Stem Cell Therapy for Early Age-Related Macular Degeneration

For this project, researchers aim to develop and test a novel material that will support targeted injections of rescue cells for local therapy of age-related macular degeneration.

Although a key feature of age-related macular degeneration is damage to the central part of the retina, recent findings show that tiny blood vessels that feed this area degenerate first. Narendra Pandala, PhD, and his colleagues want to target this first casualty of the disease by applying adult-derived stem cells to the area. To encourage the stem cells to repopulate the lost capillaries, the team plans to develop a biomaterial that supports targeted injection of the cells. One feature of their planned biomaterial, a kind of gel that can swell after injection, is an immunosuppressant that can act locally to protect the introduced cells from attack.

The group’s goal is to avoid the need for systemic immunosuppressants in recipients of donated cells, which would normally be required for such transplants. To develop and characterize their biomaterial, Dr. Pandala and his group will be using lab models of cell loss in the eye. They expect their work to yield an injectable stem cell delivery system with possible clinical applications that can be used for further research into the earliest changes in age-related macular degeneration. If successful, their strategy also would represent a potential treatment for age-related macular degeneration that could be effective quite early in the disease and prevent further damage.

This proposal involves a novel stem cell-based therapy to be used in the early stages of AMD to prevent damage and further loss to the neural retina. We have developed protocols for the development of stem cell-derived choroidal endothelial cells and their delivery using an injectable hydrogel biomaterial.

With this project completed, we will have (1) a hydrogel-based stem cell delivery system for transplantation applications as well as a bioink for 3D in vitro models; (2) an animal model to characterize the early non-neovascular AMD and a potential early intervention strategy; and (3) a localized immunosuppression regime to support stem cell integration.