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 Vescovi; Letizia Mazzini; 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?

Cytotherapy. 2016 Dec;18(12):1471-1475. doi: 10.1016/j.jcyt.2016.08.005. Epub 2016 Oct 6.

Collaborators

attendees of the International Workshop on Progress in Stem Cells Research for ALS/MND:
Caterina Bendotti¹², Enrica Bersano⁵, Simona Brajkovic¹³, Piergiorgio Car¹⁴, Fabiola De Marchi⁵, Roberto Fantozzi¹⁵, Antonia Follenzi¹⁶, Maurizio Gelati¹⁷, Cesare Giorgi¹⁸, Mariagrazia Grilli¹⁹, Pierdavide Guenzi²⁰, Vincenzo La Bella²¹, Gian Luigi Mancardi²², Gabriele Panzarasa¹³, Marco Poloni², Daniela Profico²³, Vincenzo Silani²⁴, Gianni Sorarù²⁵, Rossella Spataro²¹, Alessandro Stecco²⁶, Alessandro Vercelli²⁷

Affiliations

¹ Neurological Clinical Research Institute (NCRI), Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA.
² Department of Neuroscience, IRCCS-Institute for Pharmacological Research "Mario Negri", Milan, Italy.
³ Transplant and Cell Therapy Unit, "Virgen de la Arrixaca" University Hospital-IMIB, University of Murcia, Murcia, Spain.
⁴ Department of Neurosurgery, Emory University School of Medicine, Atlanta, Georgia, USA.
⁵ Department of Neurology, Eastern Piedmont University, Novara, Italy.
⁶ U.O. Neurology of IRCCS AOU S. Martino-IST, Largo R. Benzi, Genoa, Italy.
⁷ ALS Center, "Rita Levi Montalcini", Department of Neuroscience, Neurology II, University of Torino, Turin, Italy.
⁸ Brain Repair Group, School of Biosciences, Cardiff University, Museum Avenue, Cardiff, United Kingdom.
⁹ Department of Neurology, University of Michigan, Ann Arbor, Michigan, USA.
¹⁰ IRCCS "Casa Sollievo della Sofferenza", San Giovanni Rotondo, Italy; Biotechnology and Bioscience Department, Bicocca University, Milano, Italy.
¹¹ ALS Centre, Department of Neurology, "Maggiore della Carità", University Hospital, Novara, Italy. Electronic address: mazzini.l@libero.it.
¹² Laboratory of Molecular Neurobiology, IRCCS-Istituto di Ricerche Farmacologiche Mario Negri, Milano, Italy.
¹³ Department of Neuroscience, IRCCS Ca' Grand-Ospedale Maggiore Policlinico, Milano, Italy.
¹⁴ Department of Neurosurgery, Maggiore della Carità University Hospital, Novara, Italy.
¹⁵ Department of Pharmacology, University of Torino, Torino, Italy.
¹⁶ Department of Health Sciences, Università del Piemonte Orientale, Novara, Italy.
¹⁷ Laboratorio Cellule Staminali, Cell Factory e Biobanca, Terni, Italy.
¹⁸ Department of Neuroscience, "Santa Maria" Hospital, Terni, Italy.
¹⁹ Laboratory of Neuroplasticity, Department of Pharmaceutical Sciences, University of Piemonte Orientale, Novara, Italy.
²⁰ Theology Department, Catholic University, Milan, Italy.
²¹ ALS Clinical Research Centre, Department of Experimental Biomedicine and Clinical Neurosciences (BioNeC), University of Palermo, Palermo, Italy.
²² Neuroscience Institute, University of Genova, Genova, Italy.
²³ Department of Health Sciences, Università del Piemonte Orientale, Novara, Italy; IRCCS "Casa Sollievo della Sofferenza", San Giovanni Rotondo, Italy.
²⁴ Department of Neurology and Laboratory of Neuroscience, IRCCS Istituto Auxologico Italiano, Milano, Italy.
²⁵ Department of Neurosciences, University of Padova, Padova, Italy.
²⁶ Department of Diagnostic and Interventional Radiology, University of Piemonte Orientale, Novara, Italy.
²⁷ Department of Neuroscience, Neuroscience Institute Cavalieri Ottolenghi, Torino, Italy.

PMID: 27720637
DOI: 10.1016/j.jcyt.2016.08.005

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.

MeSH terms

Adolescent
Adult
Aged
Amyotrophic Lateral Sclerosis / therapy*
Cell- and Tissue-Based Therapy / methods*
Clinical Trials, Phase II as Topic / methods*
Clinical Trials, Phase III as Topic / methods*
Consensus
Humans
Middle Aged
Neural Stem Cells / transplantation
Safety
Stem Cell Transplantation*
Young Adult