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Potential Strategy for Acute Myeloid Leukemia Therapy Identified

The results from in vitro and in vivo studies suggest that targeting a pathway that is essential for the survival of certain types of acute myeloid leukemia (AML) could offer a new therapeutic strategy. The research, by scientists at the Wellcome Sanger Institute, found that a specific genetic mutation that is linked with poor prognosis in blood cancer is involved in the development of the disease when combined with other mutations in mice and in human cell lines.

Reported in Nature Communications, the findings provide new insights into how the loss-of-function mutation in the CUX1 gene leads to the development and survival of AML. The results suggest that identifying a pathway that is essential for these cancer cells to continue growing could lead to new, targeted therapies for some patients.

Saskia Rudat, PhD, co-first author and postdoctoral fellow at the Wellcome Sanger Institute, said, “By investigating the role of CUX1 further, we now have new insight into how this gene, and the lack of it when mutated, plays a key role in the survival of blood cancer cells. While this mutation doesn’t seem to cause the development of malignant disease on its own, focusing on the pathways involved with CUX1 is a good target for further research.”

Rudat and colleagues described their work in a paper titled, “Cut-like homeobox 1 (CUX1) tumor suppressor gene haploinsufficiency induces apoptosis evasion to sustain myeloid leukemia.”

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