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Uveitis is a severe eye inflammation that affects children and young adults. There is no cure, but it can be controlled with strong steroids and other drugs, taken every day by mouth, to suppress the immune system. However, because these drugs block the immune system, they can cause decreased kidney function, cataracts, glaucoma, and brittle bones. Researchers at the National Eye Institute (NEI) and the National Cancer Institute (NCI), both part of the NIH, may have found an alternative therapy, called humanized anti-Tac monoclonal antibody. This biological substance controlled uveitis as effectively as the standard treatment in this study, but with a marked decrease in complications and side effects. Researchers found that of the 10 patients with uveitis given the new treatment, nine had minimal side effects, no recurrence of the disease, and no loss of vision.
"The implications of these findings are significant," said Carl Kupfer, MD, director of the NEI. "They have the potential to greatly improve the quality of life for patients with uveitis. The drugs now used as standard treatment for uveitis can cause serious and unpleasant side effects in the eye and other parts of the body. This new treatment may someday allow uveitis patients to avoid these problems."
There were other benefits from the new treatment, including a drop in patients' cholesterol and glucose levels. "As patients were taken off their standard medication and given the new treatment, we saw an improvement in some body functions that are adversely affected by the standard treatment drugs," said Robert Nussenblatt, MD, chief of NEI's Laboratory of Immunology and the study's first author.
Thomas Waldmann, MD, chief of the Metabolism Branch of NCI's Division of Clinical Sciences, said the antibody used in this study has been used to help patients receiving organ transplants, but is just now being introduced for diseases that affect the body's immune system. "This antibody not only gives us a new perspective for the treatment of uveitis, but has real implications for other autoimmune disorders and how doctors may treat patients in the future," he said.
Dr. Nussenblatt said the next research step involves using the new medication to treat patients with Behçet's disease, a chronic, systemic disease characterized by recurring lesions of the mouth and skin. Uveitis is a major symptom of Behçet's disease. "We will then look to conduct a larger, multicenter study on a much greater number of patients," Dr. Nussenblatt said. "We may also study the possibility of applying the new treatment directly to the eye instead of intravenously."
Dr. Kupfer said that these initial findings provide an excellent example of "lab-to-bedside" research. "One of the greatest strengths of the intramural program at the NIH is that laboratory research can be conducted and the findings quickly applied to a small group of patients before large-scale testing," he said.