Testing the Safety of Stem Cells to Treat Progressive MS


An international research team investigated the safety of human neural stem cell transplantation for the treatment of secondary progressive multiple sclerosis (SPMS) in a phase I clinical trial. In this first-in-human study, they found the treatment approach to be safe and tolerable with no reported adverse effects. They also found that the participants’ disease stabilized, with no significant changes in clinical disability and cognitive functionin the 12-month follow-up period. While these results are encouraging, larger clinical trials are needed to better understand the treatment effect in people with MS. 


There is much interest in the use of stem cells as a treatment for MS. Stem cells may be able to better target the inflammation taking place in the brain and spinal cord and may better facilitate repair of existing damage. There is encouraging data from stem cell use in animal models, but additional clinical trials in humans are needed.  


This first-in-human phase I clinical trial aimed to determine the feasibility, safety and tolerability of human neural stem cell transplants for the treatment of secondary progressive MS (SPMS). The stem cells used were allogenic, provided by a single donor, and participants were given different dosages (5, 10, 16, 24 million cells) in each treatment arm.

This small study enrolled 15 people with SPMS (both active and non-active). Participants were treated with human neural stem cells delivered by injection into the brain in addition to receiving an immunosuppressive treatment. The participants were then followed for a 12-month period. 


The researchers found the delivery of human neural stem cells in people with SPMS was safe and well tolerated. There were no adverse events (e.g. no treatment related-deaths or significant health issues) reported following treatment. 

While this study was not designed to understand the efficacy of the treatment, participants did not report changes in clinical disability and cognitive function. They also did not report any relapses in the 12-month follow-up. These are encouraging signs and point to stability of the disease without progression.


The results from this safety study are positive for future stem cell and regenerative medicine treatments in MS. Future clinical trials (phase 2 and 3) with larger numbers of participants and controls are necessary to assess the efficacy of this treatment for MS. 


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. Phase I clinical trial of intracerebroventricular transplantation of allogeneic neural stem cells in people with progressive Multiple SclerosisCell Stem Cell. (2023). Link to article – here.


For more information on stem cells – refer here.