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Strategies converge on target in rare leukemia

John Crispino, PhD Integrated approach points to drug for AMKL

August 2012

For most cells, more than two copies of the entire genome can be a telltale sign of cancer. But for megakaryocytes - bone marrow cells that can give rise to thousands of platelets – having several genomic copies is normal. In their healthy state, these cells can harbor as many as 64 copies of the full complement of human DNA, a state known as polyploidy. When the normal development of megakaryocytes goes awry, they can lose this unique feature and start down the path toward a rare form of cancer known as acute megakaryoblastic leukemia (AMKL). Researchers can spot these cellular changes, but the underlying causes - and how to reverse the course of disease — have been difficult to pinpoint.

In order to treat AMKL in patients who do not respond to current therapies, researchers need a protein target at which to take aim. In an ongoing collaboration with the Broad Institute, Northwestern University researchers have identified such targets, and findings point to the potential of a drug currently under clinical investigation for the treatment of other forms of cancer. The results of this research appear in the most recent issue of the journal, Cell.

In AMKL, megakaryocytes divide unchecked and fail to produce their characteristic extra genomic copies. Therefore, the research team was interested in finding compounds that could reverse both of these defects. "What's most exciting about this project is that it makes advances both in basic science and in translational medicine. Very little is known about what causes the switch from a progenitor cell to become polyploid and differentiate," said John Crispino, PhD, Robert I. Lurie, MD, and Lora S. Lurie Professor of Medicine at Feinberg, Associate Director for Education and Training at the Lurie Cancer Center, and a senior author of the paper. "We now have a wealth of information about what regulates this pathway. And we've also discovered targets and compounds that could potentially be therapies for this leukemia."

(Last updated on June 25, 2013 )