At a news conference today in Chicago, Cubs first baseman Derrek Lee announced his partnership with Boston Celtics CEO and co-owner Wyc Grousbeck and the Carver Nonprofit Genetic Testing Laboratory at the University of Iowa Roy J. and Lucille A. Carver College of Medicine to combat Leber congenital amaurosis (LCA), an important cause of genetic blindness in children.
"Wyc and I look forward to lending our support to this important project," Lee said. "Seeing all the recent scientific progress up close has given us hope that treatments for LCA will not be too far away, and we hope that our efforts will bring similar encouragement to lots of other families who have been affected by this condition."
Grousbeck agreed. "It is amazing to think that we have come as far as we have. Just 10 years ago, not a single one of the LCA genes had been identified. Now the specific disease-causing gene can be found in more than two-thirds of affected patients, and some clinical trials of promising new treatments are just about to start," he said.
Lee and Grousbeck call their effort Project 3000 because they plan to raise money to provide state-of-the-art genetic testing for every man, woman and child in the United States with LCA - about 3000 individuals in all. The project's other important goals include:
- providing up-to-date information about LCA to all patients and families with the condition;
identifying the remaining genes responsible for LCA;
making genetic testing the standard of care for LCA; and
helping patients enroll in clinical trials designed to find effective treatments for the disease.
The John and Marcia Carver Nonprofit Genetic Testing Laboratory in the Carver Family Center for Macular Degeneration at the UI will be the lead research site in this genetic testing effort. The interdisciplinary Carver Family CMD is a major international center for research and treatment of degenerative diseases of the eye. Other partners in the Project 3000 effort include the Foundation Fighting Blindness of Owings Mills, Md. and the Foundation for Retinal Research of Chicago, Ill.
LCA is an inherited form of blindness that is typically recognized shortly after birth. In most cases, affected children are otherwise completely healthy, but a subset of children do have other problems such as learning disabilities, autism, seizures or kidney disease. At least nine different genes are currently known to cause LCA, and as a group these genes are responsible for about two-thirds of all cases.
"There is no cure for LCA yet, but significant progress has been made," said Edwin Stone, M.D., Ph.D., an investigator of the Howard Hughes Medical Institute at the UI and director of the Carver Family Center for Macular Degeneration. "Many LCA-causing genes have been identified, and some treatments have already been shown to work very well in animal models of the disease. We think that we can really accelerate the progress toward a cure for this disease by identifying the specific genetic cause of the disease in thousands of patients."
Project 3000 is a historic undertaking in that it marks the first time that genetic testing for any eye disease has been offered to every affected person in the United States.
"Thanks to the tremendous courage and commitment of the Lee and Grousbeck families, this project offers real hope to the patients and families affected with LCA," said Keith Carter, M.D., professor and head of the Department of Ophthalmology and Visual Sciences in the UI Carver College of Medicine. "To borrow a phrase from the world of baseball, we want to knock this one out of the park."
More information on Project 3000 is available online at www.carverlab.org.
STORY SOURCE: University of Iowa Health Science Relations, 5139 Westlawn, Iowa City, Iowa 52242-1178