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Open Access Protocol

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

Rosaria Giordano1*, Margherita Canesi2, Maurizio Isalberti3, Ioannis Ugo Isaias24, Tiziana Montemurro1, Mariele Viganò1, Elisa Montelatici1, Valentina Boldrin1, Riccardo Benti5, Agostino Cortelezzi6, Nicola Fracchiolla6, Lorenza Lazzari1 and Gianni Pezzoli2

Author Affiliations

1 Cell Factory, Unit of Cell Therapy and Cryobiology, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milano, Italy

2 Parkinson Center, Istituti Clinici di Perfezionamento, Milano, Italy

3 Interventional Neuroradiology Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milano, Italy

4 Klinik und Poliklinik für Neurologie, Universitätsklinikum Würzburg, Würzburg, Germany

5 Nuclear Medicine Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milano, Italy

6 Hematology and Transplantation Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico and University of Milan, Milan, Italy

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Journal of Translational Medicine 2014, 12:14  doi:10.1186/1479-5876-12-14

Published: 17 January 2014

Abstract

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

Keywords:
Progressive supranuclear palsy; Parkinson’s disease; Mesenchymal stem and stromal cells; Advanced therapy medicinal products; Cellular therapy