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NEI Statement

National Institutes of Health Stimulates the Development and Testing of New Therapies for Advanced Age-Related Macular Degeneration (AMD)

National Eye Institute
National Institutes of Health

The National Eye Institute (NEI) of the National Institutes of Health (NIH) will fund a new multicenter clinical trial to compare two drugs currently used to treat advanced age-related macular degeneration (AMD). AMD is the leading cause of blindness among older Americans. The two drugs are Lucentis^TM (ranibizumab) and Avastin® (bevacizumab).

Lucentis^TM was approved earlier this year by the U.S. Food and Drug Administration (FDA). The approval was based on evidence from clinical trials showing that Lucentis^TM slows the rate of progression of vision loss from advanced AMD. In addition to a low rate of developing vision loss, approximately one-third of patients treated in these trials had improved vision at 12 months.

Avastin®, a drug closely related to Lucentis^TM, has been approved by FDA for treating colorectal cancer, but not specifically for ophthalmic uses. However, Avastin® has been widely used off-label to treat advanced AMD. The new comparative study will assess the relative safety and effectiveness of Avastin® and Lucentis^TM.

Angiogenesis is a term used to describe the growth of new blood vessels. Angiogenesis plays a crucial role in the normal development and maturation of tissues. Angiogenesis is also an important feature of many diseases. In cancer, angiogenesis supplies the increased demand for oxygen and nutrients that allow tumors to grow and spread. Abnormal blood vessel growth is also sight-threatening in a complication of diabetes, diabetic retinopathy, and is an important cause of visual loss in advanced age-related macular degeneration and other eye diseases. Inhibition of angiogenesis as a possible treatment for these types of diseases is an extremely active area of research and drug development. In other diseases, such as coronary artery disease and stroke, therapeutic stimulation of angiogenesis is being investigated as a potential way to replace damaged vasculature. Over the past several decades, NIH has invested significant resources in the promising area of angiogenesis research. NEI alone has provided approximately $570 million for more than 1900 research project grants designed to improve our understanding of the role of angiogenesis in major causes of blindness and visual disability.

A large and broad body of research has established that vascular endothelial growth factor (VEGF) is an important component of the biological pathways involved in triggering and sustaining the growth of new blood vessels and in controlling vascular permeability, and that VEGF and the VEGF pathway is an important target for drug development. The pace of this research was accelerated significantly in the late 1980s when two groups of scientists working independently, one group at Monsanto Company, and a second group at Genentech, Inc., identified and characterized VEGF at the molecular and genetic level.

In advanced AMD, abnormal new blood vessels grow beneath the retina. These abnormal blood vessels can leak blood and fluid, and can cause severe vision loss. NEI-supported research has helped establish that VEGF is an important part of the biological pathways involved in triggering and sustaining the growth of new blood vessels in the retina. Research also demonstrated that VEGF is present in higher levels in a number of eye diseases, such as AMD. NEI has supported more than 300 research studies at a cost of nearly $95 million to better understand the role of VEGF in eye diseases.

At this point, two anti-VEGF therapies, Macugen® (pegaptanib sodium) and Lucentis^TM, have already been approved by FDA specifically for the treatment of advanced AMD. Paul A. Sieving, M.D., Ph.D., director of NEI, said, "We are pleased that NIH-supported research is stimulating the development and testing of new, effective treatments for AMD."

Lucentis^TM and Avastin® are products of Genentech, Inc.
Macugen® is a product of Eyetech Pharmaceuticals, Inc. and Pfizer, Inc.