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

作者：Giordano Rosaria^*; Canesi Margherita; Isalberti Maurizio; Isaias Ioannis Ugo; Montemurro Tiziana; Vigano Mariele; Montelatici Elisa; Boldrin Valentina; Benti Riccardo; Cortelezzi Agostino; Fracchiolla Nicola; Lazzari Lorenza; Pezzoli Gianni

来源：Journal of Translational Medicine, 2014, 12(1): 14.

DOI：10.1186/1479-5876-12-14

• Progressive supranuclear palsy
• Parkinson's disease
• Mesenchymal stem and stromal cells
• Advanced therapy medicinal products
• Cellular therapy

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 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: NCT01824121

• 出版日期2014-1-17