Finding a new therapeutic approach for no-option Parkinsonisms: mesenchymal stromal cells for progressive supranuclear palsy

Please always quote using this URN: urn:nbn:de:bvb:20-opus-165725
  • Background: The trophic, anti-apoptotic and regenerative effects of bone marrow mesenchymal stromal cells (MSC) may reduce neuronal cell loss in neurodegenerative disorders. Methods: We used MSC as a novel candidate therapeutic tool in a pilot phase-I study for patients affected by progressive supranuclear palsy (PSP), a rare, severe and no-option form of Parkinsonism. Five patients received the cells by infusion into the cerebral arteries. Effects were assessed using the best available motor function rating scales (UPDRS, Hoehn and Yahr,Background: The trophic, anti-apoptotic and regenerative effects of bone marrow mesenchymal stromal cells (MSC) may reduce neuronal cell loss in neurodegenerative disorders. Methods: We used MSC as a novel candidate therapeutic tool in a pilot phase-I study for patients affected by progressive supranuclear palsy (PSP), a rare, severe and no-option form of Parkinsonism. Five patients received the cells by infusion into the cerebral arteries. Effects were assessed using the best available motor function rating scales (UPDRS, Hoehn and Yahr, PSP rating scale), as well as neuropsychological assessments, gait analysis and brain imaging before and after cell administration. Results: One year after cell infusion, all treated patients were alive, except one, who died 9 months after the infusion for reasons not related to cell administration or to disease progression (accidental fall). In all treated patients motor function rating scales remained stable for at least six-months during the one-year follow-up. Conclusions: We have demonstrated for the first time that MSC administration is feasible in subjects with PSP. In these patients, in whom deterioration of motor function is invariably rapid, we recorded clinical stabilization for at least 6 months. These encouraging results pave the way to the next randomized, placebo-controlled phase-II study that will definitively provide information on the efficacy of this innovative approach.show moreshow less

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Author: Margherita Canesi, Rosaria Giordano, Lorenza Lazzari, Maurizio Isalberti, Ioannis Ugo Isaias, Riccardo Benti, Paolo Rampini, Giorgio Marotta, Aurora Colombo, Emanuele Cereda, Mariangela Dipaola, Tiziana Montemurro, Mariele Vigano, Silvia Budelli, Elisa Montelatici, Cristiana Lavazza, Agostino Cortelezzi, Gianni Pezzoli
URN:urn:nbn:de:bvb:20-opus-165725
Document Type:Journal article
Faculties:Medizinische Fakultät / Neurologische Klinik und Poliklinik
Language:English
Parent Title (English):Journal of Translational Medicine
Year of Completion:2016
Volume:14
Issue:127
Pagenumber:1-11
Source:J Transl Med (2016) 14:127, p1-11
DOI:https://doi.org/10.1186/s12967-016-0880-2
Dewey Decimal Classification:6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit
Tag:Cell therapy; Mesenchymal stem/stromal cells; Progressive supranuclear palsy; Regenerative medicine
Release Date:2021/03/02
Licence (German):License LogoCC BY: Creative-Commons-Lizenz: Namensnennung 4.0 International