Lucentis

What is Macugen?

Wet AMD

Vision deterioration in a person with age-related macular degeneration may look like this. It could also be more severe or less severe.

Macugen (scientific name pegaptanib sodium) is a drug approved by the U.S. Food and Drug Administration (FDA) for the treatment of "wet" or "neovascular" age-related macular degeneration (AMD). Wet AMD is the most aggressive form of this sight-threatening eye disease. It has the potential to cause rapid deterioration of vision, especially when left untreated. The cause of the vision loss is the growth of abnormal new blood vessels underneath parts of the retina where they do not belong. The biologic process by which these new, unwanted vessels form is called "angiogenesis."

Macugen targets a key step in angiogenesis. It is injected into the back of the eye through the sclera (white part of the eye). When it reaches the retina, it binds with a protein called vascular endothelial growth factor (VEGF), which is a stimulator of new blood vessel growth. As a result, the VEGF is unable to stimulate angiogenesis, and the eye is protected from the damage it causes.

Macugen was the first in this new class of drugs, called anti-VEGF drugs, to be FDA-approved for the treatment of wet AMD. As such, it represents a breakthrough in eye disease treatment. Previously developed treatments that involve lasers aim to destroy the results of angiogenesis, the unwanted new blood vessels. In contrast, anti-VEGF drugs target their underlying cause.

In the human clinical trials that led to its FDA approval in 2004, Macugen was effective for helping patients with AMD keep their vision longer. In one trial, 73% of the patients who were given the drug for one year lost fewer than 15 letters of visual acuity on the eye chart. In another trial 67% of the patients who were given the drug for one year lost fewer than 15 letters of visual acuity on the eye chart. Overall, the rate of vision decline in the patients who received the drug was slower than in the patients who received a sham, or fake, treatment.

What Should I Expect While Receiving This Treatment?

Macugen is usually given once every six weeks. Therefore, if you are being treated with it, you will visit your eye doctor every six weeks. He or she may want to see you in between treatments as well. It is important to remember that not all patients have the same response to the treatment. It may work better for you, or not as well, than for other patients. Also, it may not be as effective during a second year of treatment as it is during a first year. Your eye doctor may treat you with this drug alone or with this drug and another treatment for macular degeneration, such as steroid eye injections or laser.

Receiving an injection in your eye, such as with Macugen or a steroid, is a scary thought, of course. However, your eye doctor will numb your eye first and take precautions to prevent eye infection. Most patients find that receiving the injections is not as bad as they thought. Also, most prefer to continue receiving them rather than risk more vision deterioration.

In addition to slowing down vision deterioration from macular degeneration, Macugen has been shown to be a very safe treatment. Side effects that can occur are mostly in the eye, due to the injection itself and not the drug. The most common side effects are burning sensation, eye pain, redness, light sensitivity, vision loss, blurred vision, visual disturbances, high blood pressure and cataract. Your eye doctor will monitor you closely for these possible side effects as well as for an increase of pressure inside your eye, which injections can cause.

One of the reasons this treatment is so safe has to do with the way it targets VEGF. It is a "selective" VEGF blocker, which means it does not block all of the various forms of VEGF. (Some forms are actually helpful to the body.) Instead, it targets only the one most involved in new blood vessel growth, VEGF 165. This makes it less likely to cause side effects beyond the eye.

In comparison, another anti-VEGF treatment that is also available, Lucentis (ranibizumab), targets all of the various forms of VEGF, making it what is known as a "pan-VEGF" blocker. In the Lucentis clinical trials, a higher percentage of patients experienced stabilization of vision, and about one third experienced improvement. However, patients treated with Lucentis are theoretically at a higher risk for more serious side effects because of its pan-VEGF action.

While Macugen and Lucentis differ in this way, in clinical trials and so far in general use, both have been safe. Your eye doctor will choose your treatment for wet AMD based on your individual case.

Hope for the future? New clinical trials.

NeoVista, Inc. is a company that is developing an intraocular epiretinal radiation device intended for the treatment of the wet form of age-related macular degeneration. If you, or someone you know, are interested in participating in the CABERNET Trial, please follow the link below:

What is Macugen?

What is Macugen?

What Should I Expect While Receiving This Treatment?

Hope for the future? New clinical trials.

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