# Novel pro-drug pharmacotherapy to prevent neuronal and cell degeneration in AMD

> **NIH NIH R01** · UNIVERSITY OF MISSOURI KANSAS CITY · 2020 · $57,350

## Abstract

PROJECT SUMMARY/ABSTRACT
Degeneration or acute damage of retinal pigment epithelial (RPE) and nerve cells in the retina
due to Age-related Macular Degeneration (AMD) is a major cause of visual loss and blindness in
the United States and worldwide. The proposed multidisciplinary research project will focus on
the characterization of a novel signaling pathway for and the development of a novel
pharmacological intervention to control degeneration of RPE cells and neurons in AMD. To this
end, preclinical testing of the new therapeutic strategy will be performed, along with ocular
biotransformation, transport and distribution studies, in established models of human AMD.
These experiments will determine efficacy of treatment in terminating and/or preventing AMD
associated neuronal loss and preservation of visual function, and to generate data to support
feasibility for and move positive findings to phase 1 and 2 clinical trials. Specifically, we will test
the two-pronged hypothesis that treatment based on the ocular targeting of a novel chemical
antioxidant strategy protects RPE cells and neurons from apoptosis by topical delivery in
established models of human AMD and, therefore, leads to prevention or improvement of visual
impairment and functional deficits associated with AMD. The determination of neuronal viability
and the acquired knowledge on associated biopharmaceutical and pharmacological parameters
will indicate the potential of the method to remedy AMD as the overall goal of the project. This
novel approach for therapy development in AMD focuses on complementary and alternative
cellular protection and neuroprotection. The innovative strategy has the potential to generate a
first-in-class pharmacotherapy approach for dry AMD using a topical rather than a systemic or
invasive route of drug delivery. The strategy’s potentially high impact lies in its capacity to be
both preventative and therapeutic in nature and to complement existing treatment designs and
rationales addressing other aspects of AMD treatment such as those targeting
neovascularization.

## Key facts

- **NIH application ID:** 10216112
- **Project number:** 3R01EY030747-02S1
- **Recipient organization:** UNIVERSITY OF MISSOURI KANSAS CITY
- **Principal Investigator:** Peter Koulen
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $57,350
- **Award type:** 3
- **Project period:** 2019-09-30 → 2022-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10216112

## Citation

> US National Institutes of Health, RePORTER application 10216112, Novel pro-drug pharmacotherapy to prevent neuronal and cell degeneration in AMD (3R01EY030747-02S1). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10216112. Licensed CC0.

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