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Updated: Jun 23, 2026
Mouse Models of Periventricular Leukomalacia
Published on: May 18, 2010
Emerging neuroprotective interventions in periventricular leukomalacia - A systematic review of preclinical studies
Thangaraj Abiramalatha1,2, Viraraghavan Vadakkencherry Ramaswamy3, Anand Kumar Ponnala4
1Consultant Neonatologist, Kovai Medical Center and Hospital (KMCH), Coimbatore, India.
Insights
Periventricular leukomalacia (PVL) therapies show promise in animal models, with stem cells, erythropoietin, and melatonin advancing to limited clinical trials. Further research is needed for other interventions and combination therapies for PVL neuroprotection.
Area of Science:
- Neonatal Neurology
- Neuroscience
- Developmental Pediatrics
Background:
- Periventricular leukomalacia (PVL) is a brain injury in preterm neonates, often leading to cerebral palsy.
- Currently, no definitive treatment exists for PVL.
- Investigating preclinical therapies is crucial for developing effective clinical strategies.
Purpose of the Study:
- To systematically review interventions investigated in preclinical studies for neuroprotective effects against PVL.
- To assess the clinical relevance of these preclinical findings.
Main Methods:
- A systematic review of 142 studies evaluating interventions in animal models of PVL.
- Analysis of neuroprotective efficacy reported in preclinical research.
Main Results:
- Stem cells, erythropoietin, and melatonin demonstrated significant neuroprotective effects in preclinical PVL research.
- These interventions have undergone limited clinical trials.
- Numerous other therapeutic modalities showed potential but require further investigation.
Conclusions:
- Stem cells, erythropoietin, and melatonin are promising candidates for PVL treatment.
- Combination therapies and individualized treatment approaches should be explored in future research.
- Further preclinical data is necessary for many potential PVL interventions before clinical consideration.
Introduction:
Periventricular leukomalacia (PVL) is a result of various antenatal, intrapartum, or postnatal insults to the developing brain and is an important harbinger of cerebral palsy in preterm neonates. There is no proven therapy for PVL. This calls for appraisal of targeted therapies that have been investigated in animal models to evaluate their relevance in a clinical research context.
Areas Covered:
This systematic review identifies interventions that were evaluated in preclinical studies for neuroprotective efficacy against PVL. We identified 142 studies evaluating various interventions in PVL animal models (search method is detailed in section 2).
Expert Opinion:
Interventions that have yielded significant results in preclinical research, and that have been evaluated in a limited number of clinical trials include stem cells, erythropoietin, and melatonin. Many other therapeutic modalities evaluated in preclinical studies have been identified, but more data on their neuroprotective potential in PVL must be garnered before they can be considered for clinical trials. Because most of the tested interventions had only a partial efficacy, a combination of interventions that could be synergistic should be investigated in future preclinical studies. Furthermore, since the nature and pattern of perinatal insults to preterm brain predisposing it to PVL are substantially variable, individualized approaches for the choice of appropriate neuroprotective interventions tailored to different subgroups of preterm neonates should be explored.
