title: Intraspinal stem cell transplantation for amyotrophic lateral sclerosis: Ready for efficacy clinical trials?
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.
authors.affiliation: Neurological Clinical Research Institute (NCRI), Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA.
authors.forename: Nazem
authors.initials: N
authors.lastname: Atassi
authors.affiliation: Department of Neuroscience, IRCCS-Institute for Pharmacological Research "Mario Negri", Milan, Italy.
authors.forename: Ettore
authors.initials: E
authors.lastname: Beghi
authors.affiliation: Transplant and Cell Therapy Unit, "Virgen de la Arrixaca" University Hospital-IMIB, University of Murcia, Murcia, Spain.
authors.forename: Miguel
authors.initials: M
authors.lastname: Blanquer
authors.affiliation: Department of Neurosurgery, Emory University School of Medicine, Atlanta, Georgia, USA.
authors.forename: Nicholas M
authors.initials: NM
authors.lastname: Boulis
authors.affiliation: Department of Neurology, Eastern Piedmont University, Novara, Italy.
authors.forename: Roberto
authors.initials: R
authors.lastname: Cantello
authors.affiliation: U.O. Neurology of IRCCS AOU S. Martino-IST, Largo R. Benzi, Genoa, Italy.
authors.forename: Claudia
authors.initials: C
authors.lastname: Caponnetto
authors.affiliation: ALS Center, "Rita Levi Montalcini", Department of Neuroscience, Neurology II, University of Torino, Turin, Italy.
authors.forename: Adriano
authors.initials: A
authors.lastname: Chiò
authors.affiliation: Brain Repair Group, School of Biosciences, Cardiff University, Museum Avenue, Cardiff, United Kingdom.
authors.forename: Stephen B
authors.initials: SB
authors.lastname: Dunnett
authors.affiliation: Department of Neurology, University of Michigan, Ann Arbor, Michigan, USA.
authors.forename: Eva L
authors.initials: EL
authors.lastname: Feldman
authors.affiliation: IRCCS "Casa Sollievo della Sofferenza", San Giovanni Rotondo, Italy; Biotechnology and Bioscience Department, Bicocca University, Milano, Italy.
authors.forename: Angelo
authors.initials: A
authors.lastname: Vescovi
authors.affiliation: ALS Centre, Department of Neurology, "Maggiore della Carità", University Hospital, Novara, Italy. Electronic address: mazzini.l@libero.it.
authors.forename: Letizia
authors.initials: L
authors.lastname: Mazzini
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000690:Amyotrophic Lateral Sclerosis / Q000628:therapy*; D064987:Cell- and Tissue-Based Therapy / Q000379:methods*; D017322:Clinical Trials, Phase II as Topic / Q000379:methods*; D017326:Clinical Trials, Phase III as Topic / Q000379:methods*; D032921:Consensus; D006801:Humans; D008875:Middle Aged; D058953:Neural Stem Cells / Q000637:transplantation; D012449:Safety; D033581:Stem Cell Transplantation; D055815:Young Adult
keywords: ALS; clinical trials; stem cells; transplantation