For which ophthalmology indications is topical Nerve Growth Factor (NGF) currently used in clinical practice?

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Current Clinical Applications of Topical NGF in Ophthalmology

Topical nerve growth factor (NGF) is currently primarily used in clinical practice for neurotrophic keratopathy, with the first documented clinical use dating back to 2000, while other applications remain largely investigational or emerging. 1

Established Clinical Indications

Neurotrophic Keratopathy (NK)

  • First clinical use: 2000 - Bonini et al. published the first clinical study using murine NGF eye drops for NK 1
  • Current status: FDA-approved recombinant human NGF (rhNGF/Cenegermin) at 0.002% concentration
  • Treatment protocol: 6 drops daily for 8 weeks 2
  • Efficacy: Complete healing of persistent epithelial defects and corneal ulcers in both moderate (stage 2) and severe (stage 3) NK 2, 3
  • Mechanism: Promotes:
    • Corneal epithelial healing
    • Improved corneal sensitivity
    • Increased sub-basal nerve density
    • Increased nerve branching and fiber diameter 3

Emerging Applications

Neuropathic Corneal Pain (NCP)

  • Status: Investigational use based on guidelines
  • Rationale: NGF reduces allodynia and hyperalgesia through reduction of reactive astrocytosis and glial modulation 4
  • Context: Part of neuro-regenerative approaches to NCP management, alongside autologous serum tears and plasma-rich growth factors 4

Corneal Opacification with Persistent Epithelial Defects

  • Status: Recognized in guidelines as effective for non-healing epithelial defects
  • Indication: Specifically in the setting of neurotrophic keratopathy 4
  • Mechanism: Promotes epithelial healing and prevents progression to corneal opacification

Optic Pathway Gliomas in NF1

  • Status: Investigational use
  • Evidence: Phase II trial showed improved retinal ganglion cell function and visual field enlargement in patients with stable optic pathway glioma disease and severe visual impairment 4
  • Mechanism: Neuroprotective effects on retinal ganglion cells

Safety Profile

  • Generally well-tolerated with minimal side effects
  • Common side effects: Mild and transient conjunctival hyperemia and photophobia 5
  • No development of circulating antibodies to NGF reported in long-term follow-up (16-72 months) 5

Clinical Considerations

  • NGF therapy shows promise for restoring ocular surface integrity and visual function in neurotrophic corneal disease 1
  • Topical application results in significant improvement in corneal sensitivity and thickness in both moderate and severe NK 2
  • In cases of delayed healing of corneal epithelial defects, NGF should be considered before resorting to more invasive procedures 4

Common Pitfalls and Caveats

  • Limited availability and high cost may restrict widespread use
  • Proper patient selection is critical - best results seen in neurotrophic conditions
  • Complete healing may take between 12 days to 6 weeks of treatment 1
  • Patients with trigeminal nerve resection may require retreatment 1
  • Patients should be monitored for hyperemia and ocular/periocular pain during initial treatment 1

While NGF shows promise for various ophthalmic conditions, neurotrophic keratopathy remains the only firmly established clinical indication with regulatory approval and substantial evidence supporting its efficacy and safety.

References

4
Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

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Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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