Using human stem cell models, researchers at Indiana University School of Medicine found they could analyze deficits within cells damaged by glaucoma, with the potential to use this information to develop new strategies to slow the disease process. The study, published in Stem Cell Reports, focused on targeting genetic mutations within retinal ganglion cells, which serve as the connection between the eye and the brain. Researchers found that when differentiating pluripotent human stem cells into retinal ganglion cells, they were able to identify characteristics associated with neurodegeneration in glaucoma.

“Once you’ve identified a target like this — what’s going wrong in the cells — this opens up a number of possibilities for the eventual development of therapeutic approaches, especially pharmacology approaches to slow down and reverse these degenerative phenotypes,” said Jason Meyer, PhD, associate professor of medical and molecular genetics at Indiana University School of Medicine. Meyer said the team discovered dysfunction in the process of autophagy, the body’s way of removing damaged cells to regenerate healthy cells.

“We found that in the glaucoma patient cells, there are some deficits in this autophagy process, so you had too much cellular junk that was being built up,” Meyer said, adding that those deficits correlated with the degeneration of the cells, which would shrivel up and eventually die off. Using a pharmaceutical compound called rapamycin, which is known to boost the process of autophagy, Meyer said they found that many of the neurodegenerative characteristics they had previously identified slowed down and the cells seemed to recover and appear more normal.