Immunological aspects of mesenchymal stem cell therapy for ALS

Neuroinflammation contributes to ALS progression, yet effective immune-modulating therapies remain limited. Despite the enthusiasm regarding stem cell therapy as a means of regeneration, it is interesting that the first FDA approval for mesenchymal stem cells (MSC) was for immune modulation, graft versus host disease (GVHD), not rejuvenation. We focus here on the immunological mechanisms through which MSC, particularly via induction of regulatory T cells, may slow progression of amyotrophic lateral sclerosis (ALS), a uniformly fatal motor neuron disease. Clinical results to date have been mixed, and we argue that prospective monitoring of immune and blood-based biomarkers could sharpen patient selection, guide choice of cell type and adjuvant, and calibrate treatment intensity. MSC-induced Tregs may suppress neuroinflammation and offer a strategy to slow ALS progression.

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Publication Details

Journal
Journal of Translational Medicine
Published
2026-11-09
DOI
https://doi.org/10.1186/s12967-026-09086-x
Primary Topic
Mesenchymal stem cell research
Type
article
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article

Immunological aspects of mesenchymal stem cell therapy for ALS

Neil H Riordan, Thomas E. Ichim
Journal of Translational Medicine
Mesenchymal stem cell research
article

Immunological aspects of mesenchymal stem cell therapy for ALS

Neil H Riordan, Thomas E. Ichim
article en

Abstract

Neuroinflammation contributes to ALS progression, yet effective immune-modulating therapies remain limited. Despite the enthusiasm regarding stem cell therapy as a means of regeneration, it is interesting that the first FDA approval for mesenchymal stem cells (MSC) was for immune modulation, graft versus host disease (GVHD), not rejuvenation. We focus here on the immunological mechanisms through which MSC, particularly via induction of regulatory T cells, may slow progression of amyotrophic lateral sclerosis (ALS), a uniformly fatal motor neuron disease. Clinical results to date have been mixed, and we argue that prospective monitoring of immune and blood-based biomarkers could sharpen patient selection, guide choice of cell type and adjuvant, and calibrate treatment intensity. MSC-induced Tregs may suppress neuroinflammation and offer a strategy to slow ALS progression.

Journal of Translational Medicine
Healthcentric Advisors (US), Medistem (Panama) (PA)
Mesenchymal stem cell research
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