Neurology - Research & Surgery
Open AccessSafety and Efficacy of Stem Cell Therapy on the Treatment for Multiple Sclerosis
Authors: Jonathan RT Lakey, Krista Casazza, Juan MA Garcia-Lara, Virgilio Alejandro Hernandez Ruiz, Diana Andrade, Araceli Medina, Andrea Gutierrez-Contreras, Brad Robinson, Pedro Gutierrez- Castrellon.
Abstract
Background: Multiple sclerosis (MS) is a chronic autoimmune demyelinating disease of the central nervous system characterized by inflammation, axonal injury, and progressive neurological decline. Current disease-modifying therapies (DMTs) reduce relapse rates but remain insufficient for halting neurodegeneration or promoting repair, particularly in progressive MS. Stem cell–based therapies have emerged as promising strategies by integrating immunomodulation, neuroprotection, and remyelination.
Objective: This review critically evaluates the therapeutic potential, mechanisms of action, clinical evidence, and translational challenges of stem cell–based interventions for MS.
Methods: A comprehensive synthesis of preclinical and clinical studies was performed, emphasizing randomized controlled trials, systematic reviews, and meta-analyses evaluating autologous hematopoietic stem cell transplantation (aHSCT), mesenchymal stem cells (MSCs), neural stem/progenitor cells (NSPCs), and induced pluripotent stem cells (iPSCs).
Results: aHSCT achieves durable immune “resetting” and superior relapse and disability outcomes compared with high-efficacy DMTs in aggressive relapsing MS, albeit with significant procedure-related risks. MSCs exhibit favorable safety and potent paracrine immunomodulatory and neuroprotective actions; however, efficacy signals across trials remain inconsistent, underscoring the need for standardized dosing, delivery routes, and larger multicenter studies. Early investigations of NSPCs and iPSC-derived oligodendrocyte progenitors demonstrate feasibility and remyelinating potential but remain preclinical or early phase. Across modalities, mechanistic studies highlight immunoregulation, trophic support, microglial modulation, and restoration of metabolic resilience as central pathways. Emerging biomarkers, including neurofilament light (NfL), glial fibrillary acidic protein (GFAP), and advanced imaging metrics, are being integrated to establish causal links between biological effect and clinical outcome.
Conclusions: Stem cell therapies represent a transformative avenue for MS treatment, offering potential beyond immunosuppression to promote central repair. aHSCT provides high-potency immune reconstitution but at higher procedural risk, while MSCs deliver safer, paracrine-driven neurorepair with unresolved efficacy. Translation to clinical practice will require harmonized manufacturing
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