NGF eye-drops topical administration in patients with retinitis pigmentosa, a pilot study

Benedetto Falsini, Giancarlo Iarossi, Antonio Chiaretti, Antonio Ruggiero, Luigi Manni, Manni Luigi, Lucia Galli-Resta, Giovanni Corbo, Edoardo Abed

Research output: Contribution to journalArticle

Abstract

BACKGROUND: Preclinical trials have shown beneficial effects of nerve growth factor (NGF) administration on visual function in animal models of retinitis pigmentosa (RP). The aim of this pilot study was to explore the potential efficacy of short term NGF eye drops treatment in patients affected by RP.

METHODS: The trial consisted in 10 days daily administration of murine NGF as eye-drops for a total dose of 1 mg NGF/pt. Eight RP patients at an advanced stage of the disease were included in the trial. To monitor safety and potential adverse effects subjects underwent standard clinical measures and were requested to report any general or topic alterations following NGF assumption. Retinal function was assessed at baseline and after treatment by best-corrected visual acuity measurement (BCVA), macular focal electroretinogram (fERG) recording and Goldmann visual field testing.

RESULTS: A transient tolerable local corneal irritation was the only adverse effect reported. fERG and BCVA remained within the limits determined by test-retest analysis of a large cohort of RP patients. Three patients reported a subjective feeling of improved visual performance. This was associated to a temporary enlargement of the visual field in all three patients and to improved fERG in two of the three.

CONCLUSIONS: Short-term administration of NGF eye-drops caused neither significant adverse effects nor visual function losses in the tested RP patients. A minority of patients experienced an improvement of visual performance as shown by Goldmann visual field and fERG. This study supports the safety and possible efficacy of NGF eye-drops administration in RP patients.

TRIAL REGISTRATION: EudraCT n. 2008-004561-26.

Original languageEnglish
Pages (from-to)8
JournalJournal of Translational Medicine
Volume14
DOIs
Publication statusPublished - Jan 9 2016
Externally publishedYes

Fingerprint

Topical Administration
Retinitis Pigmentosa
Ophthalmic Solutions
Nerve Growth Factor
Visual Fields
Visual Acuity
Safety
Animals
Emotions
Animal Models
Testing
Therapeutics

Keywords

  • Administration, Topical
  • Adult
  • Animals
  • Electroretinography
  • Female
  • Humans
  • Male
  • Mice
  • Middle Aged
  • Nerve Growth Factor
  • Ophthalmic Solutions
  • Pilot Projects
  • Retinitis Pigmentosa
  • Time Factors
  • Visual Acuity
  • Visual Fields
  • Clinical Trial
  • Journal Article
  • Research Support, Non-U.S. Gov't

Cite this

Falsini, B., Iarossi, G., Chiaretti, A., Ruggiero, A., Manni, L., Luigi, M., ... Abed, E. (2016). NGF eye-drops topical administration in patients with retinitis pigmentosa, a pilot study. Journal of Translational Medicine, 14, 8. https://doi.org/10.1186/s12967-015-0750-3

NGF eye-drops topical administration in patients with retinitis pigmentosa, a pilot study. / Falsini, Benedetto; Iarossi, Giancarlo; Chiaretti, Antonio; Ruggiero, Antonio; Manni, Luigi; Luigi, Manni; Galli-Resta, Lucia; Corbo, Giovanni; Abed, Edoardo.

In: Journal of Translational Medicine, Vol. 14, 09.01.2016, p. 8.

Research output: Contribution to journalArticle

Falsini, B, Iarossi, G, Chiaretti, A, Ruggiero, A, Manni, L, Luigi, M, Galli-Resta, L, Corbo, G & Abed, E 2016, 'NGF eye-drops topical administration in patients with retinitis pigmentosa, a pilot study', Journal of Translational Medicine, vol. 14, pp. 8. https://doi.org/10.1186/s12967-015-0750-3
Falsini, Benedetto ; Iarossi, Giancarlo ; Chiaretti, Antonio ; Ruggiero, Antonio ; Manni, Luigi ; Luigi, Manni ; Galli-Resta, Lucia ; Corbo, Giovanni ; Abed, Edoardo. / NGF eye-drops topical administration in patients with retinitis pigmentosa, a pilot study. In: Journal of Translational Medicine. 2016 ; Vol. 14. pp. 8.
@article{6bf049be93444193916936f67a37c23d,
title = "NGF eye-drops topical administration in patients with retinitis pigmentosa, a pilot study",
abstract = "BACKGROUND: Preclinical trials have shown beneficial effects of nerve growth factor (NGF) administration on visual function in animal models of retinitis pigmentosa (RP). The aim of this pilot study was to explore the potential efficacy of short term NGF eye drops treatment in patients affected by RP.METHODS: The trial consisted in 10 days daily administration of murine NGF as eye-drops for a total dose of 1 mg NGF/pt. Eight RP patients at an advanced stage of the disease were included in the trial. To monitor safety and potential adverse effects subjects underwent standard clinical measures and were requested to report any general or topic alterations following NGF assumption. Retinal function was assessed at baseline and after treatment by best-corrected visual acuity measurement (BCVA), macular focal electroretinogram (fERG) recording and Goldmann visual field testing.RESULTS: A transient tolerable local corneal irritation was the only adverse effect reported. fERG and BCVA remained within the limits determined by test-retest analysis of a large cohort of RP patients. Three patients reported a subjective feeling of improved visual performance. This was associated to a temporary enlargement of the visual field in all three patients and to improved fERG in two of the three.CONCLUSIONS: Short-term administration of NGF eye-drops caused neither significant adverse effects nor visual function losses in the tested RP patients. A minority of patients experienced an improvement of visual performance as shown by Goldmann visual field and fERG. This study supports the safety and possible efficacy of NGF eye-drops administration in RP patients.TRIAL REGISTRATION: EudraCT n. 2008-004561-26.",
keywords = "Administration, Topical, Adult, Animals, Electroretinography, Female, Humans, Male, Mice, Middle Aged, Nerve Growth Factor, Ophthalmic Solutions, Pilot Projects, Retinitis Pigmentosa, Time Factors, Visual Acuity, Visual Fields, Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't",
author = "Benedetto Falsini and Giancarlo Iarossi and Antonio Chiaretti and Antonio Ruggiero and Luigi Manni and Manni Luigi and Lucia Galli-Resta and Giovanni Corbo and Edoardo Abed",
year = "2016",
month = "1",
day = "9",
doi = "10.1186/s12967-015-0750-3",
language = "English",
volume = "14",
pages = "8",
journal = "Journal of Translational Medicine",
issn = "1479-5876",
publisher = "BioMed Central Ltd.",

