Intranasal Stem Cell Therapy for Perinatal Arterial Ischemic Strokes

Strokes are the third most common cause of death in adults in the world, and an important cause of mortality and chronic neurological morbidity in children. 

Arterial ischemic stroke has emerged as an important cause of neurological disability in children. The reported annual incidence ranges from 1.2 to 8 per 100,000 children and 1 per 2500–4000 live births for neonates. It is also the leading known cause of cerebral palsy.

Perinatal arterial ischemic stroke (PAIS) is defined as a focal disruption of cerebral blood flow occurring between 20 weeks of gestation and postnatal day 28. Because the exact timing of the stroke usually is not clear, ischemic perinatal stroke is defined according to gestational age or postnatal age at diagnosis.

In a recently published study, researchers evaluated the use of intranasally delivered bone marrow-derived allogenic mesenchymal stem cells to treat PAIS in neonates. The study appears in the journal The Lancet. 

Study Development and Results

For the study the team included neonates born at full term (≥36 weeks of gestation) with MRI-confirmed PAIS in the middle cerebral artery region. The study included a total of 10 neonates that received one dose of 45-50 Million bone marrow-derived MSCs intranasally within 7 days of presenting signs of PAIS. 

After the procedure the team found that MRI scans did not show unexpected structural cerebral abnormalities. All patients had initial pre-Wallerian changes in the corticospinal tracts, but only 4 showed asymmetrial corticospinal tracts at follow-up MRI scans. 

According to the researchers, this is the first in its kind study that demonstrates that intranasal bone marrow-derived MSCs administration in neonates after PAIS is feasible and had no serious adverse events. 


Daniela Munoz, et al. Risk Factors for Perinatal Arterial Ischemic Stroke. 2018. Cell Med.  doi: 10.1177/2155179018785341. 

Lisanne M Baak, et al. Feasibility and safety of intranasally administered mesenchymal stromal cells after perinatal arterial ischaemic stroke in the Netherlands (PASSIoN): a first-in-human, open-label intervention study. 2022. The Lancet. DOI: 

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