Level D· Preclinical EvidenceAnimal Study

Safety and biodistribution of intrathecal administration of mesenchymal stem cells (MSCs) and neurotrophin-releasing nanoparticles in a porcine CSF-guided delivery model for amyotrophic lateral sclerosis (ALS) drug disco

Sinderewicz E., Dąbkowska M., Sarnowska A., Staszkiewicz-Chodor J., Mystkowska D., Holak P.

Animal Study on Neuroinflammation, Chronic Inflammation, published in Sci Rep (2026) — summary generated from the PubMed abstract.

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Study type
Animal Study
Journal
Sci Rep (2026)
Country
England
Reported sample size
PMID
41760732
DOI
10.1038/s41598-026-40196-0

Abstract (original English)

Amyotrophic lateral sclerosis (ALS) is a multifactorial neurodegenerative disorder that complicates the identification of effective therapeutic targets. The potential of stem cells and neurotrophins as promising candidates has become increasingly evident, owing to their neuroprotective and anti-inflammatory properties. In this study, a preclinical evaluation of the safety and biodistribution of mesenchymal stromal/stem cells (MSCs) combined with neurotrophin-releasing polyelectrolyte nanoparticles (NTs) was conducted in a porcine intrathecal delivery model relevant to ALS therapy development. Four groups of male pigs were administered saline with NTs, adipose-derived stem cells (ASCs) with NTs, Wharton's jelly-derived MSCs (WJ-MSCs) with NTs, or spinal puncture only. The safety of the treatment was assessed using magnetic resonance imaging (MRI), haematological and biochemical analyses, cerebrospinal fluid profiling, and histology. No adverse effects or significant systemic alterations were observed. It is noteworthy that C-reactive protein levels diminished following NT and NT-MSC administration, suggesting a systemic anti-inflammatory effect. The migration of MSCs was facilitated by cerebrospinal fluid, leading to their accumulation around the spinal cord and brain parenchyma. The present findings demonstrate short-term safety and biodistribution patterns following intratheca

What this study does not prove

  • This study does not prove SVF is an approved treatment or a replacement for standard care.
  • This is preclinical work; animal or laboratory results cannot be applied to humans.

Evidence level

Evidence comes from animal or laboratory studies and has not been confirmed in humans.

How we grade evidence
AnimalsAmyotrophic Lateral SclerosisNanoparticlesMesenchymal Stem Cell TransplantationMesenchymal Stem CellsMaleNerve Growth FactorsSwineTissue DistributionInjections, Spinal

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