IMAGE: These are results of a CRISPR screen for drug targets in leukemia that are both powerful in their impact and as specific possible to subtypes of leukemia. Left to right:… view more
Credit: Vakoc Lab, CSHL
Cold Spring Harbor, NY — Scientists at Cold Spring Harbor Laboratory (CSHL) have discovered a way to rein in an overactive protein that drives some aggressive leukemias. The renegade molecule, MEF2C, belongs to a class of proteins that is notoriously difficult to manipulate with drugs. But the new research suggests an opportunity to develop therapies against it.
MEF2C is a transcription factor–a regulatory protein that helps control the activity of certain genes. Its overactivity is implicated in about 15% of cases of acute myeloid leukemia (AML), a rapidly progressing cancer of the blood and bone marrow that is often fatal.
CSHL Associate Professor Christopher Vakoc, M.D., Ph.D., and colleagues now report that they can stop the growth of MEF2C-driven AML cells by blocking either of two target enzymes, known as LKB1 and salt-inducible kinase. The chemicals that the team used to interfere with the enzymes are small molecules that have the potential to be developed into drugs, Vakoc says.
The discovery is
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