What question did this study set out to answer?

This study aims to evaluate the efficacy of mesenchymal stem cell-derived exosomes in patients with neuromuscular and neurodegenerative disorders like ALS, LBD, and PD.

May 20, 2026International Journal of Molecular Sciences0 citationsOpen Access

The Molecular Basis of Partial Reversal or Significant Slowing of ALS, Parkinson’s Disease, and Lewy Body Dementia by Mesenchymal Exosomes/Secretome

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CPChadwick C. ProdromosStem Cell Institute RVRuby Del VillarStem Cell Institute ASAndrew StriegelStem Cell Institute

Key Points

This study aims to evaluate the efficacy of mesenchymal stem cell-derived exosomes in patients with neuromuscular and neurodegenerative disorders like ALS, LBD, and PD.
Conducted a case-controlled series experiment with 86 patients with ALS, LBD, or PD.
Administered intranasal stem cell-derived exosomes/secretome products to evaluate clinical response.
Followed patients at one week and one, two, three, and six months post-treatment.
77% of patients (67 of 86) showed a positive clinical response to at least one MSC-derived product.
Outcomes were linked with greater treatment frequency, especially for ALS and LBD.
No serious adverse events reported, indicating a favorable safety profile.

Abstract

Neuromuscular and neurodegenerative (NMND) disorders are diseases that cause progressive damage to the central nervous system leaving patients with symptoms that negatively affect everyday living with death almost inevitable. These include amyotrophic lateral sclerosis (ALS), Lewy body dementia (LBD), and Parkinson’s disease (PD) with cases expected to increase in the future. Intranasally administered stem cell-derived exosomes/secretome have been seen as potential therapeutic options for these disorders in preclinical animal models. This study sought to observe the efficacy of mesenchymal stem cell-derived exosomes/secretome in patients with ALS, LBD, and PD. Based off these preclinical studies, we conducted a case-controlled series experiment with 86 patients with ALS, LBD, or PD, with the independent variable being the treatment and the dependent variable being the clinical response. These patients were recruited and given intranasal instillations of various MSC-derived exosome/secretome products. Subsequent treatments were given to patients who did not have a response to one product. Patients were followed up at one week, one, two, three, and six months post-treatment. Historical external controls were used for comparison to clinical outcomes. There were no serious adverse events in any patient. A total of 67 of 86 (77%) patients showed a positive clinical response to at least one product. Outcomes were strongly associated with greater treatment frequency for ALS and LBD. Intranasal administration of MSC-derived exosome/secretome products were safe, and most patients showed overall improvement with at least one product. Some patients also saw a substantial decrease in the rate of decline compared to historical controls. These results also give rise to the hypothesis: do MSC-derived exosomes/secretome treatments show efficacy in other NMND disorders? The primary limitation of this study is the 6-month follow-up.

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Cite This Study

Prodromos et al. (Sat,) studied this question.

synapsesocial.com/papers/6a0d50f3f03e14405aa9d10a https://doi.org/https://doi.org/10.3390/ijms27104483

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