title: Finding a new therapeutic approach for no-option Parkinsonisms: mesenchymal stromal cells for progressive supranuclear palsy.
abstract: The trophic, anti-apoptotic and regenerative effects of bone marrow mesenchymal stromal cells (MSC) may reduce neuronal cell loss in neurodegenerative disorders.
  
  
  
  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.
  
  
  
  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.
  
  
  
  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. Trial registration ClinicalTrials.gov NCT01824121.
authors.affiliation: Parkinson Institute, G.Pini-CTO, exICP, Milan, Italy.
authors.forename: Margherita
authors.initials: M
authors.lastname: Canesi
authors.affiliation: Cell Factory, Unit of Cell Therapy and Cryobiology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via F Sforza 35, 20122, Milan, Italy. rosaria.giordano@policlinico.mi.it.
authors.forename: Rosaria
authors.initials: R
authors.lastname: Giordano
authors.affiliation: Cell Factory, Unit of Cell Therapy and Cryobiology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via F Sforza 35, 20122, Milan, Italy.
authors.forename: Lorenza
authors.initials: L
authors.lastname: Lazzari
authors.affiliation: Interventional Neuroradiology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Maurizio
authors.initials: M
authors.lastname: Isalberti
authors.affiliation: Julius-Maximilians-Universität Würzburg and Neurologische Klinik, Universitätsklinik Würzburg, Würzburg, Germany.|Department of Pathophysiology and Transplantation, Human Physiology Section, Università degli Studi di Milano, Milan, Italy.
authors.forename: Ioannis Ugo
authors.initials: IU
authors.lastname: Isaias
authors.affiliation: Nuclear Medicine Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Riccardo
authors.initials: R
authors.lastname: Benti
authors.affiliation: Neurosurgery Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Paolo
authors.initials: P
authors.lastname: Rampini
authors.affiliation: Nuclear Medicine Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Giorgio
authors.initials: G
authors.lastname: Marotta
authors.affiliation: Parkinson Institute, G.Pini-CTO, exICP, Milan, Italy.
authors.forename: Aurora
authors.initials: A
authors.lastname: Colombo
authors.affiliation: Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
authors.forename: Emanuele
authors.initials: E
authors.lastname: Cereda
authors.affiliation: Department of Pathophysiology and Transplantation, Human Physiology Section, Università degli Studi di Milano, Milan, Italy.
authors.forename: Mariangela
authors.initials: M
authors.lastname: Dipaola
authors.affiliation: Cell Factory, Unit of Cell Therapy and Cryobiology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via F Sforza 35, 20122, Milan, Italy.
authors.forename: Tiziana
authors.initials: T
authors.lastname: Montemurro
authors.affiliation: Cell Factory, Unit of Cell Therapy and Cryobiology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via F Sforza 35, 20122, Milan, Italy.
authors.forename: Mariele
authors.initials: M
authors.lastname: Viganò
authors.affiliation: Cell Factory, Unit of Cell Therapy and Cryobiology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via F Sforza 35, 20122, Milan, Italy.
authors.forename: Silvia
authors.initials: S
authors.lastname: Budelli
authors.affiliation: Cell Factory, Unit of Cell Therapy and Cryobiology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via F Sforza 35, 20122, Milan, Italy.
authors.forename: Elisa
authors.initials: E
authors.lastname: Montelatici
authors.affiliation: Cell Factory, Unit of Cell Therapy and Cryobiology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via F Sforza 35, 20122, Milan, Italy.
authors.forename: Cristiana
authors.initials: C
authors.lastname: Lavazza
authors.affiliation: Bone Marrow Transplantation Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Agostino
authors.initials: A
authors.lastname: Cortelezzi
authors.affiliation: Parkinson Institute, G.Pini-CTO, exICP, Milan, Italy.
authors.forename: Gianni
authors.initials: G
authors.lastname: Pezzoli
mesh_terms: D000368:Aged; D001696:Biomechanical Phenomena; D001853:Bone Marrow / Q000473:pathology; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D045164:Mesenchymal Stem Cell Transplantation; D059630:Mesenchymal Stem Cells / Q000166:cytology*; D008875:Middle Aged; D020734:Parkinsonian Disorders / Q000000981:diagnostic imaging / Q000628:therapy*; D049268:Positron-Emission Tomography; D013494:Supranuclear Palsy, Progressive / Q000000981:diagnostic imaging / Q000503:physiopathology / Q000628:therapy*; D015899:Tomography, Emission-Computed, Single-Photon
keywords: Cell therapy; Mesenchymal stem/stromal cells; Progressive supranuclear palsy; Regenerative medicine