I Can See Clearly Now: At last, therapies improve vision lost to macular degeneration

Among revolutions, this may be a quiet one, yet it's dramatically changing the lives of people with one of the most heartbreaking disorders associated with aging—failing eyesight.

After years of frustration, scientists are developing treatments that can slow the inevitable descent into darkness caused by "wet" macular degeneration, an eye disease that strikes 200,000 Americans a year. Macugen, a drug on the market since January, may in some cases reverse vision loss. And clinical trials are showing that a second drug, Lucentis, which could be available within 18 months, may be even more effective.

"For the first time we have a treatment where visual acuity is actually improved," said Robert D'Amato, M.D., an ophthalmologist with Harvard Medical School and Children's Hospital in Boston. "People can actually see better than they could when the [Macugen] trial started."

Macular degeneration is the most common cause of blindness after age 50. The dry form of the disease occurs when pigment cells beneath the retina deteriorate, diminishing eyesight. The more serious but less common wet form accounts for only about 10 percent of the more than 1.75 million cases of age-related macular degeneration in the United States.

Wet macular degeneration is caused by the abnormal growth of leaky blood vessels in the retina, the eye's light-sensing organ. The new drugs stop the action of VEGF, a natural substance that stimulates such growth and gradually damages the macula, the central area of the retina that allows fine-detail vision needed for reading or sewing.

Most patients are left with only peripheral vision, and many can no longer recognize faces, read signs or do ordinary chores.

"I went from being quite dependent on my husband to being able to drive legally" as a result of Macugen treatments, says Charlotte Warner, 83, of Strongsville, Ohio. When she joined the drug trial three years ago, vision in her right eye was gone and her left eye was deteriorating.

"I'm an avid reader, but I was having to use a magnifying glass even to see the big print," she adds.

"I started to improve gradually," she says, "and after three doses I was elated" because some vision was returning. Now the vision in her left eye is 20/25, and she can read and watch TV.

Macugen, made by Eyetech Pharmaceuticals Inc. in New York, won approval from the federal Food and Drug Administration last year. Genentech Inc., of South San Francisco, the manufacturer of Lucentis, reported in July that monthly injections of the drug benefited most of the 716 patients in a one-year clinical trial, with 34 percent of those who got a higher dose (5 milligrams instead of 3) reporting improved vision: They were able to read smaller type—three lines lower—on an eye chart.

Ironically, the discoveries leading to treatments for eye diseases came from cancer research, not eye research. Lucentis, in fact, is an altered version of Avastin, a colon cancer drug that some eye doctors use to treat wet macular degeneration. Philip Rosenfeld, M.D., of the University of Miami Bascom Palmer Eye Institute in Florida, says of his experiments with Avastin, "I'm seeing in these patients something I've never seen before: they are getting better. This is a 'Gee whiz!' 'Wow!' experience."

The medication drains fluid from retinal tissue, he says, and returns the retina to its normal shape. He says its benefit seems to last longer than that of Macugen or Lucentis, usually up to six months or more in two-thirds of his patients. Rosenfeld hopes to start a large clinical trial with Avastin soon and expects to report current results at a scientific meeting in February.

Until now, the most effective treatments have used laser beams to burn leaky blood vessels in the retina, preserving sight temporarily. But eyesight does not improve, and over time vision loss continues.

Macugen and Lucentis are injected directly into the eye, an off-putting notion to some. But side effects—inflammation, eye irritation, reduced clarity and discomfort after treatment—are minimal, experienced by less than 1 percent of patients.

Some doctors have expressed concern about price, worrying that Lucentis may cost as much as $4,000 a dose. But a Genentech representative, Dawn Kalmar, said a price has not been set. It isn't known yet if Medicare and insurers will cover the drug.

Macugen injections, given every six weeks, cost $995 a dose, and are covered by Medicare and some insurance companies.

The drugmakers are setting up programs to help people on limited incomes pay for the medications.

The possibility of curbing eye damage by blocking VEGF was discovered by Anthony Adamis, M.D., vice president and chief scientific officer at Eyetech, and his fellow ophthalmologist Joan Miller, M.D., of the Massachusetts Eye and Ear Infirmary in Boston. Such developments are propelling eye research into an exciting new phase. Adamis likens it to the Sputnik era, at the dawn of the space age. "We're right at the beginning."

Visit the websites of

The American Macular Degeration Foundation

The National Eye Institute, or

Prevent Blindness America