TY  - JOUR
A1  - Canesi, Margherita
A1  - Giordano, Rosaria
A1  - Lazzari, Lorenza
A1  - Isalberti, Maurizio
A1  - Isaias, Ioannis Ugo
A1  - Benti, Riccardo
A1  - Rampini, Paolo
A1  - Marotta, Giorgio
A1  - Colombo, Aurora
A1  - Cereda, Emanuele
A1  - Dipaola, Mariangela
A1  - Montemurro, Tiziana
A1  - Vigano, Mariele
A1  - Budelli, Silvia
A1  - Montelatici, Elisa
A1  - Lavazza, Cristiana
A1  - Cortelezzi, Agostino
A1  - Pezzoli, Gianni
T1  - Finding a new therapeutic approach for no-option Parkinsonisms: mesenchymal stromal cells for progressive supranuclear palsy
JF  - Journal of Translational Medicine
N2  - 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.
KW  - Progressive supranuclear palsy
KW  - Mesenchymal stem/stromal cells
KW  - Cell therapy
KW  - Regenerative medicine
Y1  - 2016
U6  - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-165725
VL  - 14
IS  - 127
ER  -