Researchers develop eye drops that slow progressive vision loss in mice

mda2staff — Wed, 26 Mar 2025 15:55:59 +0000

March 24, 2025 By Jordana Joy, Associate Editor

Researchers developed eye drop formulations 17-mer and H105A, both containing a short peptide, to administer to mice in the form of a drop on the eye’s surface

Eye drops that extend vision in animal models of a group of inherited diseases that lead to progressive vision loss in humans were recently developed by researchers at the National Institutes of Health (NIH).^1,2 The researchers, who published a report on the study in Communications Medicine, developed eye drops that contain a small fragment derived from a protein in the body and found in the eye known as pigment epithelium-derive factor (PEDF). It has been found that PEDF helps preserve cells in the retina.

“While not a cure, this study shows that PEDF-based eye drops can slow progression of a variety of degenerative retinal diseases in animals, including various types of retinitis pigmentosa and dry age-related macular degeneration (AMD),” said Patricia Becerra, PhD, chief of NIH’s Section on Protein Structure and Function at the National Eye Institute and senior author of the study, in a news release. “Given these results, we’re excited to begin trials of these eye drops in people.”

In the study, led by Alexandra Bernardo-Colón, researchers used specially bred mice that lose their photoreceptors shortly after birth. Once cell loss begins, the majority of the mice photoreceptors die in a week. Researchers created 2 eye drop formulations, each containing a short peptide, to administer to mice in the form of a drop on the eye’s surface. One of the formulations, 17-mer, contains 17 amino acids found in the active region of PEDF. H105A, the second formulation, is similar to 17-mer but binds more strongly to the PEDF receptor.¹

According to the release, when peptides were applied to the mice’s eyes in a 5–10 μl eye drop,² a high concentration in the retina was found within 60 minutes, slowly decreasing over the next 24 to 48 hours. Through the 1-week period of treatment, mice retained up to 75% of photoreceptors and continued to have strong retinal responses to light. Additionally, both 17-mer and H105A eye drops decreased pro-apoptotic BAX and increased anti-apoptotic BCL2 levels in photoreceptors. When given a placebo, mice had few remaining photoreceptors and little functional vision at the end of the week. Neither peptide formulation caused toxicity or other side effects in the study.¹

“For the first time, we show that eye drops containing these short peptides can pass into the eye and have a therapeutic effect on the retina,” said Bernardo-Colón in the release. “Animals given the H105A peptide have dramatically healthier-looking retinas, with no negative side effects.”

retinitis pigmentosa Archives | Macular Degeneration Association

Researchers develop eye drops that slow progressive vision loss in mice