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Drugs and Cell-Based Therapies for the Prevention and Treatment of Neonatal Brain Injury
Melanie A McNally1, Janet S Soul2
1Department of Neurology, Harvard Medical School and Massachusetts General Hospital, Boston, MA 02114, USA.
Insights
Neonatal brain injury (NBI) presents a significant global challenge. Current effective neuroprotective therapies are limited, highlighting an urgent need for novel treatments beyond existing options like therapeutic hypothermia.
Area of Science:
- Neonatal neurology
- Neuroscience
- Pediatric medicine
Background:
- Neonatal brain injury (NBI) poses a substantial international health burden.
- Existing neuroprotective strategies for NBI are limited, with recent trials showing no benefit from erythropoietin or darbepoetin.
Purpose of the Study:
- To review current evidence-based neuroprotective strategies for NBI.
- To identify promising pharmacologic agents and cell-based therapies for NBI prevention and treatment.
Main Methods:
- Literature review of clinical trials and evidence-based guidelines.
- Analysis of pharmacologic agents and stem cell therapies under investigation for NBI.
Main Results:
- Established neuroprotection for preterm NBI includes antenatal steroids, magnesium, and supportive care.
- Therapeutic hypothermia is the standard for term hypoxic-ischemic encephalopathy.
- Melatonin, topiramate, and stem cell therapies show potential for clinical implementation.
Conclusions:
- There is an urgent unmet need for more effective therapies to prevent and treat neonatal brain injury.
- Ongoing research into melatonin, topiramate, and stem cell therapies offers hope for future clinical applications.
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.
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