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Nerve growth factor improves visual loss in childhood optic gliomas: a randomized, double-blind, phase II clinical
Benedetto Falsini1, Antonio Chiaretti2, Daniela Rizzo2
11 Institute of Ophthalmology, Università Cattolica del Sacro Cuore, 00168 Rome, Italy achiaretti@yahoo.it.
Abstract:
Paediatric optic pathway gliomas are low-grade brain tumours characterized by slow progression and invalidating visual loss. Presently there is no strategy to prevent visual loss in this kind of tumour. This study evaluated the effects of nerve growth factor administration in protecting visual function in patients with optic pathway glioma-related visual impairment. A prospective randomized double-blind phase II clinical trial was conducted in 18 optic pathway glioma patients, aged from 2 to 23 years, with stable disease and severe visual loss. Ten patients were randomly assigned to receive a single 10-day course of 0.5 mg murine nerve growth factor as eye drops, while eight patients received placebo. All patients were evaluated before and after treatment, testing visual acuity, visual field, visual-evoked potentials, optic coherence tomography, electroretinographic photopic negative response, and magnetic resonance imaging. Post-treatment evaluations were repeated at 15, 30, 90, and 180 days Brain magnetic resonance imaging was performed at baseline and at 180 days. Treatment with nerve growth factor led to statistically significant improvements in objective electrophysiological parameters (electroretinographic photopic negative response amplitude at 180 days and visual-evoked potentials at 30 days), which were not observed in placebo-treated patients. Furthermore, in patients in whom visual fields could still be measured, visual field worsening was only observed in placebo-treated cases, while three of four nerve growth factor-treated subjects showed significant visual field enlargement. This corresponded to improved visually guided behaviour, as reported by the patients and/or the caregivers. There was no evidence of side effects related to nerve growth factor treatment. Nerve growth factor eye drop administration appears a safe, easy and effective strategy for the treatment of visual loss associated with optic pathway gliomas.
Insights
Nerve growth factor eye drops improved vision in children with optic pathway gliomas, a type of brain tumor. This safe treatment offers a new strategy to prevent further visual loss in affected patients.
Area of Science:
- Neuro-oncology
- Ophthalmology
- Regenerative Medicine
Background:
- Paediatric optic pathway gliomas (OPGs) are slow-growing brain tumors causing significant visual impairment.
- Current treatment strategies lack efficacy in preventing or reversing vision loss associated with OPGs.
Purpose of the Study:
- To evaluate the efficacy and safety of nerve growth factor (NGF) eye drops in preserving visual function in children with OPG-related visual impairment.
- To assess the impact of NGF on objective electrophysiological and structural parameters of the visual pathway.
Main Methods:
- A prospective, randomized, double-blind, placebo-controlled phase II clinical trial involving 18 pediatric patients (2-23 years) with OPGs and severe visual loss.
- Patients received either NGF eye drops or placebo for 10 days, with comprehensive visual assessments (visual acuity, visual field, VEP, OCT, PERG, MRI) at multiple time points up to 180 days post-treatment.
Main Results:
- NGF treatment resulted in statistically significant improvements in electroretinographic photopic negative response (PERG PhNR) amplitude and visual-evoked potentials (VEPs).
- Placebo-treated patients experienced visual field worsening, whereas NGF-treated patients showed visual field enlargement and improved visually guided behavior.
- No adverse side effects were reported with NGF eye drop administration.
Conclusions:
- Nerve growth factor eye drops represent a safe, simple, and effective therapeutic approach for managing visual loss in paediatric optic pathway gliomas.
- This novel treatment strategy shows promise in protecting and potentially improving visual function in a vulnerable patient population.
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