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.
| Original language | English |
|---|---|
| Article number | 14 |
| Journal | Journal of Translational Medicine |
| Volume | 12 |
| Issue number | 1 |
| DOIs | |
| Publication status | Published - Jan 17 2014 |
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Keywords
- Advanced therapy medicinal products
- Cellular therapy
- Mesenchymal stem and stromal cells
- Parkinson's disease
- Progressive supranuclear palsy
ASJC Scopus subject areas
- Biochemistry, Genetics and Molecular Biology(all)
- Medicine(all)
Cite this
Autologous mesenchymal stem cell therapy for progressive supranuclear palsy : Translation into a phase I controlled, randomized clinical study. / Giordano, Rosaria; Canesi, Margherita; Isalberti, Maurizio; Isaias, Ioannis U.; Montemurro, Tiziana; Viganò, Mariele; Montelatici, Elisa; Boldrin, Valentina; Benti, Riccardo; Cortelezzi, Agostino; Fracchiolla, Nicola; Lazzari, Lorenza; Pezzoli, Gianni.
In: Journal of Translational Medicine, Vol. 12, No. 1, 14, 17.01.2014.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Autologous mesenchymal stem cell therapy for progressive supranuclear palsy
T2 - Translation into a phase I controlled, randomized clinical study
AU - Giordano, Rosaria
AU - Canesi, Margherita
AU - Isalberti, Maurizio
AU - Isaias, Ioannis U.
AU - Montemurro, Tiziana
AU - Viganò, Mariele
AU - Montelatici, Elisa
AU - Boldrin, Valentina
AU - Benti, Riccardo
AU - Cortelezzi, Agostino
AU - Fracchiolla, Nicola
AU - Lazzari, Lorenza
AU - Pezzoli, Gianni
PY - 2014/1/17
Y1 - 2014/1/17
N2 - 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.
AB - 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.
KW - Advanced therapy medicinal products
KW - Cellular therapy
KW - Mesenchymal stem and stromal cells
KW - Parkinson's disease
KW - Progressive supranuclear palsy
UR - http://www.scopus.com/inward/record.url?scp=84892462886&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84892462886&partnerID=8YFLogxK
U2 - 10.1186/1479-5876-12-14
DO - 10.1186/1479-5876-12-14
M3 - Article
C2 - 24438512
AN - SCOPUS:84892462886
VL - 12
JO - Journal of Translational Medicine
JF - Journal of Translational Medicine
SN - 1479-5876
IS - 1
M1 - 14
ER -