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


CONCLUSIONS
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.


BACKGROUND
NCT01824121.
Cell Factory, Unit of Cell Therapy and Cryobiology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Italy. rosaria.giordano@policlinico.mi.it.
Rosaria
R
Giordano
Margherita
M
Canesi
Maurizio
M
Isalberti
Ioannis Ugo
IU
Isaias
Tiziana
T
Montemurro
Mariele
M
Viganò
Elisa
E
Montelatici
Valentina
V
Boldrin
Riccardo
R
Benti
Agostino
A
Cortelezzi
Nicola
N
Fracchiolla
Lorenza
L
Lazzari
Gianni
G
Pezzoli
D000328:Adult; D045744:Cell Line, Tumor; D006801:Humans; D045164:Mesenchymal Stem Cell Transplantation; D059630:Mesenchymal Stem Cells / Q000166:cytology*; D009414:Nerve Growth Factors / Q000378:metabolism; D013494:Supranuclear Palsy, Progressive / Q000628:therapy*; D057170:Translational Research, Biomedical; D014182:Transplantation, Autologous
D009414:Nerve Growth Factors