by Dan Roberts
Updated December 2006

Fenretinide (ST-602), a drug that has been used to treat certain cancers, rheumatoid arthritis, acne, and psoriasis, has been found to also slow the production and accumulation of a toxin that leads to vision loss in Stargardt's patients. The toxin, called A2E, is a byproduct of vitamin A, the formation of which encourages production of waste deposits called lipofuscin. These deposits accumulate in the retinal pigment epithelium (RPE), interfering with the RPE's ability to nourish the photoreceptors.

The efficacy study was conducted by Sytera and collaborators at The Jules Stein Eye Institute. Sytera, Inc. has since merged with Sirion Therapeutics, Inc.

Researchers found that A2E accumulation was decreased by 60% in mouse models after 28 days of treatment with fenretinide. Since accumulation of lipofuscin is also a condition of eyes affected by age-related macular degeneration (AMD), Sirion is running FDA-approved multi-center studies using up to 225 AMD patients as subjects, with a proof-of-concept trial having begun in December 2006. AMD subjects provide a larger population for the study than the relatively small number of Stargardt's patients available. If the drug proves effective with dry AMD patients, it may then be used off-label for treatment of Stargardt's disease.

See also Accutane May Inhibit Progression of Stargardt's Disease.

For further explanation of the terms used in this article, see the MD Support Glossary and Eye Anatomy pages.