Eye On the Cure Research News

Feb 2, 2021

Very-Long-Chain Polyunsaturated Fatty Acids Improve Vision in Mice with a Form of Stargardt Disease

Researchers believe the VLC-PUFAs may benefit people with autosomal dominant Stargardt disease, AMD, and possibly other retinal degenerations

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Foundation-funded researchers from the Moran Eye Center, University of Utah, improved the visual acuity and retinal sensitivity of mice with Stargardt disease (ELOVL4 mutations) by feeding them a very-long-chain polyunsaturated fatty acid (VLC-PUFA) not normally available through diet. The VLC-PUFAs also improved retinal function of mice without retinal disease.

The study results, published online in the journal Proceedings of the National Academy of Sciences (PNAS), led the investigators to believe that VLC-PUFAs may be therapeutic for people with autosomal dominant Stargardt disease, age-related macular degeneration (AMD), and potentially other retinal degenerations.

Research has shown that PUFAs such as docosahexaenoic acid (DHA) and eicosapentaenoic acid (EPA), available through foods such as coldwater fish and dietary supplements, may confer health benefits such as prevention of heart disease and inflammation. However, the benefits of DHA and EPA for retinal degenerative diseases have not been consistently observed in clinical trials.

Paul Bernstein, MD, PhD, lead investigator of the Utah team, says that VLC-PUFAs — a special class of lipids that are not as well understood as DHA and EPA — are normally synthesized in the retina and testes and are believed to help maintain the health and function of photoreceptors, the retinal cells that process light and make vision possible. Because VLC-PUFAs are otherwise scarce, the investigators produced synthetic VLC-PUFAs for the study. Dr. Bernstein says that his team’s VLC-PUFA production process can be scaled up to make sufficient quantities for evaluation in humans.

“Our results for the VLC-PUFA studies in mice are very promising. With that said, additional research is needed to optimize the formulation, dosing, and timing of the intervention, especially for evaluation in humans,” says Dr. Bernstein. “Because VLC-PUFA abnormalities are a common feature of blinding conditions such as AMD and diabetic retinopathy, further studies are warranted.”