Macular Degeneration :: Award will enable researcher to bring lab discovery to clinical trial

The number of people in the U.S. with macular degeneration is greater than that with all types of cancer combined. Macular degeneration can lead to blindness, something that people fear even more than premature death. This award will help further research into this condition.

Dr. Jayakrishna Ambati’s research into macular degeneration, which causes blindness among hundreds of thousands of people each year, has been heralded by the journal Science as one of the most exciting scientific breakthroughs of the year.

Now, because of a prestigious new national honor, the University of Kentucky researcher is getting the opportunity to take a recent discovery from the lab into clinical trials that may help patients successfully fight age-related macular degeneration.

Ambati is the first UK researcher ever to receive the Burroughs Wellcome Fund (BWF) Clinical Scientist Award in Translational Research. In fact, he is one of only 11 scientists in the nation to receive the award this year.

The award ? which carries with it a grant of $750,000 ? will allow Ambati and his team to fine-tune their discovery and develop a phase 1 clinical trial with patients.

“The number of people in the U.S. with macular degeneration is greater than that with all types of cancer combined,” Ambati said. “Macular degeneration can lead to blindness, something that people fear even more than premature death.

With the help of the Burroughs Wellcome Fund, my colleagues and I are eager to advance new preventive and therapeutic strategies to hasten the day when blindness from macular degeneration is no longer inevitable. While it is extremely gratifying to be individually recognized with this prestigious award, it rightly belongs to my entire highly talented laboratory.”

Ambati’s work centers on accelerating the discovery of classes of compounds that suppress angiogenesis, or the growth of new blood vessels. Researchers are currently looking for therapies that will inhibit the growth of new blood vessels under the retina, which is the most debilitating type of macular degeneration, referred to as the “wet” form. Ambati already has discovered one new class of compounds to inhibit blood vessel growth and the funding will help him narrow down one or two lead candidates in this class to test clinically in patients with age-related macular degeneration.

“UK’s ophthalmologists have transformed lab discoveries into innovative treatments for other conditions that threaten vision,” Ambati said. “The biggest obstacle, however, is the cost of biomedical research. We are excited about the funding from the Burroughs Wellcome Fund, and will continue to seek additional opportunities to move our ideas forward so macular degeneration can be eradicated.”

Ambati is engaged in the clinical practice of ophthalmology as well as in basic research. The foundation’s award is for independent physician-scientists who are dedicated to translational research?the two-way transfer between laboratory research and patient treatment?and mentoring physician-scientist trainees.

“We hope these awards will lead to better understanding of the mechanisms of disease as well as new methods of diagnosing, treating and preventing disease,” said BWF President Enriqueta C. Bond. “BWF is particularly interested in supporting physician-scientists who bring novel ideas and new approaches to translational research.”

In 2003, Ambati and colleagues were responsible for discovering the first animal model of age-related macular degeneration. The model provides a method to study how and why humans develop this condition and provides a platform on which to develop and validate new therapeutic strategies. Ambati’s animal model closely resembles the anatomical appearance, biochemical composition, and functional disruption of macular degeneration in humans. More recently, Ambati and his brother, Dr. Balamurali Ambati, of the Medical College of Georgia, jointly published a paper in the journal Nature detailing their discovery that a protein known as sVEGFR-1 is singularly responsible for warding off blood vessel growth in the cornea. The article “Corneal Avascularity is due to soluble VEGF receptor-1” appeared in the Oct. 26, 2006 print edition and online version of Nature, and was cited by Science as one of the breakthroughs of 2006.

Ambati is associate professor and vice-chair in the UK College of Medicine Department of Ophthalmology and Visual Sciences and is the Dr. E. Vernon Smith and Eloise C. Smith Endowed Chair in Macular Degeneration.

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