SUNY Albany doctoral student Mr. Janmeet Saini, together with researchers at the Neural Stem Cell Institute at the University’s Health Sciences Campus, has published breakthrough findings about age-related macular degeneration (AMD), the leading cause of blindness in the elderly.
A progressive degenerative disease of the retina, AMD gradually causes loss of sight in the center of the visual field, severely compromising the ability of an affected person to perform everyday tasks, including reading. AMD affects more than 10 million people in the United States, and is projected to affect 196 million worldwide by 2020. Ms. Saini, a Ph.D. student in the Biomedical Sciences Department of SUNY Albany’s School of Public Health, and investigators at the NSCI published their new findings in the journal Cell Stem Cell.
The most common form of AMD, dry AMD, is caused by the degeneration of a layer of cells beneath the retina, called the Retinal Pigment Epithelium (RPE). The damage results from accumulation of deposits of waste material under the macula, the part of the retina that offers clear central vision. Dry AMD accounts for more than 80 percent of the cases, and to date there are no approved therapies. Dry AMD can progress to wet AMD, which is accompanied by invasion of the macula by blood vessels, which can break and leak, damaging RPE and light receptor cells.
The development of induced pluripotent stem cells (iPSCs) — cells generated from adult cells (induced) and their ability (pluripotent) to program into any type of bodily cells or tissues — has enabled researchers to create cell culture models of various diseases, in order to test the effectiveness of drug therapies.
The NSCI research team derived induced pluripotent stem cell, or iPSCs, from AMD patients and healthy individuals to create a pure population of RPE cells. The iPSC derived RPE from AMD patients showed higher levels of complement and inflammatory factors than those from healthy individuals. Complement is a vital system of immune surveillance in humans that controls the inflammation and immunity, protecting the healthy host tissues by separating them from diseased cells, foreign invaders and debris. A delicate balance is maintained between the activation and regulation of complement. However, with aging and in several disorders, including Alzheimer’s Disease and AMD, the complement system can become dysregulated, resulting in host tissue damage.
The NSCI research showed that RPE treated with Nicotinamide, a vitamin B3 derivative, showed reduced signs of abnormal AMD proteins in the cultured RPE cells, significant suppression of complement and inflammatory pathways, as well as improved RPE cell survival. Nicotinamide treatment also showed promise for slowing the progression from dry to wet AMD. Further studies of the way Nicotinamide protects RPE cells should aid in the development of novel AMD therapies, with the goal of preserving vision in the elderly.
NSCI, the first independent, non-profit stem cell research institute in the United States, is a unique research organization that produces leading stem cell science with the goal of developing novel therapies for diseases of the retina, brain and spinal cord. NSCI has a team of over 30 researchers focused on bringing impactful therapies to patients.
For more information, visit NSCI.