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Autologous mesenchymal stem cell therapy for progressive supranuclear palsy: translation into a phase I controlled, randomized clinical study

Please always quote using this URN: urn:nbn:de:bvb:20-opus-117594
  • Background: Progressive Supranuclear Palsy (PSP) is a sporadic and progressive neurodegenerative disease which belongs to the family of tauopathies and involves both cortical and subcortical structures. No effective therapy is to date available. Methods/design: Autologous bone marrow (BM) mesenchymal stem cells (MSC) from patients affected by different type of parkinsonisms have shown their ability to improve the dopaminergic function in preclinical and clinical models. It is also possible to isolate and expand MSC from the BM of PSP patientsBackground: Progressive Supranuclear Palsy (PSP) is a sporadic and progressive neurodegenerative disease which belongs to the family of tauopathies and involves both cortical and subcortical structures. No effective therapy is to date available. Methods/design: Autologous bone marrow (BM) mesenchymal stem cells (MSC) from patients affected by different type of parkinsonisms have shown their ability to improve the dopaminergic function in preclinical and clinical models. It is also possible to isolate and expand MSC from the BM of PSP patients with the same proliferation rate and immuphenotypic profile as MSC from healthy donors. BM MSC can be efficiently delivered to the affected brain regions of PSP patients where they can exert their beneficial effects through different mechanisms including the secretion of neurotrophic factors. Here we propose a randomized, placebo-controlled, double-blind phase I clinical trial in patients affected by PSP with MSC delivered via intra-arterial injection. Discussion: To our knowledge, this is the first clinical trial to be applied in a no-option parkinsonism that aims to test the safety and to exploit the properties of autologous mesenchymal stem cells in reducing disease progression. The study has been designed to test the safety of this " first-in-man" approach and to preliminarily explore its efficacy by excluding the placebo effect. Trial registration: NCT01824121show moreshow less

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Author: Rosaria Giordano, Margherita Canesi, Maurizio Isalberti, Ioannis Ugo Isaias, Tiziana Montemurro, Mariele Viganò, Elisa Montelatici, Valentina Boldrin, Riccardo Benti, Agostino Cortelezzi, Nicola Fracchiolla, Lorenza Lazzari, Gianni Pezzoli
URN:urn:nbn:de:bvb:20-opus-117594
Document Type:Journal article
Faculties:Medizinische Fakultät / Neurologische Klinik und Poliklinik
Language:English
Parent Title (English):Journal of Translational Medicine
Year of Completion:2014
Volume:12
Issue:14
Source:Journal of Translational Medicine 2014 12:14. doi:10.1186/1479-5876-12-14
DOI:https://doi.org/10.1186/1479-5876-12-14
Pubmed Id:http://www.ncbi.nlm.nih.gov/pubmed?term=24438512
Dewey Decimal Classification:6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit
Tag:Parkinson's disease; Richardson-Olszewski-Syndrome; adrenal medulla; advanced therapy medicinal products; bone-marrow; cellular therapy; deep brain-stimulation; mesenchymal stem and stromal cells; multiple system atrophy; progressive supranuclear palsy; receptor tyrosine kinase; stromal cells; transplantation; treatment options
Release Date:2015/08/24
Licence (German):License LogoCC BY: Creative-Commons-Lizenz: Namensnennung