Dr. Gregory Hageman recently spoke to US Congressional leaders, briefing them on dramatic advances in age-related macular degeneration research since 2005, when his and three other research teams first identified Complement Factor H, (CFH), a gene variant strongly associated with increased risk for developing AMD. These new findings may change the way age-related macular degeneration is treated - and potentially cured.
The identified gene, Complement Factor H (CFH), and its protein product are normally engaged in the control of a portion of the body’s immune system. Normal levels of healthy CFH protein may protect against AMD, while variants in the gene result in poor regulation of this system and can lead to the development of age-related macular degeneration.
Hageman expects that Phase 1 clinical trial on a therapy using the CHF protein in an augmentation strategy similar to that of treating diabetes with insulin could start as early as mid 2009.
He also noted that since most of the CFH is made in the liver, liver transplant recipients offer an opportunity to study what occurs when an individual receives a different form of CFH following transplantation. Researchers hope that these liver studies will reinforce the concept of providing age-related macular degeneration patients with doses of the protective protein or, in the future, with gene therapy approaches that would allow the liver to produce the protein on its own.
Check the National Alliance for Eye and Vision Research website for more information on this research.
13 October 2008