Abstract
Amyotrophic Lateral Sclerosis (ALS) is a terminal neurodegenerative disease with only a few minimally effective treatment options. This study treats a patient with Spinal ALS with Bone Marrow Derived Mesenchymal Stem Cell Extracellular Vesicles (BM-MSCEVs) to slow or stop the progress of the disease. After 2 months and 4 treatments, the patient demonstrated improvement in ALS functional scores and most physical exam findings. BM-MSCEVs demonstrate the ability to effectively treat Spinal ALS in this study and open the door for future studies.
Highlights
Amyotrophic Lateral Sclerosis (ALS) is a devastating terminal disease that has perplexed medicine since its description in 1824 by Charles Bell
Research science is focusing on the loss of regulation and/or clearance of protein waste products, namely TDP-43, SOD1, and FUS [6, 7]
Dr WS is a 54 y/o Brazilian male with spinal ALS diagnosed 2 years prior without any other co-morbidities. He initially presented with weakness in his legs and arms four years ago that progressed to include muscle spasms and eventually loss of normal activities of daily living (ADLs)
Summary
Amyotrophic Lateral Sclerosis (ALS) is a devastating terminal disease that has perplexed medicine since its description in 1824 by Charles Bell. It was diagnosed and given a name in 1874 by Jean-Martin Charcot [1, 2]. 4.5 per 100,000 people are living with the disease but there has been little advancement in the understanding and treatment of it since its discovery [1, 2]. Most cases of ALS (90%-95%) are deemed sporadic and have no known cause but both genetic and environmental factors are believed to be involved. The remaining 5-10% of cases are classified as familial and have a genetic link to family history [3, 4, and 5]. Research science is focusing on the loss of regulation and/or clearance of protein waste products, namely TDP-43, SOD1, and FUS [6, 7]
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