The encouraging results of a phase II randomized, controlled, double-blind clinical trial testing the use of NGF in patients with severe optic pathway gliomas, were published in the prestigious journal Brain. The study was conducted at the Policlinico A. Gemelli in Rome by Antonio Chiaretti (Assistant Professor of Pediatrics), Benedetto Falsini (Associate Professor of Ophthalmology) and Riccardo Riccardi (Professor of Pediatric Oncology of the Catholic University of the Sacred Heart) in collaboration with CNR researchers Luigi Manni and Marzia Soligo (Institute of Pharmacology Translational) and Lucia Galli-Resta (Institute of Neuroscience). The optic pathway gliomas are slow-growing cancers that affect electively the visual pathways and the optic chiasm, leading to blindness due to infiltration and compression of the optic nerve by tumor cells. There are no effective therapies to combat this devastating outcome induced by the optic gliomas. Eighteen patients with gliomas of the optic tract and severe visual deficits induced by neoplasia, related were treated with NGF-containing eye drops administered in the conjunctiva 3 times a day, while 8 patients with the same severity of disease received placebo. Only in patients treated with NGF significant improvements in electrophysiological parameters, such PEV and ERG, were found. 75% of patients who received NGF showed a significant improvement of the visual field, which allowed them a better quality of life, by making them more autonomous in the execution of daily activities. By contrast, in the placebo group a progression of the disease with a worsening of visual acuity was observed. The route of conjunctival administration has allowed toby-pass the blood-retinal barrier, facilitating the penetration of NGF directly into the optic tract, where there are specific receptors for this neurotrophin, and where the NGF can exert its action without causing appreciable side effects. The conjunctival administration represent a non-invasive, easy, safe and effective delivery route, tested for the first time in the world in this type of pathology.
Falsini, B., A. Chiaretti, D. Rizzo, M. Piccardi, A. Ruggiero, L. Manni, M. Soligo, A. Dickmann, M. Federici, A. Salerni, L. Timelli, G. Guglielmi, I. Lazzareschi, M. Caldarelli, L. Galli-Resta, C. Colosimo and R. Riccardi (2016). "Nerve growth factor improves visual loss in childhood optic gliomas: a randomized, double-blind, phase II clinical trial." Brain 139(Pt 2): 404-414
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