According to research reviewed by Gram Research, nerve growth factor (rhNGF) showed the most promise for treating neurotrophic keratopathy among seven studies involving 494 participants, though it produced only modest corneal healing improvements compared to regular eye drops. No single ‘best’ treatment has been established, and doctors currently select therapies based on clinical experience and available resources.

Neurotrophic keratopathy is a rare eye condition where damaged nerves in the cornea (the clear front part of your eye) prevent it from healing properly. Without treatment, it can cause serious vision problems. Researchers reviewed seven studies testing different medical and surgical treatments for this condition. They found that some treatments like nerve growth factor may help the cornea heal slightly better, but the evidence isn’t strong enough yet to say which treatment works best. Doctors currently choose treatments based on their experience and what resources they have available.

Key Statistics

A systematic review of seven randomized controlled trials involving 494 people with neurotrophic keratopathy found that recombinant human nerve growth factor (rhNGF) may slightly improve corneal healing compared to standard eye drops, though the improvement was modest and evidence certainty was low.

Among 494 participants across seven studies of neurotrophic keratopathy treatments, the majority were women (60-77%), with ages ranging from 25 to 68 years, and study durations varying from 28 days to 18 months.

In the analysis of neurotrophic keratopathy treatments, nerve growth factor did not demonstrate clear benefits for vision improvement compared to regular eye drops, despite showing modest promise for corneal healing in two combined studies.

The Quick Take

  • What they studied: Do medical treatments (like special eye drops) or surgical procedures help people with neurotrophic keratopathy heal their corneas and see better?
  • Who participated: Seven research studies involving 494 people total, ranging from age 25 to 68 years old, mostly women (60-77%). Studies lasted from one month to 18 months.
  • Key finding: A treatment called recombinant human nerve growth factor (rhNGF) may slightly help the cornea heal better compared to regular eye drops, but the improvement is small and the evidence isn’t very strong. Other treatments showed mixed or unclear results.
  • What it means for you: If you have this rare condition, there’s no single ‘best’ treatment yet. Your eye doctor will choose the best option for you based on their experience and what’s available. More research is needed to find treatments that clearly work better.

The Research Details

Researchers searched medical databases for all high-quality studies (called randomized controlled trials) that tested treatments for neurotrophic keratopathy. They found seven studies that compared different treatments—some used special eye drops with growth factors, others tested surgical procedures like transplanting amniotic membrane (tissue from the placenta). The studies ranged from 28 days to 18 months long.

The researchers combined the results from similar studies to see if patterns emerged. They looked at whether treatments helped the cornea heal, improved vision, restored feeling in the cornea, and whether they caused side effects. They were careful to note when studies had small numbers of participants or other limitations that made the results less reliable.

Most studies were funded by pharmaceutical companies, which is important to know when evaluating the results. The research was conducted in the USA, Europe, and Asia, making the findings more broadly applicable.

Neurotrophic keratopathy is extremely rare, which makes it hard to study. By reviewing all available research together, scientists can get a better picture of what treatments might work, even though individual studies are small. This helps doctors make better decisions for their patients when there’s no clear ‘gold standard’ treatment.

The researchers used strict methods to evaluate study quality and found that most evidence was ’low’ or ‘very low’ certainty. This means the results should be interpreted cautiously. The main problems were: small numbers of participants in studies, differences in how studies measured results, and some studies had design issues that could bias results. However, the studies were generally well-designed randomized controlled trials, which is the gold standard for medical research.

What the Results Show

Nerve growth factor (rhNGF) showed the most promise. Two studies combined showed it may slightly improve how well the cornea heals compared to regular eye drops, though the improvement was modest. However, when researchers looked at complete healing of the cornea, the results were unclear because studies measured this differently.

Another treatment called CACICOL20 did not appear to help corneal healing better than regular eye drops. A third treatment (RGN-259) also didn’t show clear benefits, though this was based on only one very small study with just 18 people.

When looking at vision improvement, nerve growth factor did not appear to help people see better compared to regular eye drops. One treatment (rb-bFGF) may have helped restore feeling in the cornea, but the evidence was very uncertain and based on only 60 people.

Two studies suggested that nerve growth factor may prevent the disease from coming back, which is encouraging but needs more research to confirm.

