Age-related macular degeneration (AMD) is the leading cause of vision loss and blindness in people over age 60 in the developed world. Progression of this disease results in the loss of the ability to perform activities highly correlaed with quality of life. This disease progression is not well understood and treatments address only portions of the underlying mechanisms of the disease, while there is no cure. The current standard of care is repetitive intravitreal anti-vascular endothelial growth factor (anti-VEGF) pharmacologic treatment. However, only 34-40% of patients gain clinically significant vision and maintain that gain over the course of one to two years. Ocular Proteomics, LLC (OPL)'s objective is to validate a panel of biomarkers that predict clinical non-responders to anti-VEGF therapy to allow physicians to quickly identify those who would benefit from alternative therapies. The current methodology is the alternative, where non-responders are identified only after months of ineffective treatment. The OPL lab previously discovered the presence of cell receptors and their phosphorylated (activated) forms in the vitreous fluid of human eyes. Significant quantitative differences in several proteins between anti-VEGF treatment responders and non-responders were demonstrated. OPL's approach has been centered on the use of R.P.P.M. (reverse-phase protein microarray) technology to accurately discriminate different disease states in patients. Also, recent discoveries show quantitative differences between subsets of patients within some small disease groups. The next phase is to employ these past discoveries to uncover additional differences between responsive and non-responsive patients. Long term objectives include validating these unique quantitative differences among larger populations, as well as disseminating the molecular mechanisms of disease progression. The results achieved from these objectives will lead to predicting response retinal diseases, including wet AMD using the Diagnostic Vitreous Proteome.
The specific aims of this proposal include: 1) Recruit patients with wet AMD from multiple retina centers across the country to participate in the study 2) Validate Efficacy of potential Biomarkers to Predict Treatment Response and Disease Progression 3) Finalize most significant AMD biomarkers for determining and predicting Treatment Response and Disease Progression

Public Health Relevance

The proposed research will improve public health by improving the vision of patients with wet age-related macular degeneration at reduced costs. It will do this by producing a product that will allow retina physicians to perform a diagnostic test that will determine how a patient will respond to a given treatment before starting the patient on this treatment. This will allow the doctor to choose the right treatment for the right patient at te right time, providing truly personalized medicine.

National Institute of Health (NIH)
National Eye Institute (NEI)
Small Business Innovation Research Grants (SBIR) - Phase II (R44)
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Special Emphasis Panel (ZRG1-ETTN-G (12))
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Wujek, Jerome R
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Ocular Proteomics, LLC
United States
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