New Drug Approved for Common Cause of Blindness

June 30, 2006 — -- At first, Gloria Cohen thought her failing vision was simply a result of nearing 40 and needing reading glasses.

But when she noticed that people looked "wavy" when viewed from the corner of her eye, she realized it was probably something more serious. And it was -- she had wet age-related macular degeneration, a serious eye illness that can lead to blindness.

Now a 71-year-old former teacher, the Massachusetts resident has been involved in an ongoing clinical trial that started in December 2005 by Dr. Joan Miller, chief of ophthalmology at the Massachusetts Eye and Ear Infirmary.

Since starting the trial and receiving a new experimental medication, Cohen's vision has improved.

"I have a lot to thank Dr. Miller for," Cohen said. "[This] drug is wonderful. ... I would have never gotten to [this] drug if it was not for Dr. Miller."

And now others like Cohen can get the same help. The drug she was taking, Lucentis, was approved by the Food and Drug Administration today. The drug was developed by Genentech Inc. and Novartis Ophthalmics.

Most cases of macular degeneration occur with age and are termed age-related macular degeneration, or AMD. This typically affects people in one eye with a likelihood of affecting the other eye over time.

Lucentis treats the so-called "wet" form of macular degeneration, the less common form of the disease -- 85 to 90 percent of macular degeneration is actually the "dry" form.

However, even though "the dry is more common ... the wet tends to be more devastating," said Dr. Wiley Chambers, deputy director for the division of anti-infective and opthalmologic products at the FDA.

Damages the Retina

Dry AMD occurs when light-sensitive cells in the macula gradually break down and blur central vision, which leads to a slow loss of vision. Wet AMD happens when new blood vessels grow under the retina and eventually leak blood and fluids, causing damage to the macula, the part of the eye responsible for central vision and the ability to see details.

About 15 million people in the United States have AMD, according to Genentech. Current estimates show that there are more than 200,000 new patients diagnosed with wet AMD every year, according to Dr. Eugene de Juan, president of the American Society of Retina Specialists.

Lucentis works by binding to a protein, dubbed VEGF-A, which is believed to play a critical role in forming new blood vessels, the company said.

Eye doctors hailed the new drug.

"It is the first drug to substantially improve vision in a significant proportion of patients," said Dr. Jayakrishna Ambati, vice chairman of ophthalmology and visual sciences at the University of Kentucky.

In trials that compared people receiving Lucentis and those receiving placebo injections, 95 percent of patients treated with Lucentis improved or maintained vision, according to Genentech.

"There is no other effective medication [for wet AMD]," said de Juan.

Another drug, Avastin, produces effects similar to Lucentis but is used off label, meaning the FDA hasn't approved it yet for treating AMD, since it is primarily used as a nonrelated cancer treatment. And Macugen, which is FDA approved, is another wet AMD medication but does not significantly improve vision, experts said.

Despite Lucentis' successful results, some experts still have reservations about this new drug. Its cost might prove problematic for those who need the medication. One dose of Lucentis could cost about $1,500 to $2,500, said de Juan.

Has to Be Injected Into Eyes

Also, many questions have still been left unanswered, such as how long the drug works over time. Two ongoing studies are also under way to evaluate the dosage routine and the safety of doses, Genentech said.

Since the drug needs to be injected into the eyes, "one of the next advances with this drug would be to come up with a less-invasive way to deliver it," Miller said.

Also, there is concern that treating wet AMD will basically just turn it into dry AMD, said Dr. Richard Bensinger, an ophthalmologist from Seattle, who added that "dry AMD is not really treatable with anything."

A combination of certain vitamins can only slow down the progression of degeneration, not stop it.

"Until the molecular trigger for AMD is discovered so that it can be attacked directly, we will be stuck with this disease in increasing amounts as the population ages," Bensinger said.