Possible Stargardt Disease Treatment Shows Promise

A potential drug in the macular degeneration treatment pipeline rooted in initial funding from BrightFocus Foundation gives hope to those suffering from a genetic form of macular degeneration known as Stargardt disease. At the American Academy of Ophthalmology (AAO) meeting earlier this week it was announced that Gildeuretinol trials results demonstrated a significant slowing of retinal atrophic lesions in Stargardt disease. In 2010, BrightFocus Foundation funded the first study, led by Dr. Ilyas Washington, that led to the development of this novel treatment.  

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation and breakthrough therapy designation to the treatment, an enriched form of vitamin A modified to prevent the formation of toxic clumps in the eye. The drug is the first and only medicine to treat Stargardt disease by targeting the underlying causes of the disease and reducing the rate of vitamin A dimerization.  

Stargardt disease is an inherited form of macular degeneration with no FDA-approved treatment available to date. While it affects the same part of the central retinal, called the macula, as age-related macular degeneration (AMD), it is different from AMD. 

Children and young adults who are born with Stargardt disease often develop symptoms including difficulty reading, black spots in their central vision, or changes in color perception between the ages of 10 and 40. Some people begin to lose their vision in childhood, while others experience vision loss as an adult.  

Known in scientific terms as ALK-001 (C20-D3-vitamin A), the proposed therapy is based on foundational work done by Ilyas Washington, PhD, a former Columbia University chemist and co-founder of Alkeus Pharmaceuticals, the developer of this potential drug. BrightFocus awarded Dr. Washington with one of the first research grants of his scientific career, allowing him to begin exploring a possible role for enriched vitamin A in treating vision disease. 

“BrightFocus was the first major funder of my academic lab. They gave me the opportunity – and the confidence – to believe that I could someday stop someone from losing their sight,” Dr. Washington said. 

“BrightFocus Foundation is proud to have supported the initial research that led to the development of this potential breakthrough treatment that gives hope for people living with Stargardt disease,” said Stacy Pagos Haller, President and CEO of BrightFocus Foundation. “Research holds the key to defeating vision loss.” 

Macular degeneration is a leading cause of vision loss and irreversible blindness in Americans aged 60 years and older and advanced AMD is a leading cause of irreversible blindness and visual impairment in the world. As many as 20 million Americans have some form of macular degeneration, including both early and later stages of the wet and dry forms. 

BrightFocus has invested nearly $100 million in vision research worldwide on the causes and potential prevention and treatment of macular degeneration and glaucoma. BrightFocus Foundation’s Macular Degeneration Research program has funded several breakthrough studies that have led to a better understanding of the disease and novel treatments (learn more). 

Vision loss can often cause feelings of isolation, and BrightFocus is proud to offer AMD Community Circle, a free monthly Zoom program where people with macular degeneration can join together to share tips, ask questions, and connect with others experiencing similar circumstances.    

Twice per month, BrightFocus shares expert information from doctors with the low-vision community through its accessible and interactive BrightFocus Chats audio information program. During these free audio discussions, participants can learn about the latest treatment options, promising scientific research, and tips for living with macular degeneration and glaucoma. 

Explore more: 

• What is Stargardt Disease?

• Treatments for Macular Degeneration  

• Understanding Macular Degeneration (printable brochure)  

About BrightFocus Foundation  

BrightFocus Foundation, through its flagship programs Alzheimer's Disease Research, Macular Degeneration Research, and National Glaucoma Research, is on a mission to stop Alzheimer's and vision loss in its tracks. 

Alzheimer's disease, macular degeneration, and glaucoma affect more than 300 million people worldwide—1 in 7 people over age 40 in the U.S.—and disproportionately affect communities of color. BrightFocus is committed to investing in bold research that generates novel approaches, diagnostic tools, and treatments that serve all populations. Learn more at brightfocus.org.