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Mutant Genes in High-Risk Childhood Leukemias Identified
Date:5/19/2009

Willman, M.D., director and CEO of the University of New Mexico Cancer Research and Treatment Center and a co-senior author of the study. "A patient may have multiple different genetic lesions that target different cellular pathways to induce leukemia. Thus, it is very important to develop new therapies that target these specific mutations, and our discovery of JAK as target now allows us to begin to develop clinical trials with JAK inhibitors for children and adults with this form of disease."

In further studies, the researchers plan to identify mutations in kinase genes and other enzymes that underlie high-risk ALL, as well as explore how these abnormalities might work together to drive the cancers.

The discovery that mutations in JAK underlie some cases of high-risk ALL is enough to warrant clinical trials of inhibitory drugs to treat such cancers.

"JAK-inhibiting drugs are now moving into clinical trials for treatment of such adult myeloproliferative diseases as polycthemia vera, essential thrombocytosis and primary myelofibrosis," Downing said. "We expect that there will soon be initial clinical studies to assess the safety and effectiveness of these drugs in children with relapsed ALL in which JAK mutations have been identified within their leukemic cells."

Such studies would be coordinated by the COG, an international clinical trial cooperative group supported by the NCI.

Other authors of the paper are Racquel Collins-Underwood, Letha A. Phillips, Xiaoping Su, Wei Liu and Brenda Schulman (St. Jude); Sarah Tasian and Mignon Loh (University of California San Francisco); Meenakshi Devidas (Children's Oncology Group); Susan Atlas, I-Ming Chen and Richard C. Harvey (University of New Mexico Cancer Research and Treatment Center, Albuquerque); Robert J. Clifford, Daniela Gerhard, Malcolm Smith and Jinghui Zhang (National Cancer Institute); William C
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SOURCE St. Jude Children's Research Hospital
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