2023-05-21| R&D

Scientists Identified Potential New Drugs to Combat Blindness

by Nai Ye Yeat
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Recently, a research team from the University of California, Irvine (UCI) School of Medicine has reported a novel group of small-molecule drugs with potential clinical utility in the treatment of blindness caused by age-related and inherited retinal diseases, including age-related macular degeneration (AMD), diabetic retinopathy (DR), and retinitis pigmentosa (RP).  The study was published in the journal Proceedings of the National Academy of Sciences on May 1.

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Chronic Retinal Diseases as World’s Leading Causes of Blindness

AMD and DR are some examples of chronic and progressive retinal diseases. Molecular and environmental disruptions of cellular stability are believed to lead to the development of these diseases, and these disruptions accumulate over time as repeated exposures to stress lead to progressive visual impairment and eventually vision loss. 

In light of the limited therapeutic options available for these diseases in the present day, it is imperative to seek new approaches in order to treat them from an early stage when there is the greatest chance to preserve the retinal structure and visual function. At present, there are approximately 350 million affected individuals worldwide, however, more than 90 percent of these cases do not have effective treatment options. 

Novel Class of Drugs to Combat Diseases in Earlier Stages

In this current study, UCI’s scientists targeted this unmet medical need, and a systems pharmacology platform for therapeutic development was employed. First, universal molecular mechanisms across distinct models of age-related and inherited retinal degenerations were identified by using single-cell transcriptomics, proteomics, and phosphoproteomics approaches. 

As a potential treatment for neurodegenerative conditions, specifically the world’s leading causes of blindness, age-related and inherited retinal diseases, researchers have introduced an innovative class of therapeutics called “stress resilience-enhancing drugs” (SREDs). Research findings demonstrated that the SRED therapeutic intervention enhanced resilience to acute and chronic forms of stress in the degenerating retina, thereby preserving tissue structure and function across multiple models of age-related or inherited retinal disease. Collectively, these findings point to the potential clinical utility of a new class of therapeutics that could be used to treat or prevent the most common causes of blindness.

Overall, SREDs may provide new hope for patients suffering from progressive retinal diseases, as well as provide promising results at an earlier stage of disease progression. Scientists from UCI involved in this study expect to implement SREDs as a standard of care in the future, thereby helping more and more people in need, especially older adults. “SREDs represent a promising strategy for patients and clinicians to combat disease in earlier stages with superior efficacy over the current standard of care, augmenting the arsenal of ophthalmic medications presently available in anti-angiogenics, corticosteroids, and nonsteroidal anti-inflammatory drugs (NSAIDs),” said Jennings Luu, Visiting Scholar at UCI and the lead author of the research article.

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