Treatment of Retinal Degeneration in a Mouse Model for Dry AMD

Alfred Lewin, PhD University of Florida


Currently, there is no effective treatment for the early form of AMD, called dry AMD. Therefore, Dr. Lewin and colleagues aim to develop a new treatment for dry AMD by testing whether specific drugs can protect the retinal pigment epithelium (RPE) from succumbing to oxidative stress.

Project Details

Currently there is no treatment for dry AMD. To find a treatment, Dr. Lewin and colleagues are testing systemic delivery of three different drugs in mice engineered to have their RPE cells missing a gene (SOD2) that's involved in oxidative stress. Oxidative stress has been shown to play an important role in causing AMD. Results of these studies may lead to new therapies.

Lewin's use of systemically deliverable therapeutic reagents is unique, and one of the compounds the team is testing has already been through Phase III trials for another disease involving nerve cells, namely Amyotrophic Lateral Sclerosis (ALS). The drug Lewin is testing is orally available and approved for human use.

If the research aims are successful, the team will take several steps to bring this information, and a potential dry AMD treatment, to patients. The first step would be to re-test the successful reagents in different AMD mice that are not directly related to RPE oxidative stress. Since preclinical information and human safety profiles are characterized for these compounds, this may accelerate bringing the drugs to clinical trial. In addition, if either class of compound shows promise, Lewin's team would initiate a collaboration with a medicinal chemist to modify the compounds for local delivery to the eye. Fortunately, there is a strong program in Medicinal Chemistry in the College of Pharmacy at the University of Florida, and these scientists already have a collaboration with Dr. Hendrik Luesch in that department.