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Existing drugs offer new treatment options for high-risk childhood leukemia subtype
Date:8/13/2012

Scientists have identified new genetic alterations underlying a high-risk subtype of the most common childhood cancer that could be effectively targeted with existing leukemia therapies.

The study focused on a subtype of acute lymphoblastic leukemia (ALL) known as Philadelphia chromosome-like ALL (Ph-like ALL). This subgroup accounts for as much as 15 percent of childhood ALL that is associated with a high risk of relapse and a poor outcome. The genetic changes driving the disease were previously unknown for about half of all patients with Ph-like ALL. The work identified new alterations in genes that regulate how cells grow and proliferate. St. Jude Children's Research Hospital investigators led the research, which appears in the online edition of the journal Cancer Cell.

Investigators also showed that the leukemia cells were sensitive to several targeted therapeutic agents, imatinib and dasatinib, which are already being used against other leukemias, but not this subtype. The findings suggest patients with Ph-like ALL may benefit from the addition of these drugs to current chemotherapy regimens.

"One of the next steps will be to continue work on laboratory tests to rapidly identify patients whose cancer cells carry these alterations and to develop clinical trials to test targeted therapies," said Charles Mullighan, M.D., Ph.D., an associate member of the St. Jude Department of Pathology, and a corresponding author of the study.

The study involved sequencing the RNA of cancer cells from 15 patients with Ph-like ALL and whole genome sequencing of two of those patients. Whole genome sequencing involves deciphering the DNA molecule, which contains the complete set of instructions for building and maintaining life. Cells use RNA to translate DNA's instructions into proteins. Sequencing RNA provides a snapshot of gene activity in a cell.

The work was part of the National Cancer Institute's Therapeutically Appl
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Contact: Summer Freeman
summer.freeman@stjude.org
901-595-3061
St. Jude Children's Research Hospital
Source:Eurekalert

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