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

Academic Institute

Research output: Contribution to journal › Article › peer-review

18 Scopus citations

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.

Original language	English (US)
Pages (from-to)	1471-1475
Number of pages	5
Journal	Cytotherapy
Volume	18
Issue number	12
DOIs	https://doi.org/10.1016/j.jcyt.2016.08.005
State	Published - Dec 1 2016

Keywords

ALS
clinical trials
stem cells
transplantation

ASJC Scopus subject areas

Immunology and Allergy
Immunology
Oncology
Genetics(clinical)
Cell Biology
Transplantation
Cancer Research

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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",

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T1 - Intraspinal stem cell transplantation for amyotrophic lateral sclerosis

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

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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.

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KW - stem cells

KW - transplantation

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

Abstract

Keywords

ASJC Scopus subject areas

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