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A milestone in the treatment of Stargardt juvenile macular degeneration. A modified vitamin A drug slows the disease progression in a clinical trial.

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The TEASE (“Tolerability and Effects of ALK-001 on Stargardt Disease”), study was a two-year, multicenter, double-masked, randomized, placebo-controlled clinical trial evaluating ALK-001 in patients with atrophic Stargardt disease. The study took place at seven research centers across the United States.

The investigational drug, ALK-001, is a chemically modified vitamin A taken as a daily pill. In the data presented by Prof. Scholl, ALK-001 slowed the growth rate of retinal atrophy, a measure of the area of damaged retina, by approximately 30% per year.

Stargardt disease affects over 40,000 people in the United States. Typically, patients start experiencing vision loss in their teenage years. With no approved therapy, the vast majority of people become legally blind by age 50.

Vitamin A is essential for vision and retinal health. However, the vitamin forms toxic deposits in the retina. In patients with Stargardt disease, the vitamin A deposits form at an accelerated rate, leading to retinal atrophy, which grows with time, and vision loss.

To date, the TEASE study is the only trial with both a statistically significant and clinically meaningful effect on the progression of Stargardt disease.

“The study showed that ALK-001 slow the progression of Stargardt disease in its advanced stage, where many believed any slowing was nearly impossible,” said Prof. Scholl. “We are extremely excited to see such a positive effect in advanced-stage patients and look forward to studying the drug’s effects in earlier stages of Stargardt, where an even larger treatment effect could be expected.”

ALK-001 is the only investigational drug that slows the formation of the vitamin A deposit without causing side effects, such as night blindness. “These results provide the first clinical evidence that vitamin A deposits we’ve been studying for nearly 30 years contribute to the progression of retinal degeneration,” said Dr. Ilyas Washington, the scientist inventor of the modified vitamin A drug.

To read the original ARVO abstract, click here.

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