}

TY - JOUR

T1 - NGF eye-drops topical administration in patients with retinitis pigmentosa, a pilot study

AU - Falsini, Benedetto

AU - Iarossi, Giancarlo

AU - Chiaretti, Antonio

AU - Ruggiero, Antonio

AU - Manni, Luigi

AU - Luigi, Manni

AU - Galli-Resta, Lucia

AU - Corbo, Giovanni

AU - Abed, Edoardo

PY - 2016/1/9

Y1 - 2016/1/9

N2 - BACKGROUND: Preclinical trials have shown beneficial effects of nerve growth factor (NGF) administration on visual function in animal models of retinitis pigmentosa (RP). The aim of this pilot study was to explore the potential efficacy of short term NGF eye drops treatment in patients affected by RP.METHODS: The trial consisted in 10 days daily administration of murine NGF as eye-drops for a total dose of 1 mg NGF/pt. Eight RP patients at an advanced stage of the disease were included in the trial. To monitor safety and potential adverse effects subjects underwent standard clinical measures and were requested to report any general or topic alterations following NGF assumption. Retinal function was assessed at baseline and after treatment by best-corrected visual acuity measurement (BCVA), macular focal electroretinogram (fERG) recording and Goldmann visual field testing.RESULTS: A transient tolerable local corneal irritation was the only adverse effect reported. fERG and BCVA remained within the limits determined by test-retest analysis of a large cohort of RP patients. Three patients reported a subjective feeling of improved visual performance. This was associated to a temporary enlargement of the visual field in all three patients and to improved fERG in two of the three.CONCLUSIONS: Short-term administration of NGF eye-drops caused neither significant adverse effects nor visual function losses in the tested RP patients. A minority of patients experienced an improvement of visual performance as shown by Goldmann visual field and fERG. This study supports the safety and possible efficacy of NGF eye-drops administration in RP patients.TRIAL REGISTRATION: EudraCT n. 2008-004561-26.

AB - BACKGROUND: Preclinical trials have shown beneficial effects of nerve growth factor (NGF) administration on visual function in animal models of retinitis pigmentosa (RP). The aim of this pilot study was to explore the potential efficacy of short term NGF eye drops treatment in patients affected by RP.METHODS: The trial consisted in 10 days daily administration of murine NGF as eye-drops for a total dose of 1 mg NGF/pt. Eight RP patients at an advanced stage of the disease were included in the trial. To monitor safety and potential adverse effects subjects underwent standard clinical measures and were requested to report any general or topic alterations following NGF assumption. Retinal function was assessed at baseline and after treatment by best-corrected visual acuity measurement (BCVA), macular focal electroretinogram (fERG) recording and Goldmann visual field testing.RESULTS: A transient tolerable local corneal irritation was the only adverse effect reported. fERG and BCVA remained within the limits determined by test-retest analysis of a large cohort of RP patients. Three patients reported a subjective feeling of improved visual performance. This was associated to a temporary enlargement of the visual field in all three patients and to improved fERG in two of the three.CONCLUSIONS: Short-term administration of NGF eye-drops caused neither significant adverse effects nor visual function losses in the tested RP patients. A minority of patients experienced an improvement of visual performance as shown by Goldmann visual field and fERG. This study supports the safety and possible efficacy of NGF eye-drops administration in RP patients.TRIAL REGISTRATION: EudraCT n. 2008-004561-26.

KW - Administration, Topical

KW - Adult

KW - Animals

KW - Electroretinography

KW - Female

KW - Humans

KW - Male

KW - Mice

KW - Middle Aged

KW - Nerve Growth Factor

KW - Ophthalmic Solutions

KW - Pilot Projects

KW - Retinitis Pigmentosa

KW - Time Factors

KW - Visual Acuity

KW - Visual Fields

KW - Clinical Trial

KW - Journal Article

KW - Research Support, Non-U.S. Gov't

U2 - 10.1186/s12967-015-0750-3

DO - 10.1186/s12967-015-0750-3

M3 - Article

C2 - 26748988

VL - 14

SP - 8

JO - Journal of Translational Medicine

JF - Journal of Translational Medicine

SN - 1479-5876

ER -