Bone marrow mesenchymal stem cell-derived extracellular vesicle infusion for amyotrophic lateral sclerosis

• Published in: Neurodegenerative disease management Vol. 14; no. 3-4; pp. 111 - 117
• Main Authors: Crose, Joshua J, Crose, Arezou, Ransom, John T, Lightner, Amy L
• Format: Journal Article
• Language: English
• Published: England Taylor & Francis 2024
• Subjects: Adult; Aged; Amyotrophic Lateral Sclerosis - therapy; Extracellular Vesicles; Female; Humans; Infusions, Intravenous; Male; Mesenchymal Stem Cell Transplantation - methods; Mesenchymal Stem Cells; Middle Aged; Pilot Projects; Rapid Communication; Short Communication; Treatment Outcome; ALS; amyotrophic lateral sclerosis; extracellular vesicle; bone marrow mesenchymal stem cell
• ISSN: 1758-2024; 1758-2032
• DOI: 10.1080/17582024.2024.2344396

In this pilot safety study, we hypothesized that a human bone marrow stem cell-derived extracellular vesicle (hBM-MSC EV) investigational product (IP) would be safe and exhibit potential efficacy in amyotrophic lateral sclerosis (ALS) patients. Ten ALS patients received two 10-ml intravenous infusions of the IP given 1 month apart and evaluated over 3 months. There were no serious adverse events or adverse events related to the IP and 30% of subjects' ALS functional rating scale-revised (ALSFRS-R) scores did not decline. HBM-MSC EVs appear safe in ALS patients. This early investigation suggests a controlled study of EVs for the treatment of ALS is warranted.