Drugs and Cell-Based Therapies for the Prevention and Treatment of Neonatal Brain Injury.

Melanie A McNally, Janet S Soul

Journal: Clinics in perinatology 2026;53(3):727-740

PMID: 42556987

Abstract

The international burden of neonatal brain injury (NBI) remains significant with enormous potential for therapeutic intervention. Recent large trials have demonstrated no neuroprotection with either erythropoietin or darbepoetin for term or pre-term brain injury. Neuroprotective strategies with strong evidence are currently limited to antenatal steroids, antenatal magnesium, and supportive care for pre-term NBI and therapeutic hypothermia for term hypoxic-ischemic encephalopathy. Of the pharmacologic strategies currently being investigated clinically, the agents with the highest likelihood of success and closest to clinical implementation include melatonin, topiramate, and stem cell-based therapies. More therapies to prevent or treat NBI are urgently needed.

Address: Department of Neurology, Harvard Medical School and Massachusetts General Hospital, Boston, MA 02114, USA.; Fetal-Neonatal Neurology Program, Department of Neurology, Harvard Medical School and Boston Children's Hospital, Boston, MA 02115, USA. Electronic address: [email protected].

DOI: 10.1016/j.clp.2026.05.003

MeSH Terms: Humans, Infant, Newborn, Hypoxia-Ischemia, Brain, Neuroprotective Agents, Erythropoietin, Melatonin, Leukomalacia, Periventricular, Stem Cell Transplantation, Fructose, Topiramate, Brain Injuries, Darbepoetin alfa, Hypothermia, Induced