Safety and efficacy outcomes after intranasal administration of neural stem cells in cerebral palsy: a randomized phase 1/2 controlled trial

Author:

Lv Zhongyue,Li Ying,Wang Yachen,Cong Fengyu,Li Xiaoyan,Cui Wanming,Han Chao,Wei Yushan,Hong Xiaojun,Liu Yong,Ma Luyi,Jiao Yang,Zhang Chi,Li Huanjie,Jin Mingyan,Wang Liang,Ni Shiwei,Liu JingORCID

Abstract

Abstract Background 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. Methods 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. Results 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. Conclusions Our results showed that intranasal administration of NSCs was well-tolerated and potentially beneficial in children with CP. Trial registration: The study was registered in ClinicalTrials.gov (NCT03005249, registered 29 December 2016, https://www.clinicaltrials.gov/ct2/show/NCT03005249) and the Medical Research Registration Information System (CMR-20161129-1003).

Funder

Program of the China National Health Commission and National Medical Products Administration

National Natural Science Foundation of China

Liaoning Province Excellent Talent Program Project

Dalian Science and Technology Innovation Fund

Top young talents of Liaoning Provincial Government

National Defense Science and Technology Innovation Fund of China

Publisher

Springer Science and Business Media LLC

Subject

Cell Biology,Biochemistry, Genetics and Molecular Biology (miscellaneous),Molecular Medicine,Medicine (miscellaneous)

Cited by 12 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献

1. The 2023 yearbook of Neurorestoratology;Journal of Neurorestoratology;2024-09

2. A systematic review of cell therapy modalities and outcomes in cerebral palsy;Molecular and Cellular Biochemistry;2024-07-21

3. The roles of neural stem cells in myelin regeneration and repair therapy after spinal cord injury;Stem Cell Research & Therapy;2024-07-08

4. A bibliometric analysis of cerebral palsy from 2003 to 2022;Frontiers in Neurology;2024-04-02

5. DESAFIOS E AVANÇOS NO TRATAMENTO DA PARALISIA CEREBRAL INFANTIL: UMA REVISÃO BIBLIOGRÁFICA;RECIMA21 - Revista Científica Multidisciplinar - ISSN 2675-6218;2024-02-08

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3