Academic Journal
Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD
| العنوان: | Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD |
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| المؤلفون: | Kirkeby, Agnete, Nelander, Jenny, Hoban, Deirdre B, Rogelius, Nina, Bjartmarz, Hjálmar, Storm, Petter, Fiorenzano, Alessandro, Adler, Andrew F, Vale, Shelby, Mudannayake, Janitha, Zhang, Yu, Cardoso, Tiago, Mattsson, Bengt, Landau, Anne M, Glud, Andreas N, Sørensen, Jens C, Lillethorup, Thea P, Lowdell, Mark, Carvalho, Carla, Bain, Owen, van Vliet, Trinette, Lindvall, Olle, Björklund, Anders, Harry, Bronwen, Cutting, Emma, Widner, Håkan, Paul, Gesine, Barker, Roger A, Parmar, Malin |
| المصدر: | Kirkeby , A , Nelander , J , Hoban , D B , Rogelius , N , Bjartmarz , H , Novo Nordisk Cell Therapy R&D , Storm , P , Fiorenzano , A , Adler , A F , Vale , S , Mudannayake , J , Zhang , Y , Cardoso , T , Mattsson , B , Landau , A M , Glud , A N , Sørensen , J C , Lillethorup , T P , Lowdell , M , Carvalho , C , Bain , O , van Vliet , T , Lindvall , .... |
| سنة النشر: | 2023 |
| المجموعة: | Aarhus University: Research |
| مصطلحات موضوعية: | ATMP, Parkinson's, clinical trial, dopamine, minipig, neurosurgery, neurosurgical, pluripotent, regulatory, stem cell therapy, transplantation, Human Embryonic Stem Cells, Humans, Rats, Parkinson Disease/therapy, Tissue Distribution, Animals, Stem Cell Transplantation/methods, Cell Differentiation/physiology, Dopaminergic Neurons/physiology |
| الوصف: | Cell replacement therapies for Parkinson's disease (PD) based on transplantation of pluripotent stem cell-derived dopaminergic neurons are now entering clinical trials. Here, we present quality, safety, and efficacy data supporting the first-in-human STEM-PD phase I/IIa clinical trial along with the trial design. The STEM-PD product was manufactured under GMP and quality tested in vitro and in vivo to meet regulatory requirements. Importantly, no adverse effects were observed upon testing of the product in a 39-week rat GLP safety study for toxicity, tumorigenicity, and biodistribution, and a non-GLP efficacy study confirmed that the transplanted cells mediated full functional recovery in a pre-clinical rat model of PD. We further observed highly comparable efficacy results between two different GMP batches, verifying that the product can be serially manufactured. A fully in vivo-tested batch of STEM-PD is now being used in a clinical trial of 8 patients with moderate PD, initiated in 2022. |
| نوع الوثيقة: | article in journal/newspaper |
| وصف الملف: | application/pdf |
| اللغة: | English |
| DOI: | 10.1016/j.stem.2023.08.014 |
| الاتاحة: | https://pure.au.dk/portal/en/publications/97e0d460-3e16-4b39-b285-bdbaaacf8d3b https://doi.org/10.1016/j.stem.2023.08.014 https://pure.au.dk/ws/files/404617548/1-s2.0-S1934590923003211-main.pdf http://www.scopus.com/inward/record.url?scp=85174454209&partnerID=8YFLogxK |
| Rights: | info:eu-repo/semantics/openAccess |
| رقم الانضمام: | edsbas.F644B84C |
| قاعدة البيانات: | BASE |
| DOI: | 10.1016/j.stem.2023.08.014 |
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