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

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 infusio...

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Published inNeurodegenerative 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
LanguageEnglish
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
Online AccessGet full text
ISSN1758-2024
1758-2032
DOI10.1080/17582024.2024.2344396

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Abstract 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.
AbstractList 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.
Background: 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. Methods: Ten ALS patients received two 10-ml intravenous infusions of the IP given 1 month apart and evaluated over 3 months. Results: 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. Conclusion: HBM-MSC EVs appear safe in ALS patients. This early investigation suggests a controlled study of EVs for the treatment of ALS is warranted. Amyotrophic lateral sclerosis (ALS) is a nervous system disease that affects the brain and spinal cord, causing the loss of muscle control. Currently, there is no cure for ALS and the disease gets worse over time. A potential new treatment is being investigated using mesenchymal stem cell extracellular vesicles (MSC EVs). MSC EVs are small structures that contain useful molecules and proteins that can be transported to cells affected by the disease, helping to reduce inflammation and encouraging repair. This 3-month study looked at the safety of human bone marrow MSC-EVs (hBM-MSC EVs) given as treatment to ten ALS patients, as well as how well it worked at delaying worsening of the disease. They found that there were no serious side effects caused by the treatment and that hBM-MSC EVs may have the potential for delaying the progression of ALS. This indicates that more, larger studies need to be carried out to find out treatment specifics, such as dose (how much of the treatment to give) and frequency (how often to give the treatment), and how they could be related to patient outcomes.
Author Crose, Joshua J
Crose, Arezou
Ransom, John T
Lightner, Amy L
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amyotrophic lateral sclerosis
extracellular vesicle
bone marrow mesenchymal stem cell
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Snippet 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...
Background: In this pilot safety study, we hypothesized that a human bone marrow stem cell-derived extracellular vesicle (hBM-MSC EV) investigational product...
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StartPage 111
SubjectTerms 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
Title Bone marrow mesenchymal stem cell-derived extracellular vesicle infusion for amyotrophic lateral sclerosis
URI https://www.ncbi.nlm.nih.gov/pubmed/39352708
https://pubmed.ncbi.nlm.nih.gov/PMC11457643
Volume 14
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