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

Nazem Atassi, Ettore Beghi, Miguel Blanquer, Nicholas M. Boulis, Roberto Cantello, Claudia Caponnetto, Adriano Chiò, Stephen B. Dunnett, Eva L. Feldman, Angelo Luigi Vescovi, Letizia Mazzini, Caterina Bendotti, Enrica Bersano, Simona Brajkovic, Pier Giorgio Car, Fabiola De Marchi, Roberto Fantozzi, A. Follenzi, Maurizio Gelati, Cesare GiorgiMariagrazia Grilli, Pierdavide Guenzi, Vincenzo La Bella, Gianluigi Mancardi, Gabriele Panzarasa, Marco Poloni, Daniela Celeste Profico, Vincenzo Silani, G. Soraru, Rossella Spataro, Alessandro Stecco, Alessandro Vercelli

Research output: Contribution to journalArticlepeer-review


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
Issue number12
Publication statusPublished - Dec 1 2016


  • ALS
  • clinical trials
  • stem cells
  • transplantation

ASJC Scopus subject areas

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


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