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Drug offers promising approach to improve outcome for children with high-risk leukemia
Date:11/15/2013

Combining the drug gemtuzumab ozogamicin (GO) with conventional chemotherapy may improve the outcome of bone marrow transplantation for some children battling high-risk acute myeloid leukemia (AML), according to a study led by St. Jude Children's Research Hospital. The results appear in the current edition of the journal Cancer.

For young AML patients with suitable bone marrow donors, transplantation offers the best chance of a cure. Being free of even low levels of detectable cancer cells prior to transplantation is associated with better patient survival. This analysis found that combination therapy helped to eliminate minimal residual disease (MRD) in young AML patients who initially had a poor response to chemotherapy. By reducing MRD levels prior to transplantation, it is likely that GO contributed to the excellent outcome of these patients, researchers said.

The drug, trade named Mylotarg, received accelerated approval from the U.S. Food and Drug Administration in 2000 for treatment of AML in adults. The drug was voluntarily withdrawn in 2010 following questions about its safety and efficacy. Since then several studies conducted while the drug was authorized reported promising results in adults with AML. Those reports prompted this analysis of pediatric AML patients who received GO in a clinical trial conducted while the drug was available.

Researchers said the results add to evidence that both adults and children battling AML may benefit from treatments that use a similar mechanism as GO to combat AML. The drug was not associated with severe toxicity in this or other recent studies.

Unlike traditional chemotherapy that kills rapidly dividing cells, both malignant and healthy, GO was designed to kill more selectively. The drug works by targeting a protein carried on the surface of about 90 percent of AML cells. Work has begun on new strategies, including antibody-based therapies, that target the same AML surface
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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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