Related Experiment Video
Updated: Jul 10, 2026
07:56
Transient Middle Cerebral Artery Occlusion Model of Neonatal Stroke in P10 Rats
Published on: April 21, 2017
10.7K
Perinatal Arterial Stroke Treated With Stromal Cells Intranasally: 2-Year Safety and Neurodevelopment
Nienke Wagenaar1, Lisanne M Baak1, Niek E van der Aa1
1Department of Neonatology (N.W., L.M.B., N.E.v.d.A., F.G., J.D., M.L.T., C.K., L.S.d.V., F.v.B., M.J.N.L.B.), University Medical Center Utrecht Brain Center and Wilhelmina Children's Hospital, Utrecht University, the Netherlands.
Stroke
|July 14, 2025
Summary
Intranasal mesenchymal stromal cell (MSC) therapy appears safe for infants with perinatal arterial ischemic stroke, showing promising long-term motor improvements. Further trials are needed to confirm efficacy in this vulnerable population.
Area of Science:
- Neuroscience
- Regenerative Medicine
- Pediatric Neurology
Background:
- Perinatal arterial ischemic stroke (PAIS) is a leading cause of childhood neurological disability.
- Mesenchymal stromal cells (MSCs) offer potential therapeutic benefits for neuroprotection and repair.
- The PASSIoN study previously established short-term safety and feasibility of intranasal MSCs for PAIS.
Purpose of the Study:
- To assess the long-term safety and neurodevelopmental outcomes of intranasal MSC therapy in neonates with PAIS.
- To compare neuroimaging and clinical outcomes between PASSIoN participants and a historical, non-MSC-treated cohort.
Main Methods:
- Post hoc analysis of 10 infants from the PASSIoN study receiving intranasal MSCs.
- Evaluation of brain tissue loss via MRI at 3 months.
- Assessment of neurodevelopmental outcomes (cerebral palsy, motor/cognitive delays, behavioral issues, epilepsy) at 2 years.
- Comparison with a registry cohort (N=39) meeting PASSIoN inclusion criteria.
Main Results:
- No adverse events related to MSC therapy were reported up to 2 years of age.
- PASSIoN participants showed significantly less asymmetry in key brain structures (internal capsule, cerebral peduncle) on MRI compared to the registry cohort.
- Improved motor performance (Z score) was observed in PASSIoN participants versus the registry cohort at 2 years.
- 20% developed mild cerebral palsy (GMFCS I) without motor delays; cognitive/behavioral issues affected 10-20%.
Conclusions:
- Intranasal MSC therapy demonstrates long-term safety in infants with PAIS.
- The therapy may be associated with improved motor outcomes and reduced brain structure asymmetry.
- Randomized controlled trials are essential to validate the efficacy of MSCs for PAIS.
