Published in Research

New class of therapeutics may treat retinal diseases

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3 min read

A recent study published in Proceedings of the National Academy of Sciences (PNAS), led by researchers from the University of California, Irvine (UCI), identified small-molecule drugs with the potential to treat degenerative retinal diseases.

Give me some background first.

The small-molecule drugs evaluated in this study activate intrinsic biological mechanisms of stress resilience, which helps to address the degenerative damage caused by chronic and progressive retinal diseases.

Investigators evaluated the impact of these small-molecule drugs on conditions such as age-related macular degeneration (AMD), diabetic retinopathy (DR), and retinitis pigmentosa (RP).

Now talk about the study.

The study authors proposed that these small-molecule drugs, called stress resilience-enhancing drugs (SREDs), could be a new class of therapeutics used to treat acute and/or chronic stress-associated retinal conditions.

Tell me more.

Researchers used integrative single-cell transcriptomics, proteomics, and phosphoproteomics to identify universal molecular mechanisms across various models of age-related and inherited retinal degeneration, characterized by impaired physiological resilience to stress.

Findings?

The selective, targeted pharmacological blocking of cyclic nucleotide phosphodiesterases (PDEs) resulted in downstream activation of protective mechanisms and synergistic blockage of degenerative processes.

This led to a slowing—or even halting—of the development and progression of retinopathies as well as preserving tissue structure and function across genetic and environmental animal models.

Significance?

With limited options available for non-invasive treatments of progressive retinal conditions, these findings provide a promising avenue for clinicians to treat age-related and inherited retinal diseases (IRDs) earlier and with superior efficacy.

Expert thoughts?

Lead author Jennings Luu, MD/PhD, noted that “it is our expectation that SREDs will someday serve as a standard of care for human aging, effectively providing patients the means to diminish suffering from debilitating ailments for which there currently exist no viable therapeutic options, thereby extending human lifespan and healthspan irrespective of disease etiology.”

Anything else?

Based on the results of this study, a startup pharmaceutical company, Hyperion Therapeutics, Inc., was co-founded by Dr. Luu and Krzysztof Palczewski, PhD (corresponding author) to commercialize the intellectual property associated with their recent findings.

Further, they hope to bring new therapeutic agents to the market for the treatment or prevention of AMD, DR, RP, and other progressive, incurable blinding diseases.

What’s next?

Drs. Luu and Palczewski are currently acting as co-investigators on a newly awarded Proof of Product grant (thanks to a recent partnership via the UCI Beall Applied Innovation’s Wayfinder Incubator Program) that will support the development of their pipeline therapies toward clinical trials and future commercialization.