Nerve Growth Factor for Optic Pathway Gliomas: Four Years Follow-up Findings in Children after a Single Treatment Course.

Abstract

Purpose : A previous double-blind, randomized, placebo-controlled, 6-month phase II clinical trial indicated potential beneficial effects of a short-term treatment (10 days) with mouse nerve growth factor (mNGF) on visual function of children with optic pathway gliomas (OPG) (Falsini et al., Brain 2016). The aim of the present study was to evaluate the long-term follow-up of clinical and neuroradiological parameters in the cohort of patients with OPG who had previously undergone the phase II mNGF study

Methods : Fifteen of the 18 patients originally enrolled in the previous mNGF trial (median age 16, range 6-22, eight NF1 patients), agreed to be monitored clinically and neuroradiologically over a 48-month follow-up. This extended follow-up was initiated 6 to 12 months after the end of the original mNGF trial. Patients underwent every 6 months a general clinical and neuro-ophthalmological examination, visual evoked potentials and electroretinograms photopic negative response. Brain MRI was performed every 12 months

Results : When comparing the final and initial follow-up median values, no significant changes were found in visual acuity, visual evoked potential/electroretinogram amplitudes, and visual field radius. No significant differences were found in any parameter compared to the values
of the baseline mNGF. There were no significant differences in the follow-up changes between patients initially assigned to mNGF and those to placebo in the original phase II study. Brain MRI showed stable disease in all patients

Conclusions : The results of this study indicate good long-term safety and tolerability of a short-term treatment with mouse NGF in children with OPG. Further clinical studies are needed to confirm short- and long-term clinical efficacy of NGF treatment for preventing OPG-induced visual loss

Falsini B et al Nerve growth factor improves visual loss in childhood optic gliomas: a randomized, double-blind, phase II clinical trial. Brain. 2016 Feb;139(Pt 2):404-14. doi: 10.1093/brain/awv366. Epub 2016 Jan 14. PMID: 26767384.

This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.