Expert Answers on Stargardt’s Disease By THE NEW YORK TIMES Michael Nagle for The New York Times Katie James, who learned she had Stargardt’s disease when she was 11, uses a screen to magnify text. She is one of the patients featured in Patient Voices: Macular Degeneration. Stephen Rose of the Foundation Fighting Blindness responds to reader questions about Stargardt’s disease, an illness similar to macular degeneration that arises in late childhood. Stem Cells vs. Gene Therapy Question. Which holds more promise for treating Stargardt’s — stem cells or gene therapy? Aside from those, are you aware of other treatments being researched? James Ledous, Austin, Tex. Answer. Dr. Stephen Rose responds: This is a great question. Both gene therapy and a stem cell treatment for Stargardt’s disease are in Phase I clinical trials. In both of these studies, safety is the primary outcome being measured. In a few years, as these trials move forward, we’ll know better about how well these treatment approaches work for saving or restoring vision in those with Stargardt’s. With that said, if gene therapy proves safe and works as we hope, it will be the best approach in most cases for treating people with less advanced vision loss. By correcting the genetic defect, gene therapy halts the disease in its tracks. A single treatment will hopefully halt vision loss for several years, perhaps a lifetime. Stem cells can work in two different ways to treat retinal disease. In some cases they will act like a “drug factory,” providing sustained release of vision-saving proteins. In other cases, especially in advanced conditions, they will serve as functional replacements for retinal cells lost to disease. Stargardt’s disease is similar in some ways to the dry form of age-related macular degeneration, so some emerging dry A.M.D. treatments may benefit people with Stargardt’s. Both diseases involve the accumulation of damaging waste products in the retina. Trials are currently under way for various dry A.M.D. treatments — for example, ReVision’s fenretinide and Acucela’s ACU-4429 — that reduce or slow the buildup of these waste products. These approaches may also benefit people with Stargardt’s disease. Early Results for Stargardt’s Treatment? Q. Now that the Phase I trials are under way to treat Stargardt’s patients, thanks to the persistence of Advanced Cell Technologies, have any early results been leaked out to the research community, and if not, when can we expect to learn the results of these first rounds of trials? RCRCGutz, Colorado A. Dr. Stephen Rose responds: The Advanced Cell Technology clinical trial is just beginning to treat participants, so no results are known yet. Also, the current clinical trial is designed to assess the safety of the implanted cells (that is, verify they do no harm) and to try different doses of cells to see if any changes in vision can be detected. This clinical trial is scheduled to enroll its last participant in July 2013. Saffron for Stargardt’s Disease Q. Can you please comment on the use of saffron as a way to rescue damaged retinal cells? Clinicaltrials.gov lists a trial of saffron 20 mg daily as a potential therapy for fundus flavimaculatus. Doc Rock, Los Angeles A. Dr. Stephen Rose responds: The pilot clinical trials you reference are testing saffron as a potential treatment in participants with early-stage age-related macular degeneration and Stargardt’s disease, a related retinal degeneration that appears in late childhood that is also known as fundus flavimaculatus. These clinical trials are still recruiting participants, and therefore no results are known; the AMD study is slated to have enrolled all its participants by January 2012, and the Stargardt’s trial by March 2012. Preclinical evidence indicates the potential effectiveness of saffron as a retinal protectant in animal models of retinal degenerative disorders. Patients’ macular function will be tested by visual acuity and macular cone-mediated electroretinogram (focal electroretinogram, or FERG) according to a standardized technique.
Posted on: Fri, 30 Aug 2013 10:13:38 +0000
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