Researchers at the University of Adelaide have conducted the first in-human clinical trial of a new type of treatment for the leading cause of blindness in working-age adults, with promising results.
Retinitis pigmentosa is a genetic disease in which the retinal cells responsible for detecting light do not function properly, causing progressive blindness. Current treatment options for late stages of the disease are limited and there is no cure.
Now, new approaches to treating the disease are offering new hope. Experts from the University of Adelaide, in collaboration with researchers from the University of Washington, conducted a small pilot trial to see if a potential molecular-based treatment could be safely tolerated in humans.
Researchers found that when injected into the eye, some of the damaged retinal cells revived and became sensitive to light again. This occurred even after normal light-sensing cells were lost.
What originally began as a very small trial to test the safety of a drug has now opened up the possibility of a whole new way to treat degenerative eye diseases. ”
Robert Casson, Professor and Principal Investigator, Faculty of Medicine, University of Adelaide
“This is the first clinical trial of a photoswitch drug in humans. Unlike gene therapy, which targets specific mutations, this approach could be used against many different forms of retinal degeneration.”
“It also avoids the need for genetic modification, potentially simplifying treatment and reducing risks.”
Several participants in the small trial reported short-term improvements in their ability to perform visual tests, including walking tasks. One participant with severely damaged retinal cells reported better light perception within two days of treatment.
“We found this treatment to be well tolerated, with no evidence of serious adverse events or adverse ocular effects.” said Professor Casson.
“We also saw early signals suggesting that the drug may have a biological effect. Some participants reported changes in light perception, and brain imaging showed activity in visual areas of the brain after treatment.”
“While this is certainly positive, we must also emphasize that these are preliminary results and need to be confirmed in larger studies.”
Kiora Pharmaceuticals provided industry support for this research, and the results were published in the journal. natural medicine.
A large phase 2 trial is currently underway to more rigorously evaluate whether this treatment can improve vision.
“More broadly, this study establishes a new platform for vision restoration that could be further developed for retinal diseases beyond retinitis pigmentosa.” said Professor Casson.
