Autologous Mesenchymal Stem Cell Transplantation in Amyotrophic Lateral Sclerosis: A Case Report

INTRODUCTION AND IMPORTANCE Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease with limited treatment options, leading to severe motor impairment and premature death. Current therapies provide only marginal benefits, necessitating novel approaches. Mesenchymal stem cells (MSCs) have gained interest due to their immunomodulatory, neuroprotective, and regenerative potential. CASE PRESENTATION This study assesses the safety and therapeutic potential of MSC transplantation in a 52-year-old ALS patient, evaluating functional changes and disease progression. The patient underwent two MSC transplantations via intrathecal and intravenous administration. Clinical evaluations, electromyography (EMG), and ALS Functional Rating Scale-Revised (ALSFRS-R) assessments were conducted over 5 months post-treatment. CLINICAL DISCUSSION Motor function improved post-transplantation, with increased limb mobility and better swallowing. The ALSFRS-R score, which typically declines in ALS, showed a 15% increase, suggesting possible stabilization. EMG revealed preserved sensory fibers despite ongoing motor neuron degeneration. No severe adverse effects were observed. CONCLUSION These findings suggest that MSC may have a disease-modifying effect in ALS, offering both functional and emotional benefits. However, this case report has inherent limitations, including the lack of a control group and a small sample size. Key learning points include the safety and tolerability of MSC, its potential to improve motor and bulbar function, and the importance of combining MSC with physical and neurorehabilitation therapies. Future research should focus on larger, controlled trials to confirm efficacy, optimize treatment protocols, and identify patient subgroups most likely to benefit. This case highlights the potential of MSC as a promising therapeutic approach for ALS, warranting further investigation.