While treatments to prevent and treat neonatal brain injury remain scarce, several therapies that showed early promise are under further investigation, according to a recently published review.
The review, published in the journal Clinics in Perinatology by researchers at Harvard Medical School and Boston Children’s Hospital, surveyed the drugs and cell-based therapies being tested to prevent and treat neonatal brain injury.
The condition, which refers to damage to a baby’s brain around the time of birth, is one of the leading causes of newborn death and lifelong disability worldwide and can follow oxygen deprivation, stroke or bleeding in the brain.
Current care is limited to two measures: therapeutic hypothermia (TH) and antenatal magnesium.TH, or cooling a baby for about 72 hours after birth, is used for full-term babies with moderate to severe brain injury from a lack of oxygen and blood flow around the time of birth. Antenatal magnesium, given to mothers at risk of very premature delivery, lowers the baby’s risk of cerebral palsy.
Researchers reviewed experimental options, most prominently stem cell-based therapies, which have shown significant promise in laboratory and animal models. Stem cells are immature cells that can develop into different cell types, such as those derived from the umbilical cord or bone marrow, and may help protect or repair damaged tissue.
Early studies have since tested stem cells in newborns whose brain injury arose from different causes, including a lack of oxygen, stroke and bleeding in the brain, with no serious adverse events attributed to the treatment.
Although the review does not address fetal and neonatal alloimmune thrombocytopenia (FNAIT) directly, the condition is a recognized cause of bleeding in the newborn brain. In FNAITFetal and neonatal alloimmune thrombocytopenia A rare condition in which a mother’s immune system attacks fetal platelets, leading to dangerously low platelet levels before and/or after birth., the mother’s immune system produces antibodies that cross the placenta and destroy the baby’s platelets, the cell fragments that help blood clot. The resulting shortage can lead to bleeding, and bleeding in the brain, or intracranial hemorrhageBrain bleed Bleeding inside the fetal brain, also known as an intracranial hemorrhage. In FNAIT, it's often caused by severe thrombocytopenia and can lead to long-term disability or death. , is the disorder’s most serious complication.
Learn more about FNAIT signs and symptoms
Not every promising drug has held up after further testing. Erythropoietin, a hormone that stimulates red blood cell production, and darbepoetin, a longer-acting version of it, both failed to improve long-term development in full-term or premature babies in recent large trials. Erythropoietin also carries a higher rate of serious side effects.
Besides stem cell-based therapies, the authors pointed to melatonin, widely recognized as a sleep supplement, and the anti-seizure medicine topiramate as the approaches most likely to succeed and closest to clinical use.
They called for both new therapies and broader access to existing ones, especially in low-resource settings, describing these as urgent steps toward better care and neurological outcomes for newborns worldwide.
