Stem Cell Clinical Research Center, The First Affiliated Hospital of Dalian Medical University, No. 193, Lianhe Road, Shahekou District, Dalian, 116011, Liaoning, China.
Dalian Innovation Institute of Stem Cell and Precision Medicine, Dalian, Liaoning, China.
Stem Cell Res Ther. 2023 Feb 9;14(1):23. doi: 10.1186/s13287-022-03234-y.
Neural stem cells (NSCs) are believed to have the most therapeutic potential for neurological disorders because they can differentiate into various neurons and glial cells. This research evaluated the safety and efficacy of intranasal administration of NSCs in children with cerebral palsy (CP). The functional brain network (FBN) analysis based on electroencephalogram (EEG) and voxel-based morphometry (VBM) analysis based on T1-weighted images were performed to evaluate functional and structural changes in the brain.
A total of 25 CP patients aged 3-12 years were randomly assigned to the treatment group (n = 15), which received an intranasal infusion of NSCs loaded with nasal patches and rehabilitation therapy, or the control group (n = 10) received rehabilitation therapy only. The primary endpoints were the safety (assessed by the incidence of adverse events (AEs), laboratory and imaging examinations) and the changes in the Gross Motor Function Measure-88 (GMFM-88), the Activities of Daily Living (ADL) scale, the Sleep Disturbance Scale for Children (SDSC), and some adapted scales. The secondary endpoints were the FBN and VBM analysis.
There were only four AEs happened during the 24-month follow-up period. There was no significant difference in the laboratory examinations before and after treatment, and the magnetic resonance imaging showed no abnormal nasal and intracranial masses. Compared to the control group, patients in the treatment group showed apparent improvements in GMFM-88 and ADL 24 months after treatment. Compared with the baseline, the scale scores of the Fine Motor Function, Sociability, Life Adaptability, Expressive Ability, GMFM-88, and ADL increased significantly in the treatment group 24 months after treatment, while the SDSC score decreased considerably. Compared with baseline, the FBN analysis showed a substantial decrease in brain network energy, and the VBM analysis showed a significant increase in gray matter volume in the treatment group after NSCs treatment.
Our results showed that intranasal administration of NSCs was well-tolerated and potentially beneficial in children with CP.
The study was registered in ClinicalTrials.gov (NCT03005249, registered 29 December 2016, https://www.
gov/ct2/show/NCT03005249 ) and the Medical Research Registration Information System (CMR-20161129-1003).
神经干细胞（NSCs）被认为对神经疾病具有最大的治疗潜力，因为它们可以分化为各种神经元和神经胶质细胞。本研究评估了经鼻给予神经干细胞治疗脑瘫（CP）儿童的安全性和疗效。基于脑电图（EEG）的功能脑网络（FBN）分析和基于 T1 加权图像的体素形态计量学（VBM）分析用于评估大脑的功能和结构变化。
共纳入 25 例 3-12 岁 CP 患儿，随机分为治疗组（n=15）和对照组（n=10）。治疗组给予载有神经干细胞的鼻贴片经鼻输注联合康复治疗，对照组仅给予康复治疗。主要终点为安全性（通过不良事件（AE）发生率、实验室和影像学检查评估）和粗大运动功能测量-88 量表（GMFM-88）、日常生活活动量表（ADL）、儿童睡眠障碍量表（SDSC）及部分适应量表的变化。次要终点为 FBN 和 VBM 分析。
在 24 个月的随访期间，仅发生 4 例 AE。治疗前后实验室检查无显著差异，磁共振成像未见异常鼻内和颅内肿块。与对照组相比，治疗组 GMFM-88 和 ADL 在治疗后 24 个月时明显改善。与基线相比，治疗组精细运动功能、社交能力、生活适应性、表达能力、GMFM-88 和 ADL 评分在治疗后 24 个月时显著增加，而 SDSC 评分明显降低。与基线相比，FBN 分析显示脑网络能量明显降低，VBM 分析显示治疗后神经干细胞治疗组灰质体积显著增加。
我们的研究结果表明，经鼻给予神经干细胞在 CP 儿童中具有良好的耐受性和潜在的益处。
本研究在 ClinicalTrials.gov（NCT03005249，注册日期 2016 年 12 月 29 日，https://www.clinicaltrials.gov/ct2/show/NCT03005249）和医学研究注册信息系统（CMR-20161129-1003）进行了注册。
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