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Dr. Hendrik Scholl and Dr. Mahmood Shah Launch Clinical Trial to Study Potential Stargardt Therapy

October 23, 2015

Dr. Hendrik SchollDr. Syed Mahmood Shah

Drs. Hendrik Scholl (left) and Mahmood Shah hope to learn more about a potential treatment for Stargardt disease.


Dr. Hendrik Scholl, the Dr. Frieda Derdeyn Bambas Professor of Ophthalmology at the Wilmer Eye Institute, and Dr. Mahmood Shah, assistant professor of ophthalmology at Wilmer, have launched a Phase 2 clinical trial to investigate a novel drug that could potentially treat Stargardt disease. The drug, which can be taken by mouth in capsule form once a day, contains a form of Vitamin A that has been modified with deuterium, a heavier form of hydrogen. Past research has shown that deuterium, found naturally in the human body, is non-radioactive and safe. Pre-clinical studies have suggested that the deuterium-bound Vitamin A may retard the formation of toxic byproducts that occur due to genetic mutations in the retina of patients with Stargardt disease.

“We are excited to be able to move this novel compound from bench to bedside, and bring new hope to patients who would eventually go blind.” said Dr. Scholl, an expert in retinal degeneration and chair of a multinational collaborative effort to study the progression of Stargardt disease. “Since this is a genetic disease with onset as early as childhood, the best method to prevent long-term visual loss is to start treatment as early as possible. Our plan is to enroll adults and children aged 12 and older across multiple sites in the United States where we will measure the progression of disease over a two-year period."

Dr. Scholl’s team, located at seven universities across the U.S., will use advanced outcome measures, including imaging and psychophysical measurements of retinal function, in addition to standard measures such as visual acuity, to evaluate the extent that the drug, known as ALK-001, slows the progression of vision loss.

Stargardt disease is an inherited disease where mutations in one gene impair the transport of Vitamin A inside the retina. Consequently, Vitamin A reacts with itself to form Vitamin A dimers, or aggregates, that have been found to be toxic to retina cells. Ultimately, the disease leads to the loss of central vision and the inability to read, write, drive and recognize faces. There is currently no FDA-approved treatment to slow, prevent or cure Stargardt disease. Animal studies have shown ALK-001 may slow the formation of Vitamin A dimers; the next step is to study the drug in humans. Drs. Scholl and Shah are excited to begin this next phase of research that may result in a new treatment option for Stargardt patients.

Said Dr. Shah: “The launch of this study is the end result of many years of research and signifies a new chapter in our understanding of Stargardt disease.”

For more information on the clinical trial and enrollment criteria, contact:

Shazia Khan, M.D.
Senior Research Project Manager
Wilmer Eye Institute
410-955-5818
skhan79@jhmi.edu

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