Phase I clinical trial of intracerebroventricular transplantation of allogeneic neural stem cells in people with progressive multiple sclerosis
Year: 2023
Type: article
Study design: Clinical Trial
Abstract: We report the analysis of 1 year of data from the first cohort of 15 patients enrolled in an open-label, first-in-human, dose-escalation phase I study (ClinicalTrials.gov: NCT03282760, EudraCT2015-004855-37) to determine the feasibility, safety, and tolerability of the transplantation of allogeneic human neural stem/progenitor cells (hNSCs) for the treatment of secondary progressive multiple sclerosis. Participants were treated with hNSCs delivered via intracerebroventricular injection in combination with an immunosuppressive regimen. No treatment-related deaths nor serious adverse events (AEs) were observed. All participants displayed stability of clinical and laboratory outcomes, as well as lesion load and brain activity (MRI), compared with the study entry. Longitudinal metabolomics and lipidomics of biological fluids identified time- and dose-dependent responses with increased levels of acyl-carnitines and fatty acids in the cerebrospinal fluid (CSF). The absence of AEs and the stability of functional and structural outcomes are reassuring and represent a milestone for the safe translation of stem cells into regenerative medicines.
Source: Cell stem cell
Authors: Maurizio A. Leone, Maurizio Gelati, Daniela C. Profico, Claudio Gobbi, Emanuele Pravatà, Massimiliano Copetti, Carlo Conti, Lucrezia Abate, Luigi Amoruso, Francesco Apollo, Rosario F. Balzano, Ilaria Bicchi, Massimo Carella, Alessandro Ciampini, Carlo Colosimo, Paola Crociani, Giada D’Aloisio, Pietro Di Viesti, Daniela Ferrari, Danilo Fogli, Andrea Fontana, Domenico Frondizi, Valentina Grespi, Jens Kuhle, Antonio Laborante, Ivan Lombardi, Gianmarco Muzi, Francesca Paci, Giuliana Placentino, Teresa Popolizio, Claudia Ricciolini, Simonetta Sabatini, Giada Silveri, Cristina Spera, Daniel Stephenson, Giuseppe Stipa, Elettra Tinella, Michele Zarrelli, Chiara Zecca, Yendri Ventura, Angelo D’Alessandro, Luca Peruzzotti-Jametti, Stefano Pluchino, Angelo L. Vescovi
Institutions: Casa Sollievo della Sofferenza, Università della Svizzera italiana, University of Colorado Anschutz, Azienda Ospedaliera S.Maria, University of Milano-Bicocca, University of Basel, University Hospital of Basel, Abu Dhabi Stem Cells Center, University of Cambridge, National Institute for Health and Care Research, NIHR Cambridge Biomedical Research Centre, Imperial College London
Language: English
Subfield: Clinical Neurology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $9,080
OpenAlex ID: W4389047249