A recent NIH study explores the potential of eye drops to slow vision loss in animal models. These drops contain a peptide derived from pigment epithelium-derived factor, a protein naturally found in the eye. The drops demonstrated promising results by preserving retinal cells in diseases like retinitis pigmentosa and age-related macular degeneration. Researchers are optimistic about future human trials following these encouraging outcomes.
March 24, 2025
New eye drops slow vision loss in animals: Study
"This study shows PEDF-based eye drops can slow
progression of retinal diseases." - Patricia Becerra/NIH
Researchers have developed
eye drops that extend vision in animal models of a group of inherited diseases
that lead to progressive vision loss in humans, known as retinitis pigmentosa.
Key Points
1 PEDF-based
drops tested in animal models of retinitis pigmentosa
2 Study
led by Patricia Becerra and Alexandra Bernardo-Colon
3 Eye
drops contain peptides derived from natural proteins
4 Plans
to start human trials following promising animal results
The eye drops contain a small fragment derived from
a protein made by the body and found in the eye, known as pigment
epithelium-derived factor (PEDF).
PEDF helps preserve cells in the eye's retina. A
report on the study is published in Communications Medicine.
"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, Ph.D.,
chief of NIH's Section on Protein Structure and Function at the National Eye
Institute and senior author of the study.
"Given these results, we're excited to begin
trials of these eye drops in people."
All degenerative retinal diseases have cellular
stress in common. While the source of the stress may vary -- dozens of
mutations and gene variants have been linked to retinitis pigmentosa, AMD, and
other disorders -- high levels of cellular stress cause retinal cells to
gradually lose function and die.
Progressive loss of photoreceptor cells leads to
vision loss and eventually blindness.
In this new study, led by first author Alexandra
Bernardo-Colon, Becerra's team created two eye drop formulations, each
containing a short peptide. The first peptide candidate, called
"17-mer," contains 17 amino acids found in the active region of PEDF.
A second peptide, H105A, is similar but binds more
strongly to the PEDF receptor. Peptides applied to mice as drops on the eye's
surface were found in high concentration in the retina within 60 minutes,
slowly decreasing over the next 24 to 48 hours. Neither peptide caused toxicity
or other side effects.
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