دورية أكاديمية
Results from Phase I Clinical Trial with Intraspinal Injection of Neural Stem Cells in Amyotrophic Lateral Sclerosis: A Long‐Term Outcome
| العنوان: | Results from Phase I Clinical Trial with Intraspinal Injection of Neural Stem Cells in Amyotrophic Lateral Sclerosis: A Long‐Term Outcome |
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| المؤلفون: | Letizia Mazzini, Maurizio Gelati, Daniela Celeste Profico, Gianni Sorarù, Daniela Ferrari, Massimiliano Copetti, Gianmarco Muzi, Claudia Ricciolini, Sandro Carletti, Cesare Giorgi, Cristina Spera, Domenico Frondizi, Stefano Masiero, Alessandro Stecco, Carlo Cisari, Enrica Bersano, Fabiola De Marchi, Maria Francesca Sarnelli, Giorgia Querin, Roberto Cantello, Francesco Petruzzelli, Annamaria Maglione, Cristina Zalfa, Elena Binda, Alberto Visioli, Domenico Trombetta, Barbara Torres, Laura Bernardini, Alessandra Gaiani, Maurilio Massara, Silvia Paolucci, Nicholas M. Boulis, Angelo L. Vescovi, on behalf of the ALS‐NSCs Trial Study Group |
| المصدر: | Stem Cells Translational Medicine, Vol 8, Iss 9, Pp 887-897 (2019) |
| بيانات النشر: | Oxford University Press, 2019. |
| سنة النشر: | 2019 |
| المجموعة: | LCC:Medicine (General) LCC:Cytology |
| مصطلحات موضوعية: | Adult stem cells, Cellular therapy, Clinical trials, Fetal stem cells, Medicine (General), R5-920, Cytology, QH573-671 |
| الوصف: | Abstract The main objective of this phase I trial was to assess the feasibility and safety of microtransplanting human neural stem cell (hNSC) lines into the spinal cord of patients with amyotrophic lateral sclerosis (ALS). Eighteen patients with a definite diagnosis of ALS received microinjections of hNSCs into the gray matter tracts of the lumbar or cervical spinal cord. Patients were monitored before and after transplantation by clinical, psychological, neuroradiological, and neurophysiological assessment. For up to 60 months after surgery, none of the patients manifested severe adverse effects or increased disease progression because of the treatment. Eleven patients died, and two underwent tracheotomy as a result of the natural history of the disease. We detected a transitory decrease in progression of ALS Functional Rating Scale Revised, starting within the first month after surgery and up to 4 months after transplantation. Our results show that transplantation of hNSC is a safe procedure that causes no major deleterious effects over the short or long term. This study is the first example of medical transplantation of a highly standardized cell drug product, which can be reproducibly and stably expanded ex vivo, comprising hNSC that are not immortalized, and are derived from the forebrain of the same two donors throughout this entire study as well as across future trials. Our experimental design provides benefits in terms of enhancing both intra‐ and interstudy reproducibility and homogeneity. Given the potential therapeutic effects of the hNSCs, our observations support undertaking future phase II clinical studies in which increased cell dosages are studied in larger cohorts of patients. Stem Cells Translational Medicine 2019;8:887&897 |
| نوع الوثيقة: | article |
| وصف الملف: | electronic resource |
| اللغة: | English |
| تدمد: | 2157-6580 2157-6564 |
| العلاقة: | https://doaj.org/toc/2157-6564Test; https://doaj.org/toc/2157-6580Test |
| DOI: | 10.1002/sctm.18-0154 |
| الوصول الحر: | https://doaj.org/article/1b266c15eb0b4c92a1cb1b70a47533cfTest |
| رقم الانضمام: | edsdoj.1b266c15eb0b4c92a1cb1b70a47533cf |
| قاعدة البيانات: | Directory of Open Access Journals |
Full Text Finder | تدمد: | 21576580 21576564 |
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| DOI: | 10.1002/sctm.18-0154 |