Repair of neonatal brain injury: bringing stem cell-based therapy into clinical practice

Hypoxic-ischaemic brain injury is one of most important causes of neonatal mortality and long-term neurological morbidity in infants born at term. At present, only hypothermia in infants with perinatal hypoxic-ischaemic encephalopathy has shown benefit as a neuroprotective strategy. Otherwise, current treatment options for neonatal brain injury mainly focus on controlling (associated) symptoms. Regeneration of the injured neonatal brain with stem cell-based therapies is emerging and experimental results are promising. At present, increasing efforts are made to bring stem cell-based therapies to the clinic. Among all progenitor cell types, mesenchymal stromal (stem) cells seem to be most promising for human use given their neuroregenerative properties and favourable safety profile. This review summarizes the actual state, potential hurdles and possibilities of stem cell-based therapy for neonatal brain injury in the clinical setting. An early version of this paper was presented at the Groningen Early Intervention Meeting which was held in April 2016.