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Promising Treatments for Retinitis Pigmentosa are in the Pipeline: Presented at AAO

By Pippa Wysong

CHICAGO, IL -- October 26, 2005 -- Retinitis pigmentosa results from a large number of hereditary conditions and leads to progressive loss of vision. While there are no effective therapies available at the moment to cure any form of the disease (aside from cases due to vitamin A deficiencies), there are several fields of research that yield promise.

According to John Heckenlively, MD, Professor of Ophthalmic Genetics, University of Michigan, Ann Arbor, Michigan, United States, "The possibility of having treatments for some forms of retinitis pigmentosa in the foreseeable future is realistic."

Dr. Heckenlively provided an overview of research being done in this area on October 18^th at the annual meeting of the American Academy of Ophthalmology (AAO).

He divided therapeutic approaches that are under investigation into six main categories. Dietary, nutritional, and environmental approaches include treatments that use vitamin A and antioxidants, docosahexaenoic acid (DHA), and sunlight exposure.

A second therapeutic approach is gene therapy. So far, more than 70 gene mutations have been identified as being causes for retinitis pigmentosa. For instance, researchers are investigating gene replacement using a benign vector, as well as gene ablation therapy which can target DNA, RNA or a protein product, Dr. Heckenlively.

Other areas of interest include stem cell therapy, and retinal transplants. The use of specific growth factors to protect against, prevent or slow apoptosis are also being investigated. Advances are also being made in the field of retinal prostheses for patients with advanced disease, and various approaches are being tried in prototypes.

However, Dr. Heckenlively said, there are barriers to many of these efforts getting to market quickly. One is cost -- getting a treatment to phase-1 trial costs between $5-million and $20-million. There are also ethical issues related to bringing investigational treatments and technologies from animal models to the clinic.

"There are so many forms of retinitis pigmentosa, it is hard for the field to focus on just a few types and treatments," Dr. Heckenlively said.


[Presentation title: Update on Retinitis Pigmentosa: Is Therapy Close? Retina Sub-specialty Day]

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