Side effects were generally similar between treatments and regular eye drops. Nerve growth factor and CACICOL20 both appeared to have similar safety profiles to standard care. Only one study tested a surgical approach—amniotic membrane transplantation—and it showed no clear advantage over other surgical options like tarsorrhaphy or bandage contact lenses for corneal healing or vision improvement.

This is the first comprehensive review of all available research on neurotrophic keratopathy treatments. Previous knowledge was based on individual studies and clinical experience. This review confirms that while some treatments show promise, the evidence overall is weaker than doctors would like. The findings suggest that current treatment choices are based more on clinical judgment than strong scientific proof.

The biggest limitation is that there are very few studies—only seven total—and most had small numbers of participants (18 to 156 people). Small studies are less reliable. Studies measured outcomes differently, making it hard to compare results. Most studies were funded by companies making the treatments, which could introduce bias. The condition is so rare that recruiting enough participants for large studies is extremely difficult. Finally, follow-up times varied from one month to 18 months, so we don’t know about long-term effects.

The Bottom Line

If you have neurotrophic keratopathy, work closely with your eye doctor to choose a treatment plan tailored to your specific situation. Nerve growth factor (rhNGF) may be worth considering as it showed the most promise in research, though improvements are modest (low confidence). Standard care with artificial tears and protective contact lenses remains important. Discuss both medical treatments and surgical options with your doctor. More research is urgently needed to find better treatments.

This research matters most for people with neurotrophic keratopathy and their eye doctors. It’s also important for researchers developing new treatments. People with diabetes, those who’ve had eye surgery, or anyone with corneal nerve damage should be aware of this condition. This research is less relevant for people without this rare condition.

If treatments do help, improvements in corneal healing typically appear within one to three months. Vision improvements may take longer or may not occur even if the cornea heals. Some people may see benefits within weeks, while others may need several months to know if a treatment is working. Your doctor should monitor your progress regularly.

Frequently Asked Questions

What is the best treatment for neurotrophic keratopathy?

No single best treatment has been established. Gram Research analysis of seven studies found nerve growth factor (rhNGF) showed the most promise with modest corneal healing improvements, but evidence remains weak. Doctors currently select treatments based on clinical experience and available resources.

Does nerve growth factor help people with neurotrophic keratopathy see better?

Nerve growth factor may slightly improve corneal healing, but research shows it did not appear to help patients achieve better vision compared to regular eye drops, according to the analysis of available studies.

How many people have been studied for neurotrophic keratopathy treatments?

A total of 494 people participated across seven research studies examining treatments for neurotrophic keratopathy, with individual studies ranging from 18 to 156 participants and lasting between one month and 18 months.

Are surgical treatments effective for neurotrophic keratopathy?

Only one study tested surgical approaches like amniotic membrane transplantation, finding no clear advantage over other surgical options like tarsorrhaphy or bandage contact lenses for corneal healing or vision improvement.

Want to Apply This Research?

  • If using a treatment, track daily: (1) corneal comfort level (1-10 scale), (2) vision clarity (1-10 scale), (3) eye redness (none/mild/moderate/severe), and (4) medication adherence (did you use drops as prescribed?). Take photos of your eye weekly in consistent lighting to visually monitor healing.
  • Set phone reminders for medication timing to ensure consistent use. Keep a simple log noting any changes in symptoms, vision, or discomfort. Schedule regular eye exams as recommended by your doctor—don’t skip appointments. Protect your eye from irritants and follow your doctor’s instructions about contact lens use or eye protection.
  • Use the app to track symptoms weekly and share reports with your eye doctor at each visit. Compare photos over time to see if healing is progressing. If you notice worsening symptoms or no improvement after 6-8 weeks, discuss with your doctor about trying a different treatment. Long-term tracking (6+ months) helps determine if treatments are truly working.

This research summary is for educational purposes only and should not replace professional medical advice. Neurotrophic keratopathy is a serious eye condition requiring specialized care from an ophthalmologist. Treatment decisions should be made in consultation with your eye doctor based on your individual condition, medical history, and specific circumstances. The evidence reviewed here is of low to very low certainty, meaning treatments may not work as expected for every person. Do not start, stop, or change any eye treatments without consulting your healthcare provider. If you experience sudden vision changes, severe eye pain, or signs of infection, seek immediate medical attention.

This research translation is published by Gram Research, the science division of Gram, an AI-powered nutrition tracking app.

Source: Medical and surgical interventions for neurotrophic keratopathy.The Cochrane database of systematic reviews (2025). PubMed 41347649 | DOI