An international team tested a novel strategy to transplant nerve stem cells directly into the brains of 15 people with secondary progressive
multiple sclerosis. No serious adverse events occurred, and participants’ physical and cognitive functions remained stable for one year afterward. Further study is necessary to determine the potential of this strategy for stopping MS progression and restoring function in people with MS.
Background: Stem cell therapy
is any treatment that uses or targets stem cells, which are the types of cells that can turn into many different specialized cells. Many types of stem cells are being explored for their potential benefits for treating multiple sclerosis and continued research will determine the effectiveness and safety of these approaches for treating MS.
Dr. Stefano Pluchino and other investigators at the University of Cambridge, University of Milan Bicocca and Hospital Casa Sollievo della Sofferenza recruited 15 people with active or inactive secondary progressive MS. This was an open label study, meaning that there was no “control” group to which cell therapy was compared.
The researchers transplanted nerve stem cells obtained from a donor directly into the brains of participants, and participants also were treated with oral and intravenous medication to suppress the immune system, which might react to the presence of “foreign” cells. The primary goal of the study was to determine whether there were any safety concerns in this group, who were followed for one year after undergoing the procedure. Secondary outcomes explored included how the treatment affected physical and cognitive function, using questionnaires, MRI scans and blood tests.
There were no serious adverse events reported from this study. One seizure occurred, and it is unclear if it was related to the procedure. The seizure, as well as other adverse events that were caused by the immune-suppressing treatments, were effectively treated.
Physical and cognitive function remained stable throughout the year.
Why does this matter?
This is a small, early study of a novel experimental strategy with the potential to stop MS progression and promote tissue regeneration. Although the results suggest safety and hints of benefits, much further study is needed to determine what the optimal cells, delivery methods, safety and actual effectiveness of these experimental cell therapies might be for different people with MS. The authors comment that a strategy that involves a person’s own adult stem cells would be preferable, to avoid the potential adverse effects of dealing with donor tissue. These researchers and other researchers are continuing to work on this strategy.
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“Phase I clinical trial of intracerebroventricular transplantation of allogeneic neural stem cells in people with progressive Multiple Sclerosis
” by 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, and Angelo L. Vescovi is published in Cell Stem Cell
(published online November 27, 2023).