Intraspinal stem cell transplantation for amyotrophic lateral sclerosis: Ready for efficacy clinical trials?

the attendees of the International Workshop on Progress in Stem Cells Research for ALS/MND

doi:10.1016/j.jcyt.2016.08.005

Intraspinal stem cell transplantation for amyotrophic lateral sclerosis: Ready for efficacy clinical trials?

the attendees of the International Workshop on Progress in Stem Cells Research for ALS/MND

Risultato della ricerca: Contributo su rivista › Articolo in rivista › peer review

Abstract

Intraspinal stem cell (SC) transplantation represents a new therapeutic approach for amyotrophic lateral sclerosis (ALS) clinical trials. There are considerable difficulties in designing future efficacy trials, some related to the field of ALS and some that are specific to SCs or the mode of delivery. In October 2015, the most controversial points on SC transplantation were addressed during an international workshop intended to bring together international SC and ALS researchers in a public discussion on a topic for which expertise is limited. During the meeting, a discussion was started on the basic structure of the ideal clinical trial testing the efficacy and safety of SC transplantation. The current document includes a number of consensus points reflecting the design of phase II/III clinical trials.

Lingua originale	Inglese
pagine (da-a)	1471-1475
Numero di pagine	5
Rivista	Cytotherapy
Volume	18
Numero di pubblicazione	12
DOI	https://doi.org/10.1016/j.jcyt.2016.08.005
Stato di pubblicazione	Pubblicato - 1 dic 2016

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10.1016/j.jcyt.2016.08.005

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title = "Intraspinal stem cell transplantation for amyotrophic lateral sclerosis: Ready for efficacy clinical trials?",

abstract = "Intraspinal stem cell (SC) transplantation represents a new therapeutic approach for amyotrophic lateral sclerosis (ALS) clinical trials. There are considerable difficulties in designing future efficacy trials, some related to the field of ALS and some that are specific to SCs or the mode of delivery. In October 2015, the most controversial points on SC transplantation were addressed during an international workshop intended to bring together international SC and ALS researchers in a public discussion on a topic for which expertise is limited. During the meeting, a discussion was started on the basic structure of the ideal clinical trial testing the efficacy and safety of SC transplantation. The current document includes a number of consensus points reflecting the design of phase II/III clinical trials.",

keywords = "ALS, clinical trials, stem cells, transplantation",

author = "\{the attendees of the International Workshop on Progress in Stem Cells Research for ALS/MND\} and Nazem Atassi and Ettore Beghi and Miguel Blanquer and Boulis, \{Nicholas M.\} and Roberto Cantello and Claudia Caponnetto and Adriano Chi{\`o} and Dunnett, \{Stephen B.\} and Feldman, \{Eva L.\} and Angelo Vescovi and Letizia Mazzini and Caterina Bendotti and Enrica Bersano and Simona Brajkovic and Piergiorgio Car and \{De Marchi\}, Fabiola and Roberto Fantozzi and Antonia Follenzi and Maurizio Gelati and Cesare Giorgi and Mariagrazia Grilli and Pierdavide Guenzi and \{La Bella\}, Vincenzo and Mancardi, \{Gian Luigi\} and Gabriele Panzarasa and Marco Poloni and Daniela Profico and Vincenzo Silani and Gianni Sorar{\`u} and Rossella Spataro and Alessandro Stecco and Alessandro Vercelli",

note = "Publisher Copyright: {\textcopyright} 2016 International Society for Cellular Therapy",

year = "2016",

month = dec,

day = "1",

doi = "10.1016/j.jcyt.2016.08.005",

language = "English",

volume = "18",

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journal = "Cytotherapy",

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T2 - Ready for efficacy clinical trials?

AU - the attendees of the International Workshop on Progress in Stem Cells Research for ALS/MND

AU - Atassi, Nazem

AU - Beghi, Ettore

AU - Blanquer, Miguel

AU - Boulis, Nicholas M.

AU - Cantello, Roberto

AU - Caponnetto, Claudia

AU - Chiò, Adriano

AU - Dunnett, Stephen B.

AU - Feldman, Eva L.

AU - Vescovi, Angelo

AU - Mazzini, Letizia

AU - Bendotti, Caterina

AU - Bersano, Enrica

AU - Brajkovic, Simona

AU - Car, Piergiorgio

AU - De Marchi, Fabiola

AU - Fantozzi, Roberto

AU - Follenzi, Antonia

AU - Gelati, Maurizio

AU - Giorgi, Cesare

AU - Grilli, Mariagrazia

AU - Guenzi, Pierdavide

AU - La Bella, Vincenzo

AU - Mancardi, Gian Luigi

AU - Panzarasa, Gabriele

AU - Poloni, Marco

AU - Profico, Daniela

AU - Silani, Vincenzo

AU - Sorarù, Gianni

AU - Spataro, Rossella

AU - Stecco, Alessandro

AU - Vercelli, Alessandro

PY - 2016/12/1

Y1 - 2016/12/1

N2 - Intraspinal stem cell (SC) transplantation represents a new therapeutic approach for amyotrophic lateral sclerosis (ALS) clinical trials. There are considerable difficulties in designing future efficacy trials, some related to the field of ALS and some that are specific to SCs or the mode of delivery. In October 2015, the most controversial points on SC transplantation were addressed during an international workshop intended to bring together international SC and ALS researchers in a public discussion on a topic for which expertise is limited. During the meeting, a discussion was started on the basic structure of the ideal clinical trial testing the efficacy and safety of SC transplantation. The current document includes a number of consensus points reflecting the design of phase II/III clinical trials.

AB - Intraspinal stem cell (SC) transplantation represents a new therapeutic approach for amyotrophic lateral sclerosis (ALS) clinical trials. There are considerable difficulties in designing future efficacy trials, some related to the field of ALS and some that are specific to SCs or the mode of delivery. In October 2015, the most controversial points on SC transplantation were addressed during an international workshop intended to bring together international SC and ALS researchers in a public discussion on a topic for which expertise is limited. During the meeting, a discussion was started on the basic structure of the ideal clinical trial testing the efficacy and safety of SC transplantation. The current document includes a number of consensus points reflecting the design of phase II/III clinical trials.

KW - ALS

KW - clinical trials

KW - stem cells

KW - transplantation

UR - https://www.scopus.com/pages/publications/84992153785

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DO - 10.1016/j.jcyt.2016.08.005

M3 - Article

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SP - 1471

EP - 1475

JO - Cytotherapy

JF - Cytotherapy

IS - 12

ER -

Intraspinal stem cell transplantation for amyotrophic lateral sclerosis: Ready for efficacy clinical trials?

Abstract

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