Researchers discover promising drugs for treating leading causes of blindness

Researchers at the University of California, Irvine, have made a significant discovery in the field of ophthalmology. They have identified a class of small-molecule drugs called “Stress Resilience-Enhancing Drugs” (SREDs) that show promise in treating age-related macular degeneration (AMD), diabetic retinopathy (DR), and retinitis pigmentosa (RP). These retinal diseases are leading causes of blindness globally, affecting over 350 million individuals. Unfortunately, current treatment options for these conditions are limited, leaving many patients with minimal or no effective therapies.

Led by Dr. Krzysztof Palczewski, the researchers developed a novel approach to drug discovery, focusing on preserving retinal structure and function in neurodegenerative conditions. They used selective inhibition of cyclic nucleotide phosphodiesterases, a process targeted by SREDs, to slow down or halt the progression of retinopathies in various animal models representing genetic and environmental factors.

Retinal diseases like AMD, DR, and RP result from disturbances in cellular and tissue stability, which accumulate over time due to repeated exposure to stress. By enhancing stress resilience in the degenerating retina, SREDs demonstrated the ability to preserve tissue structure and function across different models of retinal disease. This breakthrough offers hope for developing effective treatments or preventive measures for these common causes of vision loss.

The study, titled “Stress resilience-enhancing drugs preserve tissue structure and function in degenerating retina via phosphodiesterase inhibition,” was published in the Proceedings of the National Academy of Sciences. The researchers employed a systems pharmacology platform, utilizing advanced disease modeling and characterization techniques, to identify innovative therapies that target the root causes of retinal diseases.

According to lead author Jennings Luu, MD/Ph.D. Doctoral Fellow of Pharmacology in the Medical Scientist Training Program at Case Western Reserve University and Visiting Scholar at the University of California, Irvine, SREDs offer a promising strategy to combat retinal diseases in their early stages, surpassing the efficacy of current standard treatments such as anti-angiogenics, corticosteroids, and nonsteroidal anti-inflammatory drugs (NSAIDs). This approach expands the range of ophthalmic medications available to patients and clinicians.

Luu envisions SREDs becoming the standard of care for age-related diseases, providing relief and improving the quality of life for patients suffering from debilitating ailments. With the lack of viable therapeutic options for these conditions, the potential of SREDs is significant, potentially extending both human lifespan and healthspan regardless of the underlying disease causes.

Based on the groundbreaking discoveries outlined in the published study, Luu and Palczewski have co-founded a startup pharmaceutical company named Hyperion Therapeutics, Inc. The company aims to commercialize the intellectual property associated with their recent findings and introduce new therapeutic agents to the market for the treatment and prevention of retinal diseases like AMD, DR, and RP, as well as other progressive and incurable blinding conditions. Hyperion Therapeutics has received recognition, winning first place in the Morganthaler-Pavey Startup Competition hosted by the Veale Institute for Entrepreneurship. They have also partnered with UCI Beall Applied Innovation in the Wayfinder Incubator Program, further supporting their mission. Through a recently awarded Proof of Product grant, Luu and Palczewski will advance their pipeline therapies towards clinical trials and eventual commercialization.

Source: University of California, Irvine

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