Research: SAAD and WASHINGTON,

Listed in Issue 263

Abstract

SAAD and WASHINGTON, 1. Alkeus Pharmaceuticals, Inc., 21 Drydock Ave 6th Floor, 02210, Boston, MA, USA. leonide@alkeus.com ; 2. Department of Ophthalmology, Columbia University Medical Center, Eye Research, 10032, New York NY USA. iw2101@columbia.edu discuss how an alternative form of vitamin A which forms dimers more slowly could be used to understand how vitamin A dimers contribute to vision loss in dry age-related macular degeneration (AMD), Stargardt disease and retinal diseases marked by such vitamin A dimers.

Background

We discuss how an imperfect visual cycle results in the formation of vitamin A dimers, thought to be involved in the pathogenesis of various retinal diseases, and summarize how slowing vitamin A dimerization has been a therapeutic target of interest to prevent blindness.

Methodology

To elucidate the molecular mechanism of vitamin A dimerization, an alternative form of vitamin A, one that forms dimers more slowly yet manoeuvres effortlessly through the visual cycle, was developed.

Results

Such a vitamin A, reinforced with deuterium (C20-D3-vitamin A), can be used as a non-disruptive tool to understand the contribution of vitamin A dimers to vision loss. Eventually, C20-D3-vitamin A could become a disease-modifying therapy to slow or stop vision loss associated with dry age-related macular degeneration (AMD), Stargardt disease and retinal diseases marked by such vitamin A dimers.

Conclusion

Human clinical trials of C20-D3-vitamin A (ALK-001) are underway.

References

Saad L1, Washington I2. Can Vitamin A be Improved to Prevent Blindness due to Age-Related Macular Degeneration, Stargardt Disease and Other Retinal Dystrophies? Adv Exp Med Biol.854:355-61.  doi: 10.1007/978-3-319-17121-0_47. 2016.

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