Treatment of Spinocerebellar Ataxia with Mesenchymal Stem Cells: A Phase I/IIa Clinical Study
Ataxia is one of the most devastating symptoms of many neurodegenerative disorders. As of today, there is not any effective treatment to retard its progression. Mesenchymal stem cells (MSCs) have shown promise in treating neurodegenerative diseases. We hereby report the results of a phase I/IIa clin...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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SAGE Publishing
2017-03-01
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| Series: | Cell Transplantation |
| Online Access: | https://doi.org/10.3727/096368916X694373 |
| _version_ | 1818446011868119040 |
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| author | Yun-An Tsai Ren-Shyan Liu Jiing-Feng Lirng Bang-Hung Yang Chin-Hao Chang Yi-Chen Wang Yu-Shan Wu Jennifer Hui-Chun Ho Oscar K. Lee Bing-Wen Soong |
| author_facet | Yun-An Tsai Ren-Shyan Liu Jiing-Feng Lirng Bang-Hung Yang Chin-Hao Chang Yi-Chen Wang Yu-Shan Wu Jennifer Hui-Chun Ho Oscar K. Lee Bing-Wen Soong |
| author_sort | Yun-An Tsai |
| collection | DOAJ |
| description | Ataxia is one of the most devastating symptoms of many neurodegenerative disorders. As of today, there is not any effective treatment to retard its progression. Mesenchymal stem cells (MSCs) have shown promise in treating neurodegenerative diseases. We hereby report the results of a phase I/IIa clinical study conducted in Taiwan to primarily evaluate the safety, tolerability, and, secondarily, the possible efficacy of intravenous administration of allogeneic adipose tissue-derived MSCs from healthy donors. Six patients with spinocerebellar ataxia type 3 and one with multiple system atrophy-cerebellar type were included in this open-label study with intravenous administration of 10 6 cells/kg body weight. The subjects were closely monitored for 1 year for safety (vital signs, complete blood counts, serum biochemical profiles, and urinalysis) and possible efficacy (scale for assessment and rating of ataxia and sensory organization testing scores, metabolite ratios on the brain magnetic resonance spectroscopy, and brain glucose metabolism of 18-fluorodeoxyglucose using positron emission tomography). No adverse events related to the injection of MSCs during the 1-year follow-up were observed. The intravenous administration of allogeneic MSCs seemed well tolerated. Upon study completion, all patients wished to continue treatment with the allogeneic MSCs. We conclude that allogeneic MSCs given by intravenous injection seems to be safe and tolerable in patients with spinocerebellar ataxia type 3, thus supporting advancement of the clinical development of allogeneic MSCs for the treatment of spinocerebellar ataxias (SCAs) in a randomized, double-blind, placebo-controlled phase II trials. |
| first_indexed | 2024-12-14T19:40:57Z |
| format | Article |
| id | doaj.art-c1fb672177624d75a341a515e2802d4d |
| institution | Directory Open Access Journal |
| issn | 0963-6897 1555-3892 |
| language | English |
| last_indexed | 2024-12-14T19:40:57Z |
| publishDate | 2017-03-01 |
| publisher | SAGE Publishing |
| record_format | Article |
| series | Cell Transplantation |
| spelling | doaj.art-c1fb672177624d75a341a515e2802d4d2022-12-21T22:49:42ZengSAGE PublishingCell Transplantation0963-68971555-38922017-03-012610.3727/096368916X694373Treatment of Spinocerebellar Ataxia with Mesenchymal Stem Cells: A Phase I/IIa Clinical StudyYun-An Tsai0Ren-Shyan Liu1Jiing-Feng Lirng2Bang-Hung Yang3Chin-Hao Chang4Yi-Chen Wang5Yu-Shan Wu6Jennifer Hui-Chun Ho7Oscar K. Lee8Bing-Wen Soong9Department of Neurosurgery, Neurological Institute, Taipei Veterans General Hospital, Taipei, TaiwanFaculty of Biomedical Imaging and Radiological Sciences, National Yang-Ming University, Taipei, TaiwanDepartment of Radiology, Taipei Veterans General Hospital, Taipei, TaiwanFaculty of Biomedical Imaging and Radiological Sciences, National Yang-Ming University, Taipei, TaiwanDepartment of Medical Research, National Taiwan University Hospital, Taipei, TaiwanSteminent Biotherapeutics Inc., Taipei, TaiwanSteminent Biotherapeutics Inc., Taipei, TaiwanSteminent Biotherapeutics Inc., Taipei, TaiwanInstitute of Clinical Medicine, National Yang-Ming University, Taipei, TaiwanDepartment of Neurology, National Yang-Ming University, Taipei, TaiwanAtaxia is one of the most devastating symptoms of many neurodegenerative disorders. As of today, there is not any effective treatment to retard its progression. Mesenchymal stem cells (MSCs) have shown promise in treating neurodegenerative diseases. We hereby report the results of a phase I/IIa clinical study conducted in Taiwan to primarily evaluate the safety, tolerability, and, secondarily, the possible efficacy of intravenous administration of allogeneic adipose tissue-derived MSCs from healthy donors. Six patients with spinocerebellar ataxia type 3 and one with multiple system atrophy-cerebellar type were included in this open-label study with intravenous administration of 10 6 cells/kg body weight. The subjects were closely monitored for 1 year for safety (vital signs, complete blood counts, serum biochemical profiles, and urinalysis) and possible efficacy (scale for assessment and rating of ataxia and sensory organization testing scores, metabolite ratios on the brain magnetic resonance spectroscopy, and brain glucose metabolism of 18-fluorodeoxyglucose using positron emission tomography). No adverse events related to the injection of MSCs during the 1-year follow-up were observed. The intravenous administration of allogeneic MSCs seemed well tolerated. Upon study completion, all patients wished to continue treatment with the allogeneic MSCs. We conclude that allogeneic MSCs given by intravenous injection seems to be safe and tolerable in patients with spinocerebellar ataxia type 3, thus supporting advancement of the clinical development of allogeneic MSCs for the treatment of spinocerebellar ataxias (SCAs) in a randomized, double-blind, placebo-controlled phase II trials.https://doi.org/10.3727/096368916X694373 |
| spellingShingle | Yun-An Tsai Ren-Shyan Liu Jiing-Feng Lirng Bang-Hung Yang Chin-Hao Chang Yi-Chen Wang Yu-Shan Wu Jennifer Hui-Chun Ho Oscar K. Lee Bing-Wen Soong Treatment of Spinocerebellar Ataxia with Mesenchymal Stem Cells: A Phase I/IIa Clinical Study Cell Transplantation |
| title | Treatment of Spinocerebellar Ataxia with Mesenchymal Stem Cells: A Phase I/IIa Clinical Study |
| title_full | Treatment of Spinocerebellar Ataxia with Mesenchymal Stem Cells: A Phase I/IIa Clinical Study |
| title_fullStr | Treatment of Spinocerebellar Ataxia with Mesenchymal Stem Cells: A Phase I/IIa Clinical Study |
| title_full_unstemmed | Treatment of Spinocerebellar Ataxia with Mesenchymal Stem Cells: A Phase I/IIa Clinical Study |
| title_short | Treatment of Spinocerebellar Ataxia with Mesenchymal Stem Cells: A Phase I/IIa Clinical Study |
| title_sort | treatment of spinocerebellar ataxia with mesenchymal stem cells a phase i iia clinical study |
| url | https://doi.org/10.3727/096368916X694373 |
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