Blind World


Macular Degeneration.
'Gene Silencing' Experiment Aims to Save Eyesight.





November 9, 2004.

By ANTONIO REGALADO,
Staff Reporter,
THE WALL STREET JOURNAL.




At 11:30 a.m. today at an eye clinic in Cleveland, 79-year-old Carla McCullough is scheduled to become the first person ever to receive a potentially powerful new form of medicine known as "gene silencing."


The new type of genetic drug, to be injected by doctors inside Mrs. McCullough's right eye, is designed to shut off a growth factor that is causing blood vessels in her eye to grow excessively, the main symptom of a progressive form of blindness called wet macular degeneration.


The test will be a big step for a new class of genetic medicine that has generated unusual enthusiasm among scientists and venture capitalists. Gene-silencing drugs are based on a recently discovered biological process that cells naturally use to block certain genes from making proteins.


As scientists have learned to control that silencing process, companies have raced into the field with potential drugs. Acuity Pharmaceuticals of Philadelphia is sponsoring the study at Cleveland's Retina Associates Inc., where Mrs. McCullough is scheduled for treatment.


Publicly traded Sirna Therapeutics Inc. of Boulder, Colo., expects to launch its own human tests for macular degeneration as early as this week. In June, Merck & Co. entered an alliance with another start-up company to create its own eye drugs.


Howard Robin, president and chief executive at Sirna, says gene-silencing drugs could be more potent and present fewer side-effects than other types of drugs. In the future, they could be "formulated as an eye-drop," he believes.


The drug Mrs. McCullough will receive is designed to block production of vascular endothelial growth factor, a protein that causes blood vessels to grow. The studies by Acuity and Sirna will involve only a few dozen patients and are designed to determine only if the gene silencing drugs are safe.


Mrs. McCullough said she was nervous about her pioneering role. "I am really terrified and crossing my fingers. I don't want to be blind. And I am the only driver in our family," she said.



Copyright 2004, THE WALL STREET JOURNAL.




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