Advances in the clinical application of mesenchymal stem cells for neurological disorders

Abstract

Therapeutic approaches employing mesenchymal stem cells (MSCs) have emerged as a promising avenue for investigating treatments for neurological disorders. This strategy aims to capitalize on the biological properties of MSCs to support the repair of damaged neural tissue and modulate pathological processes. This review provides a comprehensive overview of the current clinical evidence regarding MSC applications in major neurological disorders, including Parkinson’s disease (PD), Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), stroke, spinal cord injury (SCI), and other pertinent disorders. Across clinical studies, MSC administration has generally demonstrated a favorable safety profile and procedural feasibility. However, therapeutic efficacy remains variable and inconsistent across trials. Importantly, differences in MSC sources, manufacturing procedures, delivery routes, dosing strategies, and patient selection contribute substantially to the heterogeneity of reported outcomes. Therefore, the current body of evidence supports the safety of MSC-based interventions, but their clinical effectiveness has not yet been consistently demonstrated. Future progress will depend largely on the standardization of cell preparation and treatment protocols, improved biomarker-driven patient stratification, and rigorously designed large-scale randomized trials.

1. Introduction

Neurological disorders, encompassing neurodegenerative diseases, traumatic CNS injuries, and neurodevelopmental abnormalities, constitute a major global health challenge and are among the principal causes of long-term disability and mortality [1, 2]. These conditions are commonly characterized by progressive neuronal dysfunction, persistent neuroinflammatory responses, and the inherently limited regenerative potential of the central nervous system, ultimately leading to permanent neurological impairment and considerable socioeconomic consequences [3]. Although substantial progress has been achieved in the clinical management of these disorders, existing therapeutic strategies remain predominantly palliative, offering symptomatic relief with limited ability to modify disease progression or promote meaningful restoration of damaged neural tissue and function [4, 5].

The pathogenesis of neurological disorders is highly complex and involves the interplay of several interconnected processes, including neuronal damage, dysregulated immune responses, oxidative stress, vascular dysfunction, and insufficient intrinsic repair mechanisms. This multifaceted nature has driven the development of therapeutic strategies that can simultaneously address multiple pathological pathways rather than targeting a single disease component. Among these approaches, cell-based therapies have gained considerable attention owing to their ability to exert broad biological effects, including modulation of the local microenvironment, enhancement of tissue repair, and attenuation of disease progression through diverse and complementary mechanisms [6–8].