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Re: anything new about stargardts

From: Robert B. Greer
Email: rbgreer@uclink.berkeley.edu
URL: spectacle.berkeley.edu
Date: July 21, 2003
Time: 18:33:09

Comments

I’m unaware of any discoveries that will reverse the effects of Stargardt disease or have been shown to slow or stop progression of the disease in humans. On the other hand, there have been some interesting findings reported in the literature recently about studies in animal models. In the April 15, 2003, issue of the Proceedings of the National Academy of Sciences of the United States of America (http://www.pnas.org/) there is an article titled: “Treatment with isotretinoin inhibits lipofuscin accumulation in a mouse model of recessive Stargardt’s macular degeneration.” Isotretinoin, also known as Accutane, was found to inhibit the accumulation of lipofuscin in mice and, thus, retain retinal function. This is encouraging but does not address the issue of reversal, nor does it directly apply to humans. Human trials with Accutane may happen some day but care must be taken because Accutane is known to cause birth defects and its long-term side effects are not well understood.

Also in the Proceedings of the National Academy of Sciences of the United States of America, June 20, 2000, was an article published titled “Biosynthesis of a major lipofuscin fluorophore in mice and humans with ABCR-mediated retinal and macular degeneration” that found lipofuscin accumulation was inhibited in mice with Stargardt disease reared in the dark. While no human trials have been conducted to see if similar results would occur, the study did go so far as to say “The observed inhibition of A2E accumulation in abcr-/- mice raised under total darkness suggests that limiting light exposure may reduce the rate of disease progression in humans with recessive ABCR-mediated diseases.” Once again, there is no direct evidence in humans that limiting light will slow or stop the progression of Stargardt disease nor does the evidence suggest that the disease can be reversed.

 
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