Phase I clinical trial of intracerebroventricular transplantation of allogeneic neural stem cells in people with progressive multiple sclerosis
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.PMID:38016468 | DOI:10.1016/j.stem.2023.11.001
Source: Cell Stem Cell - Category: Stem Cells Authors: Maurizio A Leone Maurizio Gelati Daniela C Profico Claudio Gobbi Emanuele Pravat à Massimiliano Copetti Carlo Conti Lucrezia Abate Luigi Amoruso Francesco Apollo Rosario F Balzano Ilaria Bicchi Massimo Carella Alessandro Ciampini Carlo Colosimo Paola Cro Source Type: research
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