Friedlander said that the researchers hope to advance this approach with clinical trials; a potential pharmaceutical partner is interested in partnering with them once the therapy is optimized for human use. Clinical trials may take several years, and any such treatment would have to prove safe and effective before it would be routinely available.
"Are we ready to go to the clinic tomorrow?no," said Friedlander. "But is this class of therapeutics 'druggable'the answer is 'yes.'"
Diseases Tied to Protein Called VEGF
Many types of blindness can be tied directly to the abnormal growth of blood vessels in the back of the eyethe retina, a soft tissue already rich with vasculature and crowded with light-sensing cells that capture visual cues and send signals to the brain.
In diseases like "wet" macular degeneration and diabetic retinopathy, abnormal blood vessels proliferate under or on top of the retina, respectively, presumably in response to hypoxia, or low oxygen levels. While the precise cause of the hypoxia is not clear, in the case of macular degeneration it may be due to deposits of abnormal molecules leading to inflammation and neovascularization. In diabetic retinopathy, scientists believe that the vessels themselves function abnormally, leaking fluid and bleeding, leading to loss of vision and the growth of additional abnormal vessels.
For many years, scientists have sought to address vision loss by stemming this sort of aberrant growth of blood vessels. In the last decade, much of the focus has been on a molecule found in the human body called VEGF (vascular endothelial growth factor).
VEGF is central to many types of aberrant blood vessel growth. When the body senses too little oxygen, it produces VEGF, and when vessels in the eye sense the elevated levels of that molecule, they sprout new shoots. VEGF and other molecules that promote blood vessel growth activa
|Contact: Mika Ono|
Scripps Research Institute