Assessing the Potential of AI-Driven Drug Repurposing in Ophthalmology: An Analysis of ChatGPT's Therapeutic Recommendations

PurposeThis study aimed to evaluate the novelty and potential value of therapeutic suggestions made by an artificial intelligence large language model for treating various ophthalmic diseases. MethodsChatGPT-3.5 was used to suggest novel ophthalmic indications for available medications. The generation of therapeutic suggestions was performed by inputting standardized queries about treatments for common ophthalmic conditions and then categorizing the responses by drug type. Data tables were organized by ophthalmic condition, with consistent quality checks to ensure accuracy. Literature searches were conducted to determine the FDA-approval status of each therapy, and whether the suggested application was novel in the context of the condition. Therapies were categorized according to current use and level of evidence for use. ResultsChatGPT proposed 180 medications and treatment options for 36 eye conditions. Of the 180 medications, 143 (79.4%) were FDA-approved for general medical use and 32 out of 180 (17.7%) were specifically approved for the recommended ophthalmological conditions. The majority of suggested treatments were for corneal and anterior segment disease (82/180 or 46%), with other categories being retina (23%), glaucoma (8.9%), pediatrics and strabismus (12%), neuro-ophthalmology (0.55%), and uveitis (10%). The proposed treatments were then evaluated by the degree to which the literature supported additional investigation. The majority, 86/180 (48%), were already being used in the clinic, while 27/180 (15%) represented a novel ophthalmic use that appeared to be a reasonable hypothesis to test, and 20/180 (11%) were novel, but appeared unlikely to succeed, based on their mechanism of action. The level of novelty for each treatment was also evaluated, with categories spanning from pre-existing testing in animal models to repurposed for novel ophthalmic use. ConclusionThese findings suggest that ChatGPT is capable of formulating novel treatment options for a range of ophthalmic diseases. Of the suggestions, 27/180 (15%) appeared novel, and reasonable suggestions, based on their mechanism of action. ChatGPT can potentially suggest novel ophthalmic applications for existing medications, which could be evaluated with further laboratory and clinical research.