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Nerve Growth Factor in Pediatric Brain Injury: From Bench to Bedside
Lorenzo Di Sarno1, Serena Ferretti1, Lavinia Capossela1
1Department of Pediatrics, Fondazione Policlinico Universitario "A. Gemelli", IRCCS, 00168 Rome, Italy.
Pharmaceuticals (Basel, Switzerland)
|June 27, 2025
Summary
Nerve growth factor (NGF) shows promise for treating pediatric traumatic brain injury (TBI) and hypoxic-ischemic encephalopathy (HIE). Early studies suggest NGF enhances neural repair and improves outcomes with no significant adverse effects.
Area of Science:
- Pediatric Neurology
- Neuroscience
- Regenerative Medicine
Background:
- Traumatic brain injury (TBI) and hypoxic-ischemic encephalopathy (HIE) cause significant long-term neurological disability in children.
- Current treatment options for neuronal recovery are limited.
- Nerve growth factor (NGF) has emerged as a potential therapeutic agent due to its neuroprotective and neuroregenerative properties.
Purpose of the Study:
- To review the current evidence on nerve growth factor (NGF) for pediatric brain injury.
- To evaluate NGF's mechanisms of action and translational clinical applications.
- To assess NGF's potential in treating TBI and HIE in children.
Main Methods:
- A comprehensive literature review was performed using PubMed, Scopus, and Cochrane CENTRAL.
- Studies published from January 1978 to March 2025 investigating NGF in brain injury were included.
- Inclusion criteria focused on studies assessing neurological outcomes via clinical, biochemical, neuroimaging, or electrophysiological methods.
Main Results:
- Seventeen studies (preclinical and clinical) met the criteria.
- Preclinical models showed NGF reduced neuroinflammation and enhanced neurogenesis.
- Pediatric case reports indicated intranasal NGF improved motor/cognitive function with no adverse effects.
Conclusions:
- NGF shows significant promise as a neuroprotective and neuroregenerative treatment for pediatric brain injury.
- Experimental and early clinical data support NGF's safety and efficacy.
- Large-scale controlled trials are needed to confirm findings and optimize NGF treatment protocols.

