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.

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

CHIO', Adriano;FANTOZZI, Roberto;FOLLENZI, Antonia;VERCELLI, Alessandro
2016-01-01

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.
18
12
1471
1475
http://www.journals.elsevier.com/cytotherapy
ALS; clinical trials; stem cells; transplantation; Immunology and Allergy; Immunology; Oncology; Genetics (clinical); Cell Biology; Cancer Research; Transplantation
Atassi, Nazem; Beghi, Ettore; Blanquer, Miguel; Boulis, Nicholas M.; Cantello, Roberto; Caponnetto, Claudia; Chiò, Adriano; Dunnett, Stephen B.; Feldman, Eva L.; Vescovi, Angelo; Mazzini, Letizia; Bendotti, Caterina; Bersano, Enrica; Brajkovic, Simona; Car, Piergiorgio; De Marchi, Fabiola; Fantozzi, Roberto; Follenzi, Antonia; Gelati, Maurizio; Giorgi, Cesare; Grilli, Mariagrazia; Guenzi, Pierdavide; La Bella, Vincenzo; Mancardi, Gian Luigi; Panzarasa, Gabriele; Poloni, Marco; Profico, Daniela; Silani, Vincenzo; Sorarù, Gianni; Spataro, Rossella; Stecco, Alessandro; Vercelli, Alessandro
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2318/1636979
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