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2025 Oxford-Harrington Rare Disease Scholar Award
Ophthalmology
Systems Pharmacology Platform-Based Therapeutics for the Treatment or Prevention of Common Age-Related and Inherited Retinal Diseases
2023 Harrington-MSTP Scholar
Degeneration of photoreceptor cells, the retinal pigment epithelium and other cell types in the retina is the underlying cause of vision loss in progressive retinal diseases, such as age-related macular degeneration, retinitis pigmentosa, diabetic retinopathy and glaucoma; a large majority of these cases (up to 90%) have only minimally effective or no treatment options available.
According to Dr. Luu, conventional treatments targeting singular disease pathways have not been successful because these diseases are multifactorial. Therefore, he and his team have pursued a systems pharmacology approach.
“Systems pharmacology holds promise as a safe and highly efficacious therapeutic approach for mitigating complex neurodegenerative disorders through synergistic modulation of multiple pathways underlying retinal health and function,” Dr. Luu explained. “In preclinical studies we have demonstrated the efficacy of our SP3 therapeutic platform, based on systems pharmacology, which improved both retinal structure and function across a variety of genetic and environmental disease models.”
With support from Harrington Discovery Institute, Dr. Luu and his team are now focusing on formulation and manufacturing of the SP3 oral medication as a prelude to human clinical trials for degenerative retinal diseases.
“The data that we have supports broad efficacy,” Dr. Luu said. “Beyond retinal disease we hope to achieve validation of the systems pharmacology platform in treating a variety of neurodegenerative conditions, with clinical trials started for dementia, multiple sclerosis and other indications. Ultimately, I am optimistic our efforts will culminate in an entire class of broad-spectrum therapies representing a new standard of care for acute and chronic disorders associated with aging, stress and/or neurodegeneration.”