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Children's Hospital researcher leads multipronged attack on infant leukemia

America. "Even the largest medical center has a relatively small number of patients with infant leukemia," said Dr. Felix. "Collaborating with the Children's Oncology Group allows us to capture data about and treat the vast majority of infants throughout North America who have acute leukemia."

The full grant encompasses four projects, each of which hones in on a piece of the infant leukemia puzzle. In infant leukemia, a gene called mixed lineage leukemia (MLL) breaks and recombines with one of many partner genes to form a translocation, an abnormal rearrangement within a chromosome. Because MLL plays a critical role in blood cell development, the translocation causes the overproduction of defective white blood cells that are the hallmark of leukemia.

Dr. Felix has pioneered methods to identify and describe the features of MLL translocations. Her project within the SCOR grant will investigate how potential drugs may trigger programmed cell death, called apoptosis, in leukemia cells in infants. Another project, led by Cheryl L. Willman, M.D., of the University of New Mexico, will use microarrays (DNA chips) to identify drugs that may selectively attack cells with MLL translocations. The project also seeks to unravel the molecular circuitry in leukemia cells in order to predict those infants most likely to benefit from specific targeted drugs.

Donald Small, M.D., Ph.D., of Johns Hopkins University, leads a SCOR project to advance the development of new drugs to target abnormalities in a gene called FLT3 that are associated with uncontrolled growth of the leukemia cell population.

A final team under the SCOR grant, led by Michael Cleary, M.D., of Stanford University, will characterize the MLL leukemia stem cell that gives rise to all other leukemia cells, because the stem cells are essential targets for new treatment to eradicate the disease. Core research facilities at several other academic medical centers will provide specialize
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Contact: John Ascenzi
Ascenzi@email.chop.edu
267-426-6055
Children's Hospital of Philadelphia
5-Dec-2006


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