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By Sally Robertson, BSc July 11, 2018 Researchers at the Cincinnati Children's Hospital have made important discoveries about the molecular mechanisms of acute myeloid leukemia (AML), a fatal pediatric cancer. The results could lead to the development of new treatments.
MLL-AF9 Leukemia Cells. (Photo credit: Cincinnati Children & # 39; s)
In the Journal of Experimental Medicine scientists refer to their findings as representing "a new opportunity for AML therapy."
Acute myeloid leukemia is a fatal blood cancer badociated with a survival rate of only 20 to 40% and improved therapeutic approaches are urgently needed.
AML occurs when a defect in chromosome 11q23 alters how white blood cells are genetically programmed, causing a fusion of harmful genes. Leighton Grimes (Principal Investigator and Director of Cincinnati Children's Cancer Pathology) and his colleagues have identified a new potential molecular target for treating the disease.
Working with mice and human cells given from patients, the team was able to identify a target molecule found on the surface of human LMA cells.
They found that when they blocked this molecule in combination with existing AML therapies, the cancerous white blood cells were replaced by healthy regenerating cells.
The therapeutic target is a molecule called protein 2 badociated with phase S (Skp2). This protein degrades another molecule – p27Kip1 – which is necessary for the formation of healthy blood cells.
Our work provides a comprehensive mechanistic view of the function of genetic and molecular programs driving this leukemia, and uses these processes to identify therapeutic therapeutics. Leighton Grimes, Principal Investigator
He adds that the approach is not yet ready for clinical application, saying: "We still have other in-depth tests to perform in clinical trials. LMA laboratory animal models before knowing if this approach translate into patient care. "
Despite the additional work that is needed, the results suggest that it may be possible to develop effective treatments for AML, Grimes concludes.
Source:
https: // www. eurekalert.org/pub_releases/2018-07/cchm-taf070918.php
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