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

  • N. Atassi
  • , E. Beghi
  • , M. Blanquer
  • , N. M. Boulis
  • , Roberto CANTELLO
  • , C. Caponnetto
  • , A. Chiò
  • , S. B. Dunnett
  • , E. L. Feldman
  • , A. Vescovi
  • , LETIZIA MAZZINI
  • , C. Bendotti
  • , Enrica BERSANO
  • , S. Brajkovic
  • , P. Car
  • , Fabiola DE MARCHI
  • , R. Fantozzi
  • , Antonia FOLLENZI
  • , M. Gelati
  • , C. Giorgi
  • Mariagrazia GRILLI, P. Guenzi, V. La Bella, G. L. Mancardi, G. Panzarasa, M. Poloni, D. Profico, V. Silani, G. Sorarù, R. Spataro, ALESSANDRO STECCO, A. Vercelli

Research output: Contribution to journalArticlepeer-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.
Original languageEnglish
Pages (from-to)1471-1475
Number of pages5
JournalCytotherapy
Volume18
Issue number12
DOIs
Publication statusPublished - 2016

Keywords

  • ALS
  • clinical trials
  • stem cells
  • transplantation

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