Intranasal Human-Recombinant Nerve Growth Factor Enhances Motor and Cognitive Function Recovery in a Child with Severe Traumatic Brain Injury

first_pagesettingsOrder Article Reprints Open AccessCase Report Intranasal Human-Recombinant Nerve Growth Factor Enhances Motor and Cognitive Function Recovery in a Child with Severe Traumatic Brain Injury by Lorenzo Di Sarno 1ORCID,Lavinia Capossela 1,Serena Ferretti 1,Luigi Manni 2ORCID,Marzia Soligo 2ORCID,Susanna Staccioli 3,Eleonora Napoli 4ORCID,Riccardo Burattini 3ORCID,Antonio Gatto 1ORCID andAntonio Chiaretti 5,* 1 Institute of Pediatrics, Fondazione Policlinico A. Gemelli IRCCS, 00168 Rome, Italy 2 Istituto di Farmacologia Traslazionale, Consiglio Nazionale delle Ricerche (CNR), 00133 Rome, Italy 3 Dipartimento di Neuroriabilitazione Intensiva, Ospedale Pediatrico “Bambino Gesù”, 00050 Rome, Italy 4 Unit of Neurorehabilitation, Department of Neurosciences, Bambino Gesù Children’s Hospital, IRCCS, 00050 Rome, Italy 5 Institute of Pediatrics, Fondazione Policlinico A. Gemelli IRCCS, Università Cattolica Sacro Cuore, 00168 Rome, Italy * Author to whom correspondence should be addressed. Pharmaceuticals 2025, 18(2), 163; https://doi.org/10.3390/ph18020163 Submission received: 31 December 2024 / Revised: 17 January 2025 / Accepted: 22 January 2025 / Published: 25 January 2025 (This article belongs to the Special Issue Drug Delivery across the Blood–Brain Barrier) Downloadkeyboard_arrow_down Versions Notes Abstract Introduction: Traumatic brain injury (TBI) in pediatric population is responsible for significant mortality and morbidity, particularly among children aged 0–4 and young adults aged 15–24. The developing brain’s unique characteristics may increase vulnerability to injuries, potentially leading to long-term cognitive and motor deficits. Current therapeutic options for neuronal regeneration post-TBI are limited, although neurotrophins, especially nerve growth factor (NGF), show promise in enhancing recovery. NGF can mitigate excitotoxicity and promote neuroprotection, particularly by intranasal administration, which is attractive because of its non-invasive nature. Case Presentation: A three-year-old boy suffered from severe TBI due to a car accident, leading to multiple complications, including a basilar skull fracture and cerebral venous sinus thrombosis. Initial assessments revealed significant neurological impairments. After intensive care and rehabilitation, the child exhibited gradual improvements in consciousness and motor functions but continued to face challenges, particularly with left-sided hemiparesis. Nine months post-injury, he began intranasal administration of human-recombinant NGF (hr-NGF) as part of a clinical trial. Discussion: Following hr-NGF treatment, the child demonstrated notable advancements in motor function, achieving independent standing and walking. Cognitive assessments indicated improvements in various domains, including verbal comprehension and executive functioning. EEG results showed reduced epileptiform activity. These findings suggest that hr-NGF may facilitate recovery in pediatric TBI cases by enhancing both motor and cognitive outcomes. Conclusions: This case highlights the potential role of intranasal hr-NGF administration as a therapeutic strategy for improving neurological recovery in children with severe TBI. The positive clinical outcomes support further exploration of NGF as a viable treatment option to mitigate long-term sequelae associated with pediatric brain injuries.