Aggressive infant leukemia has surprisingly few genetic changes

James Downing, MD

This year acute lymphoblastic leukemia (ALL) will be diagnosed in about 3,000 U.S. children and teenagers. Some of the new patients will be infants or even newborns. Researchers with the St. Jude Children’s Research Hospital – Washington University Pediatric Cancer Genome Project have completed the most comprehensive DNA analysis yet of the most common form of ALL in infants. What scientists found surprised them.

The leukemia cells of most infants with ALL - as many as eight out of every 10 patients - have a genetic rearrangement. The alteration affects a gene named MLL. Researchers showed these patients had few other genetic changes. In fact, the patients had one of the lowest mutation rates of any cancer. That was a surprise because infant ALL with the MLL rearrangement is an aggressive disease with a poor prognosis.

“This is one of the rare subtypes of pediatric ALL where there has been little progress in the last 15 years,” said James R. Downing, MD, St. Jude president and chief executive officer. “This study provided insight into the biology of this tumor and told us where we need to focus our efforts to develop new therapies.”

At St. Jude, work has begun to find new compounds and develop drug combinations to improve the cure rates for infants with this high-risk leukemia.

The study was published online in the journal Nature Genetics. The corresponding authors are Downing, Tanja Gruber, MD, PhD, of St. Jude Oncology, and Anna Andersson, PhD, formerly of St. Jude.

March 6, 2015

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