David Sarraf, MD, on New Vision
Research and Treatments

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By Frances Benham

David Sarraf, M.D., Ophthalmologist, School of Medicine, UCLA, is a clinical practitioner, researcher, teacher and guide to the formation of new physicians. In this work he encounters many patients struggling with the tragedy of Age Related Macular Degeneration (AMD). Rarely does he deal with those who suffer from PXE though he is quite familiar with its impact and therapies, which generally result from research for AMD treatments.

Dr. Sarraf presented to our Los Angeles Conference a comprehensive overview of PXE and its manifestation in the skin, heart and circulatory, gastric and vision systems. He focused on the eye, providing detailed examples of damage resulting from neovascularization in the classic or wet form of macular degeneration, also seen in PXE. This selective review of Dr. SarrafÕs presentation highlights information which may be new to our readers.

Among the most important discoveries in our understanding of neovascularization is the recent identification of VEGF, a protein produced in the body, as a stimulus for the growth of the blood vessels which develop in the retina where they leak and damage the retina, including the macula. The identification of VEGF stimulated research efforts which have, in rapid succession, produced three promising therapies. These are chemicals which, injected into the eye, inhibit VEGFÕs ability to stimulate new blood vessel growth. In addition, some patients have enjoyed improved vision. Unfortunately, these therapies are not permanent. After a time, VEGF levels increase and new blood vessels again develop. Injections must be repeated at intervals with research in progress to increase interval periods.

Macugen, injected at six-week intervals, was approved by the FDA for the treatment of AMD in January 2005. PXE patients also are offered the treatment. At the time of Dr. SarrafÕs report (July 2005) only a few PXE Macugen-treated patients were known about, and those had recently started the process. Since July 2005, other reports have been received with one known case of improved vision.

Two other injected treatments are in clinical trials Ð Lucentis and Avastin. Early reports also indicate vision improvement. The trials also look at safety with reports of a few problems of inflammation and/or infection. Most patients tolerate treatment well even with reservations regarding injection directly into oneÕs eye. Treatment costs and whether they will be covered by Medicare, Medicaid and other insurance programs is a concern. Macugen is covered by Medicare at a cost of around $900 per treatment.

Reports of the efficacy of Lucentis and Avastin are not yet available other than scattered indications that they have been tried in PXE. Dr. Sarraf noted that these encouraging efforts should be followed closely for possible treatment of PXE.

NAPE will continue to follow these efforts and welcomes reports by readers of their experiences with Macugen, Lucentis or Avastin. Several patients have called indicating concern that after one or two treatments they saw no improvement. It should be understood that this is not unusual. Those who are being treated should work with their doctors to achieve an optimal outcome.