Medical Breakthroughs: Cancer Drug for Vision

BACKGROUND: Uveitis is a condition characterized by inflammation in the eye. Specifically, the part of the eye known as the uveal tract. The uveal tract is the middle layer of the eye that is considered the nutritional layer because it is rich in blood vessels.

Uveitis has a number of subcategories depending on which part of the uveal tract is inflamed. Because of its rich blood supply, the uveal tract is a natural target for diseases that start in other parts of the body. It can also be caused by other problems within the eye, such as cataract or other changes in the lens or ulcer.

Symptoms of uveitis can be as simple and vague as blinking, squinting or watery discharge from the eye, or it can be as severe as total vision loss in that eye. In uveitis, the cornea, which is normally clear, becomes blue and hazy or dull. The cornea can also become cloudy and the white of the eye can become red and swollen. The colored part of the eye can even become red in some cases.

In up to 75 percent of uveitis cases, the cause of the condition is never determined.

There are two types of uveitis, acute disease that comes on suddenly, runs its course in about six weeks and is done, and chronic disease, which comes on, waxes and wanes over time, and lasts months, years or even decades.

TREATMENT: The normal treatment for uveitis is steroids. While these may help people, steroids come with a whole list of side effects that make many people unable to continue using them. The steroids can be delivered by drops, oral medication, or even injections into the eye depending on the severity of the condition. For many patients, this treatment proves effective, but it is not for everyone.

A DIFFERENT APPROACH: Debra A. Goldstein, M.D., of the University of Illinois at Chicago, is studying a different treatment for her patients who don't respond to steroids. She is using the cancer drug chlorambucil. Chlorambucil is known as an alkylating agent, which means it acts on the patient's immune system. The interesting thing about this drug is that past research has suggested long-term use of chlorambucil can actually cause some people to develop cancer.

Dr. Goldstein and colleagues looked at a different use of the drug -- short-term, high-dose -- and they found success. Her study of 53 uveitis patients showed, after four years, 77 percent of the treated patients experienced no recurrence of the disease and all patients remained cancer --free. Vision also improved an average of three-and-a-half lines on the eye chart. The drug is given in 2 mg pills. Patients in the study took up to 30 mg a day, with an average of 20 mg a day.

SIDE EFFECTS: Dr. Goldstein notes chlorambucil has some important side effects that should be addressed. The drug can cause sterility in men of child-bearing age or can cause women near menopause to go into early menopause. There is a chance it can render women infertile as well. Also, during the study, some patients developed shingles, and two patients required transfusions of platelets when their levels dropped.

FUTURE USES: Dr. Goldstein says the other exciting part of these findings is what it can mean for patients with other inflammatory conditions.

"It may be that patients with, for example, lupus or rheumatoid arthritis, who are progressing despite all of their other medications, or who don't tolerate available medications, may be able to be put into remission with a similar type of treatment," said Dr. Goldstein.


Nan Hoffman
University of Illinois at Chicago
Public Affairs
(312) 355-2954

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