Case Report: Amyotrophic Lateral Sclerosis Treatment with Extracellular Vesicles derived from Mesenchymal Stromal Cells

Abstract

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder marked by motor neuron loss, resulting in muscle weakness, respiratory failure, and death. Current treatment options offer limited survival benefit, highlighting the urgent need for novel therapeutic strategies. Extracellular vesicles (EVs), particularly those derived from human bone marrow, have shown promise in preclinical models due to their anti-inflammatory and neuroprotective properties. We report the case of an 81-year-old male with a two-year history of ALS who received a novel investigational therapy consisting of intravenous infusions of human bone marrow-derived EVs. At baseline, the patient exhibited bulbar and respiratory involvement, as well as progressive weakness in the upper and lower extremities, and significant impairment in activities of daily living. Pulmonary function testing revealed a forced vital capacity (FVC) of 52% predicted, and the patient relied on non-invasive ventilation for up to 9 hours nightly. The treatment was well-tolerated with no adverse events reported. Clinical monitoring showed stabilization of respiratory function, maintained ambulatory ability for short distances, and no further significant decline in bulbar or limb strength over the treatment period. Subjective improvements were noted in energy levels, mood, and overall quality of life, though these outcomes require further investigation in controlled studies.