University of Utah doctor working on pill for age-related blindness

National health surveys have estimated that more than 21.2 million adult Americans report they either "have trouble" seeing, even when wearing glasses or contact lenses, or that they are blind or unable to see at all, according to the American Foundation for the Blind.

Those not born with sight-inhibiting illnesses have a good chance of developing it as they age, even more so when it's prevalent in the family in the form of age-related macular degeneration and diabetic retinopathy.

Until now, there has been no treatment for the devastating eye diseases, which are mostly caused by leaky blood vessels that end up blurring or eliminating vision in old age. But an international research group led by a University of Utah cardiologist say a pill may one day effectively treat such conditions by tricking cells into making blood vessels stronger.

"By fooling the cell, we've prevented inflammation from damaging blood vessels of the eye," said Dr. Dean Y. Li, professor of internal medicine and director of the molecular medicine program at the U.'s School of Medicine.

In a study published Sunday in Nature Cell Biology online, Yi and his colleagues identify a potential "small molecule" or pharmaceutical drug that stabilizes weak blood vessels and prevents them from leaking. Leaky blood vessels are a primary cause of age-related macular degeneration and diabetic retinopathy, two of the leading causes of blindness, and other diseases.

The drug would likely work by fooling cells into sensing that a receptor has been activated, triggering a process in which vessels are made stronger and less affected by inflammation.

"Our study is the proof of concept that this molecular pathway can be manipulated by a drug," Li said.

The most recent discovery, developed with researchers at the University of California, San Diego, as well as the University of Bonn, Germany, builds on ground-breaking research led by Li last spring. That study exhibited that when a cellular receptor was activated, blood vessels became stronger and stopped leaking in mouse models with the debilitating conditions.

Researchers identified a protein that activates the receptor, a protein that is hard for humans to generate on their own.

Li said that if the cell is activated, perhaps by a drug, it can then "instruct blood vessels to strengthen."

The potential drug would not necessarily activate the cell but would fool it into thinking it had, putting the cell into action to produce stronger blood vessels, thus putting off blindness.

Scientists have identified the process, but drug proteins have been too large to help. This new research further develops the possibility for a drug with a smaller molecular structure to help stabilize blood vessels in the eyes, making it possible for the search for a usable compound to begin, Li said.

It will take years, however, before a drug can be introduced on the market, and the process will have to be undertaken by private industry. The resources and money required to develop a drug that receives U.S. Food and Drug Administration approval, are well beyond that of an academic laboratory, Li said.

"We've taken it as far as we can," he said.

e-mail: wleonard@desnews.com