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Study identifies potential treatment for lethal childhood leukemia
Date:4/16/2012

New York, NY (April 16, 2012) Columbia University Medical Center (CUMC) scientists have demonstrated that two related enzymes phosphoinositide-3 kinase (PI3K) gamma and delta play a key role in the development of T-cell acute lymphoblastic leukemia (T-ALL), a highly aggressive childhood leukemia that is difficult to treat. The study also showed that a dual PI3K gamma/delta inhibitor can significantly prolong survival in a mouse model of the disease. Further, the dual inhibitor was shown to prevent proliferation and to reduce the survival rate of human T-ALL cells in laboratory cultures, setting the stage for clinical trials. The study appears today in the online edition of Cancer Cell.

"Clearly, we have a drug that is extremely effective against this type of cancer in mice," said study leader Thomas Diacovo, MD, associate professor of pediatrics and pathology and cell biology at CUMC. "If this treatment strategy can safely and selectively target the activity of these enzymes in T-ALL tumors, we might be able to reduce the need for conventional chemotherapies that more broadly affect proliferating cells, including those in healthy tissues. This would be a major advancement in helping to reduce drug toxicities in young patients."

The dual inhibitor was developed by Gilead Sciences.

T-ALL is a cancer that arises during the development of T-cells, a type of white blood cell. The abnormal T cells multiply rapidly, invading and impairing the function of organs critical for sustaining life. T-ALL typically begins in childhood but can also appear later in life. The disease is caused by mutations in DNA, which permit the cancer cell to continue growing and dividing, when a healthy cell would normally die. Left untreated, T-ALL is invariably fatal. It is highly resistant to chemotherapy, compared with other forms of leukemia. The relapse rate is about 25 percent in children and 50 percent in adults.

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Contact: Karin Eskenazi
ket2116@columbia.edu
212-342-0508
Columbia University Medical Center
Source:Eurekalert

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