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Gene Mutations Seem to Make a Leukemia More Deadly
Date:11/10/2010

By Amanda Gardner
HealthDay Reporter

WEDNESDAY, Nov. 10 (HealthDay News) -- Researchers have pinpointed a gene with multiple mutations that seems to separate people who die quickly from acute myeloid leukemia (AML) from those who do not.

The findings, appearing in the Nov. 11 issue of the New England Journal of Medicine, could help identify which AML patients are the best candidates for more aggressive initial treatment. The insights might also provide a target for future therapies.

"We have to know the gene before we can design the targeted therapy, and we need to understand the mechanism," explained study senior author Dr. Timothy Ley, a professor of medicine and genetics at Washington University in St. Louis. "The mutations provide a new clue to find out how the mechanism might affect gene regulation in patients with these mutations. Hopefully, [additional studies] will tell us a lot more about how to think about reversing effects of mutations in patients with this disease."

"The whole idea is to move towards personalizing our health-care choices and to do that we have to better understand the pathways that lead to the development and progression of each person's cancer," added Dr. David Rizzieri, director of the hematologic malignancy program at Duke University. "This is a potential major step forward to better understanding the role of this molecular pathway in both the development and progression of AML."

AML is a cancer of the blood-forming cells, affecting about 13,000 Americans every year, 9,000 of whom will die.

Overall, only 20 percent of patients with AML will survive five years but there are three risk groups of patients, each of which confers a different prognosis.

"Favorable" risk patients tend to do better with standard therapy, while those with poor risk need to get a bone-marrow transplant when they go into their first remission, Ley explained.

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