Saturday, September 08, 2012

AMD Update 21: Good News for Patients With Stargardt’s Disease

Stargardt’s disease (also known as Stargardt macular dystrophy) is the most common form of inherited juvenile macular degeneration. Inherited as an autosomal recessive trait, it is a severe form of macular degeneration that begins in late childhood, leading to legal blindness. Stargardt’s disease is symptomatically similar to age-related macular degeneration, and it affects approximately one in 10,000 children.

There are currently two approaches underway to treat Stargardt’s, the use of embryonic stem cells to produce retinal pigmented epithelial (RPE) cells, which are injected subretinally, to replace damaged RPE cells (which take care of the photoreceptor cells) (sponsored by Advanced Cell Technology), and the use of gene therapy, to replace mutations in the ABCA4 gene, which cause Stargardt’s disease, and produces a dysfunctional protein (RPE) that cannot perform its transport function. As a result, photoreceptor cells degenerate, and vision loss occurs (sponsored by Oxford BioMedica).

A few days ago, the Foundation Fighting Blindness announced a new program to 1) study people with Stargardt’s to gain information about how best to treat it, 2) to better understand the disease’s progression, and 3) to identify potential patients for future clinical trials.

Here is how FFB described their ProgStar program, an important step in understanding and treating Stargardt’s disease.

Sept. 4, 2012 - The Foundation Fighting Blindness Clinical Research Institute is launching a natural history study of people affected by Stargardt disease. Known as ProgStar, the study has three primary goals: 1) determine the best outcome measures to accelerate evaluation of emerging treatments; 2) better understand disease progression for selecting future clinical trial participants; and 3) identify potential participants for forthcoming clinical trials.

The study, taking place in multiple locations worldwide, will combine both prospective and retrospective analyses. The prospective portion, for which physicians will track the progression of disease in participants, will last at least a year, with a possible one-year extension. Physicians will also retrospectively analyze disease progression by reviewing participants' past medical records. 

"Stargardt disease has a complex effect on the retina," says Dr. Hendrik Scholl, of the Wilmer Eye Institute, Johns Hopkins Hospital, ProgStar's protocol principal investigator and study director. "Also the rate of disease progression can vary between people and is difficult to measure. These issues can make selection of clinical trial outcome measures and participants challenging. But ProgStar will help us design better human studies of potential treatments and increase our chances of success."

During ProgStar, researchers will evaluate a number of outcome measures, including retinal images, visual fields and visual acuity.

ProgStar is preparing to enroll its first participants in early 2013. The study's clinician-scientists, listed below, will recruit as many as 300 patients from their practices. No additional recruitment will be conducted, and ProgStar will not involve administration of potential therapies.

Principal investigators for the study are:

    Dr. Paul Bernstein, University of Utah (Salt Lake City)
    Dr. David Birch, Retina Foundation of the Southwest (Dallas)
    Dr. Gerald Fishman, The Chicago Lighthouse (Chicago)
    Dr. Samuel Jacobson, University of Pennsylvania (Philadelphia)
   Dr. Michel Michaelides, Moorfields Eye Hospital (London, United Kingdom)
    Dr. José Sahel, Institut de la Vision (Paris, France)
    Dr. Hendrik Scholl, Johns Hopkins Hospital (Baltimore)
    Dr. Janet Sunness, Greater Baltimore Medical Center (Baltimore)
    Dr. Eberhart Zrenner, University of Tübingen (Tübingen, Germany)

Participants for the study are being recruited only from the existing patient populations of the nine principal investigators listed above. People who are not existing patients of one of these physicians are not eligible for the study.

Participating in study design are Drs. Frederick Ferris and Brian Brooks, of the National Eye Institute, Dr. Johanna Seddon, of Tufts Medical Center, and Dr. Richard Weleber, of Oregon Health & Science University.

Dr. Srinivas Sadda, of the Doheny Eye Institute, University of Southern California, will be analyzing imaging and visual field data collected from the study centers.


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