A new report by British physicians published online in the journal Retinal Cases & Brief Reports used the antiangiogenic drug Avastin® (bevacizumab) to treat Sorsby Fundus Dystrophy (SFD), a genetic, early onset form of macular degeneration. It is apparently the first time Avastin has been used to treat the rare genetic condition, which causes a build-up of leaky blood vessels behind the retina, resulting in vision loss.

Two patients with SFD, both in their 30s, were treated with injections of Avastin, which resulted in resolution of abnormal blood vessel growth and significantly improved vision in the treated eyes. Avastin, best known as a cancer therapy that blocks the growth of tumor blood vessels, is also used to treat “wet” age related macular degeneration (AMD), the leading cause of blindness in the western world in people over age 50.

Prognosis in SFD is poor, as patients lose central visual function in the fourth or fifth decades of life mainly because of the development of new blood vessels in the eye. SFD patients generally respond poorly to conventional treatments.

Professor Andrew Lotery, a consultant ophthalmologist at Southampton General Hospital, United Kingdom, who submitted the report for publication said, “Since SFD patients have not responded well to treatments, and because mutations that lead to the condition are not yet fully understood, we wanted to investigate other methods of treatment, including Avastin. We discovered that the drug prevented loss of sight and improved vision in the younger patients suffering from SFD over a long period. This is an exciting new treatment for what is a rare condition affecting young people.”