Findings: Advanced forms of dry age-related macular degeneration (AMD), called geographic atrophy, can lead to severe vision loss or blindness. Currently, there are no effective treatments for dry AMD (unlike wet AMD that has many treatment options). A team of researchers, including BrightFocus-funded Dr. Judit Baffi, has identified three “guilty parties” that can trigger the death of retinal pigmented epithelial (RPE) cells, which leads to blindness. It found that these three culprits—the NLRP3 Inflammasome, IL-18, and MyD88 proteins—could be prevented from killing the RPE cells in mice by using drugs or genetics techniques to block their activity.
Significance: This research is the continuation of a pivotal BrightFocus-funded discovery made last year by the same research team. It showed that uncontrolled buildup of a “junk” DNA product, called Alu RNA, can hijack the immune system to kill RPE cells. In this latest breakthrough, the team identified three culprits that participated in the hijacking and killing. Drugs targeted against these proteins may one day be the first treatment option for the hundreds of thousands of Americans with this vision-robbing, advanced form of dry AMD.
BrightFocus currently funds Dr. Judit Baffi, a pivotal member of this research team, for this research. Senior author on this paper, Dr. Jayakrishna Ambati and co-author, Dr. Joshua Dunaief, are former BrightFocus grantees.
This is the continuation of research on an important discovery reported in the journal, Nature, on February 6, 2011 by Dr. Baffi and colleagues.