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

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• 作者：Nazem Atassi¹ ; Ettore Beghi² ; Miguel Blanquer³ ; Nicholas M. Boulis⁴ ; Roberto Cantello⁵ ; Claudia Caponnetto⁶ ; Adriano Chi&ograve ; ⁷ ; Stephen B. Dunnett⁸ ; Eva L. Feldman⁹ ; Angelo Vescovi¹⁰ ; ¹¹ ; Letizia Mazzini¹² ; ^{mazzini.l@libero.it" class="auth_mail" title="E-mail the corresponding author} ; on behalf of the attendees of the International Workshop on Progress in Stem Cells Research for ALS/MND^* ; Caterina Bendotti¹³ ; Enrica Bersano¹⁴ ; Simona Brajkovic¹⁵ ; Piergiorgio Car¹⁶ ; Fabiola De Marchi¹⁴ ; Roberto Fantozzi¹⁷ ; Antonia Follenzi¹⁸ ; Maurizio Gelati¹⁹ ; Cesare Giorgi²⁰ ; Mariagrazia Grilli²¹ ; Pierdavide Guenzi²² ; Vincenzo La Bella²³ ; Gian Luigi Mancardi²⁴ ; Gabriele Panzarasa¹⁵ ; Marco Poloni²⁵ ; Daniela Profico¹⁸ ; ²⁶ ; Vincenzo Silani²⁷ ; Gianni Sorar&ugrave ; ²⁸ ; Rossella Spataro²³ ; Alessandro Stecco²⁹ ; Alessandro Vercelli³⁰
• 关键词：ALS ; clinical trials ; stem cells ; transplantation
• 刊名：Cytotherapy
• 出版年：2016
• 出版时间：December 2016
• 年：2016
• 卷：18
• 期：12
• 页码：1471-1475
• 全文大小：136 K

文摘

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.