Age-related macular degeneration patient receives investigational eye drops

An 88-year-old man at The Methodist Hospital in Houston is one of two patients in the world today to receive an investigational eye drop that may restore sight for those suffering from neovascular (wet) age-related macular degeneration, a major cause of central visual loss and one of the leading causes of blindness in people over 60 in the United States.

Currently, the standard treatment requires multiple injections into the diseased eye.

Today, the patient received the topical administration of the drug called TG100801 in a Phase IIa, multi-center trial sponsored by TargeGen, Inc. This represents the first wet AMD patient in Texas and one of the first worldwide to be treated with eye drops alone for the management of the disease. In pre-clinical models, the investigational drug blocked new blood vessel growth and leakiness, which lead to AMD, by inhibiting VEGF and other related proteins that are believed to play a critical role in the formation of new blood vessels. TG100801 is also designed to reduce inflammation (including edema), a common symptom of AMD, and other back of the eye diseases, including diabetic macular edema and diabetic retinopathy. In as little as a month, investigators may learn if the eye drops were effective.

“If the eye drops work, it will be much better for the patient,” said Dr. David Brown, a retinal surgeon at Methodist and primary investigator at Vitreoretinal Consultants. “We're continually looking for more effective treatments for our many AMD patients. If we can get this effect without injections, that would be a major breakthrough.”

AMD occurs in two forms: dry and wet. While all cases begin as the dry form, wet AMD accounts for about 85 percent of all AMD-related blindness and can result in sudden and severe vision loss. The dry form is associated with atrophic cell death of the central retina or macula. The wet form is caused by growth of abnormal blood vessels that leak fluid and blood under the macula causing scar tissue that destroys the central retina. In 2006, the FDA approved the injectable drug Lucentis for wet AMD, based on data from two large Phase III clinical trials, in which Brown and his colleagues enrolled the most patients worldwide and published their data in the New England Journal of Medicine. Dry AMD, the most common form of the disease, represents approximately 85 to 90 percent of all AMD cases in the country. Currently, there is no FDA approved treatment for dry AMD.