Human Neural Stem Cells for Cell-Based Medicinal Products.

Cells. 2021-9-9

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