Preprogrammed Bone Marrow Cells as a Systemic Therapy for Dry AMD

Summary

Age-related macular degeneration is the leading cause of irreversible blindness in the elderly. Dry AMD, which represents 85% of AMD, is associated with retinal pigment epithelial (RPE) dysfunction and death for which there is currently no treatment. The development of a minimally invasive cellular therapy that can be given systemically will a) overcome the need for invasive ocular surgery and b) offer the potential for prevention rather than intervention since this therapy can be given much earlier in the disease. Successful development of this approach will offer an important treatment for the 1.7 million plus Americans who are threatened with visual loss from dry AMD, improve quality of life and reduce social and healthcare costs. We predict that the BrightFocus funding for this proposed preclinical study will provide the necessary data to confirm the efficacy of this cellular therapy and allow this pioneering research to translate into phase 1 and 2 clinical trials.

Project Details

Dry age‐related macular degeneration (AMD) represents 80 to 90 percent of the population currently diagnosed with AMD, yet there is no effective disease‐modifying treatment. Retinal pigmented epithelial (RPE) cells— important for waste recycling and delivery of nutrients to the light‐detecting cells—are mysteriously killed off in dry AMD. Therefore, there is much promise in replacing the damaged RPE cells with healthy ones.

In previous studies, Drs. Michael Boulton, Maria Grant and colleagues made the exciting discovery that changing the expression of one gene in isolated bone marrow‐derived plasma cells (BMPCs)—a type of adult stem cell— transforms them into RPE‐like cells. When these cells are injected back into the blood of mice with physically damaged eyes, they go to the retina, renew the single‐layer of RPE cells, and re‐establish normal vision. Drs. Boulton and Grant plan to test this RPE‐replacement treatment in mice with AMD. If this treatment is proven to be effective in these mice, it could lead to human clinical trials and treatment possibilities without the need for invasive eye surgery or injections.