There is currently no proven treatment for Stargardt disease (SD), an inherited eye condition that affects the retina and causes a slow loss of central vision over years. It affects about one in 10,000 people and may begin to cause symptoms early or late in life.
In Stardardt disease there is an abnormal protein that is involved with transporting vitamin A. In normal eyes Rim protein transports vitamin A from the retina to cells that recycle it for continued use. The retina requires vitamin A to process light for functional vision. Eyes with Stargardt disease have a defective Rim protein. As a result, vitamin A that has been used by the retina tends to accumulate in the retina in the form of toxic byproducts called vitamin A dimers (pronounced, “DYE-mer”). Vitamin A dimers are thought to contribute to permanent retinal damage over time.
Alkeus is a company that is researching the use of a drug that may slow the loss of vision in Stargardt disease. This pill (ALK-001) is taken by mouth once a day. In laboratory studies it appears to slow the formation of vitamin A dimers. It has been studied in the laboratory and now has been cleared by the FDA for study in humans. The study will compare the effect of ALK-001 to placebo (sugar pills) to determine the effectiveness and safety. Eligible patients are healthy individuals between 12 and 60 years of age. Candidates must undergo genetic testing to confirm the diagnosis. The study will last for 24months. If this and other studies prove the usefulness of ALK-001 it will likely be approved by the FDA for general use by ophthalmologists outside of clinical trials in the future.
UPDATE OCT 2022: The TEASE 1 Study reported very favorable results at the American Academy of Ophthalmology meeting in Chicago this month. There was slowing of the rate of vision loss and no prominent safety issues.
For further information and to find the study sites see: Clinical Trials
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