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

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• 作者：Rosaria Giordano (1)
  Margherita Canesi (2)
  Maurizio Isalberti (3)
  Ioannis Ugo Isaias (2) (4)
  Tiziana Montemurro (1)
  Mariele Viganò (1)
  Elisa Montelatici (1)
  Valentina Boldrin (1)
  Riccardo Benti (5)
  Agostino Cortelezzi (6)
  Nicola Fracchiolla (6)
  Lorenza Lazzari (1)
  Gianni Pezzoli (2)
  
• 关键词：Progressive supranuclear palsy ; Parkinson’s disease ; Mesenchymal stem and stromal cells ; Advanced therapy medicinal products ; Cellular therapy
• 刊名：Journal of Translational Medicine
• 出版年：2014
• 出版时间：December 2014
• 年：2014
• 卷：12
• 期：1
• 全文大小：615 KB
• 作者单位：Rosaria Giordano (1)
  Margherita Canesi (2)
  Maurizio Isalberti (3)
  Ioannis Ugo Isaias (2) (4)
  Tiziana Montemurro (1)
  Mariele Viganò (1)
  Elisa Montelatici (1)
  Valentina Boldrin (1)
  Riccardo Benti (5)
  Agostino Cortelezzi (6)
  Nicola Fracchiolla (6)
  Lorenza Lazzari (1)
  Gianni Pezzoli (2)
  
  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, Universit?tsklinikum 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
  
• ISSN：1479-5876

文摘

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