AMD & Stargardt's Disease- New Clinical Studies

Gene therapy, stem cell therapy and vitamin A are being clinically tested as potential cures for improved vision in people suffering from age related macular degeneration and Stargardt’s disease.

It’s a fundamental breakthrough,” said Paul Sieving, director of the National Eye Institute.

Nearly 200 genes involved in vision loss have been identified and biochemical interactions have been explained.

Stargardt’s is linked to a single gene (ABCA4) so it is easier to study. Vitamin A provides the fuel for photoreceptor cells in the eye to sense light but when it remains in the eye it forms dangerous clumps that are trapped in the retinal cells, impairing vision. Dr. Ilyas Washington, Columbia University professor, devised a molecule that doesn’t clump as much but still aids vision. This form of vitamin A has showed promise in animals with the Stargardt gene defect. A clinical trial in 100 patients may start next year. Hopefully, it will prevent further deterioration of vision, said the CEO of the company making the pills.

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