Mutant Genes in High-Risk Childhood Leukemias ...( Results of collaborative study could ...),Health

Results of collaborative study could lead to development of new diagnostic tools and a new therapeutic target in high-risk disease

MEMPHIS, Tenn., May 19 /PRNewswire-USNewswire/ -- A research team has pinpointed a new class of gene mutations, which identify cases of childhood acute lymphoblastic leukemia (ALL) that have a high risk of relapse and death. The finding suggests specific drugs that could treat this high-risk leukemia subtype in children, particularly because such drugs are already in clinical trials for similar blood diseases in adults.

While the cure rate in pediatric ALL has reached about 85 percent, the remaining high-risk cases have proven especially intractable because they arise from different, unidentified genetic mutations.

Discovery of the mutations was led by scientists from St. Jude Children's Research Hospital, the Children's Oncology Group (COG), the University of New Mexico Cancer Research and Treatment Center, Albuquerque, N.M., and the National Cancer Institute (NCI), part of the National Institutes of Health (NIH). This research was done as part of the NCI Therapeutically Applicable Research to Generate Effective Treatments (TARGET) initiative, which seeks to utilize the study of genomics to identify therapeutic targets in order to develop more effective treatments for childhood cancers. The article appears online May 18 in the early edition of the Proceedings of the National Academy of Sciences.

"We have made such great progress in curing children with ALL that the main challenge is now the remaining high-risk patients," said St. Jude Scientific Director, James Downing, M.D., a co-senior author of the study. "We still do not know how to accurately identify these patients and effectively treat them to provide the highest chance for a cure. The problem is that this high-risk group is likely a heterogeneous mixture of biologic subtypes."

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