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Promise and Progress - New Leukemia Findings

Reprogramming Cancer Cells - The Story of Epigenetics
Issue No. 1

New Leukemia Findings

Date: July 16, 2014


Blood, March 2014

Proceedings of the National Academy of Science, November 2013

A new genetically engineered mouse model that mimics a common form of leukemia may help researchers better understand and treat resistant forms of the disease.  The mice were bred with mutation of the FLT3 gene, an alteration common in adult and pediatric acute myeloid leukemia.  The mouse model will allow researchers, led by leading FLT3 expert Donald Small, M.D., the Kyle Haydock Professor of Oncology and Director of Pediatric Oncology, to explore the full impact of FLT3 mutations, which occur in various regions of the gene. Dr. Small led the team of researchers that cloned the FLT3 gene and linked it to leukemia two decades ago.

In their ongoing research, Dr. Small and team revealed that the region where the FLT3 gene is mutated significantly impacts the aggressiveness of the disease.  He believes the mice provide a new platform for dissecting the molecular mechanisms resulting in poor prognosis in AML and for screening drugs that may work against them.

He and his team used the model in a recent study to explore a promising new drug that blocks FLT3.  In preliminary laboratory studies, he compared the new drug to other FLT3 inhibitors. The new agent appeared to be more active against all types of FLT3 activating mutations and resistance mutations and also blocked downstream signaling in the FLT3 pathway.

The “Blood” research was funded by Alex’s Lemonade Stand Foundation for Childhood Cancer, the National Institutes of Health’s National Cancer Institute grants CA 90668, CA 70970, and RR 025005, Giant Food Pediatric Cancer Research Fund, and the Pharmacology Analytical Core of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.

The Proceedings of the National Academy of Science research was funded by National Institutes of Health grants CA 090668 and CA 070970, the Leukemia & Lymphoma Society, and the Giant Food Pediatric Cancer Research Fund.

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