中国神经再生研究(英文版)

中国神经再生研究(英文版) ›› 2019, Vol. 14 ›› Issue (2): 313-318.doi: 10.4103/1673-5374.243723

• 原著:退行性病与再生 • 上一篇    下一篇

脐带间充质干细胞鞘内注射治疗肌萎缩侧索硬化症的安全性

  

  • 出版日期:2019-02-15 发布日期:2019-02-15
  • 基金资助:

    这项研究得到了奥尔什丁细胞治疗研究所（InstytutTerapiiKomórkowych，FamiCord Group）的支持

Safety of intrathecal injection of Wharton’s jelly-derived mesenchymal stem cells in amyotrophic lateral sclerosis therapy

Monika Barczewska1, 3, 4, Mariusz Grudniak5, Stanisław Maksymowicz2, 4, Tomasz Siwek1, 3, 4, Tomasz Ołdak5, Katarzyna Jezierska-Woźniak6, Dominika Gładysz5, Wojciech Maksymowicz1, 3   

  1. 1 Department of Neurology and Neurosurgery, School of Medicine, Collegium Medicum - University of Warmia and Mazury in Olsztyn, Olsztyn, Poland
    2 Department of Psychology, Clinical Logopedics and Social Science in Medicine, Faculty of Health Sciences, Collegium Medicum - University of Warmia and Mazury in Olsztyn, Olsztyn, Poland
    3 University Clinical Hospital in Olsztyn, Olsztyn, Poland
    4 Instytut Terapii Komórkowych w Olsztynie (Cell Therapies Institute, FamiCord Group), Olsztyn, Poland
    5 Polski Bank Komórek Macierzystych (PBKM, FamiCord Group), Warszawa, Poland
    6 Department of Neurology and Neurosurgery, Laboratory of Regenerative Medicine, School of Medicine, Collegium Medicum-University of
    Warmia and Mazury in Olsztyn, Olsztyn, Poland
  • Online:2019-02-15 Published:2019-02-15
  • Contact: Stanis?aw Maksymowicz, PhD, stanislaw.maksymowicz@uwm.edu.pl.
  • Supported by:

    This research was supported by Cell Therapy Institute (Instytut Terapii Komórkowych, FamiCord Group) in Olsztyn (to MB, SM, TS).

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摘要：

动物实验已证实，间充质干细胞可抑制运动神经元凋亡和炎症因子的表达，且可提高神经营养因子的表达，因而研究者们认为间充质干细胞在肌萎缩侧索硬化症治疗方面具有应用前景，但其临床应用安全性仍有待验证，为评估来源于脐带华通胶的间充质干细胞鞘内注射治疗肌萎缩侧索硬化症的安全性。试验纳入43例患者（平均年龄57.3岁，女16例，男27例），一次鞘内注射的脐带华通胶间充质干细胞的平均剂量为0.42×106个细胞/kg。根据患者的临床表现，鞘内注射部位为颈、胸和腰部髓鞘，每次注射的时间间隔为2个月。评估治疗6个月过程中的不良事件。结果无严重的不良事件发生，仅有1例患者首次注射干细胞时发生头痛，但在接下来的2次治疗中未出现这种现象。说明肌萎缩侧索硬化症患者对鞘内脐带华通胶间充质干细胞治疗的耐受良好。该研究经波兰Olsztyn的Warmia大学和Mazury医学院生物伦理委员会批准（36/2014，8/2016），且已于2016-08-29在北美临床试验中心注册（NCT02881476）.

orcid: 0000-0002-6606-9575(Stanis?aw Maksymowicz)

关键词: 肌萎缩侧索硬化, 干细胞疗法, 鞘内注射, WJ-MSC, 不良事件, 安全性, 脑脊液

Abstract:

Animal experiments have confirmed that mesenchymal stem cells can inhibit motor neuron apoptosis and inflammatory factor expression and increase neurotrophic factor expression. Therefore, mesenchymal stem cells have been shown to exhibit prospects in the treatment of amyotrophic lateral sclerosis. However, the safety of their clinical application needs to be validated. To investigate the safety of intrathecal injection of Wharton’s jelly-derived mesenchymal stem cells in amyotrophic lateral sclerosis therapy, 43 patients (16 females and 27 males, mean age of 57.3 years) received an average dose of 0.42 × 106 cells/kg through intrathecal administration at the cervical, thoracic or lumbar region depending on the clinical symptoms. There was a 2 month interval between two injections. The adverse events occurring during a 6-month treatment period were evaluated. No adverse events occurred. Headache occurred in one case only after first injection of stem cells. This suggests that intrathecal injection of Wharton’s Jelly-derived mesenchymal stem cells is well tolerated in patients with amyotrophic lateral sclerosis. This study was approved by the Bioethical Committee of School of Medicine, University of Warmia and Mazury in Olsztyn, Poland (approval No. 36/2014 and approval No. 8/2016). This study was registered with the ClinicalTrials.gov (identifier: NCT02881476) on August 29, 2016.

Key words: amyotrophic lateral sclerosis, stem cells therapy, intrathecal injections, Wharton’s jelly-derived mesenchymal stem cells, adverse events, safety, cerebrospinal fluid, neural regeneration