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Updated: Nov 3, 2025
A Piglet Model of Neonatal Hypoxic-Ischemic Encephalopathy
Published on: May 16, 2015
Cell-based treatment for perinatal hypoxic-ischemic encephalopathy
You Jeong Park1, Cesario V Borlongan1, Mari Dezawa2
1Department of Neurosurgery and Brain Repair, Morsani College of Medicine, University of South Florida, Tampa, FL, USA.
Insights
Cell-based therapies show promise for treating neonatal hypoxic-ischemic encephalopathy (HIE). Stem cell treatments may reduce inflammation and aid brain repair, with ongoing trials exploring efficacy and safety.
Area of Science:
- Neuroscience
- Regenerative Medicine
- Neonatal Research
Background:
- Hypoxic-ischemic encephalopathy (HIE) is a leading cause of neonatal brain injury with limited treatment options.
- Current treatment, hypothermia, is only effective within 6 hours for full-term infants.
- Cell-based therapies offer a promising avenue for HIE treatment, targeting neonatal brain injury.
Purpose of the Study:
- To review cell sources used in clinical trials for HIE.
- To evaluate the therapeutic mechanisms of cell-based HIE treatments.
- To assess the potential of stem cells in managing neonatal neurological complications.
Main Methods:
- Systematic review of preclinical and clinical studies on HIE cell-based therapies.
- Searches conducted in PubMed, Web of Science, and ClinicalTrials.gov databases.
- Analysis of therapeutic effects and underlying mechanisms of various stem cell types.
Main Results:
- Umbilical cord blood cells, placenta-derived stem cells, and mesenchymal stem cells (MSCs) show promising results in HIE trials.
- Therapeutic effects are largely attributed to the bystander effect, reducing inflammation and promoting neurogenesis.
- Multilineage-differentiating stress-enduring (Muse) cells, a subset of MSCs, demonstrate potential in replacing damaged cells and have advanced to clinical trials.
Conclusions:
- Cell-based therapies, particularly MSCs and Muse cells, hold significant potential for treating HIE.
- Further research and multi-center trials are crucial to optimize protocols and confirm the safety and efficacy of these treatments.
- Stem cell transplantation offers a novel strategy to mitigate long-term neurological deficits following neonatal hypoxic-ischemic injury.
Abstract:
Hypoxic-ischemic encephalopathy (HIE) is a major cause of acute neonatal brain injury and can lead to disabling long-term neurological complications. Treatment for HIE is limited to supportive care and hypothermia within 6 h injury which is reserved for full-term infants. Preclinical studies suggest the potential for cell-based therapies as effective treatments for HIE. Some clinical trials using umbilical cord blood cells, placenta-derived stem cells, mesenchymal stem cells (MSCs), and others have yielded promising results though more studies are needed to optimize protocols and multi-center trials are needed to prove safety and efficacy. To date, the therapeutic effects of most cell-based therapies are hypothesized to stem from the bystander effect of donor cells. Transplantation of stem cells attenuate the aberrant inflammation cascade following HIE and provide a more ideal environment for endogenous neurogenesis and repair. Recently, a subset of MSCs, the multilineage-differentiating stress-enduring (Muse) cells have shown to treat HIE and other models of neurologic diseases by replacing dead or ischemic cells and have reached clinical trials. In this review, we examine the different cell sources used in clinical trials and evaluate the underlying mechanism behind their therapeutic effects. Three databases-PubMed, Web of Science, and ClinicalTrials.gov-were used to review preclinical and clinical experimental treatments for HIE.
