Intranasal Stem Cell Therapy Brings Hope to Newborns with Brain Damage

Oxygen deprivation during childbirth can result in serious brain injury in infants, potentially leading to long-term neurological issues. Recent advancements in stem cell research are paving the way for innovative treatments, with intranasal administration of stem cells showing promising potential.

At the University Medical Center Utrecht, a safety study named PASSIoN explored this novel approach. Involving ten newborns who experienced perinatal strokes—brain injuries occurring shortly before, during, or after birth—each received a single dose of mesenchymal stem cells via nasal drops within their first week of life. Follow-up assessments at two years showed encouraging results: most children developed typically, began walking earlier than similar untreated cases, and showed no motor impairments, epilepsy, or visual problems. The extent of brain tissue loss was smaller than anticipated, given the severity of their initial injuries.

While the primary goal was to evaluate safety, the outcomes exceeded expectations. Only two children exhibited mild cerebral palsy, notably lower than historical rates. All children initially showed damage to motor pathways, yet none developed epilepsy or vision issues. The positive developmental trajectory observed is considered highly remarkable by pediatric experts.

The findings, published in Stroke, do not yet confirm efficacy but provide critical insights. The researchers are preparing for the upcoming iSTOP-CP trial launching in early 2026, which will involve 162 newborns receiving either stem cells or a placebo, with their development tracked up to age two.

According to lead researcher Prof. Manon Benders, these results foster hope for future interventions, emphasizing the potential of stem cell therapy to revolutionize treatment for neonatal brain injuries. The ongoing research combines foundational lab work with clinical applications, aiming to enhance and optimize this promising therapy.

As the scientific community anticipates further evidence, this innovative approach could significantly change how brain injuries are managed in newborns, offering new hope to families affected by perinatal strokes.