Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Cancer

Leukemia

2-Hydroxyglutarate (2-HG); isocitrate dehydrogenase 1 (IDH1)

Cell culture studies suggest inhibiting the (R)-enantiomer of 2-HG could help treat leukemia. In two human leukemia cell lines, a membrane-permeable version of (R)-2-HG promoted leukemic transformation, whereas (S)-2-HG and vehicle did not. In the two cell lines, a compound that blocked 2-HG production by a cancer-associated mutant IDH1 reversed the markers of leukemic transformation, whereas vehicle did not. Next steps include evaluating the inhibition of (R)-2-HG signaling in leukemia mouse models and in other types of IDH1-driven cancers.

SciBX 6(8); doi:10.1038/scibx.2013.193
Published online Feb. 28, 2013

Unpatented; licensing status not applicable

Losman, J.-A. et al. Science; published online Feb. 7, 2013;
doi:10.1126/science.1231677
Contact: William Kaelin Jr., Dana-Farber Cancer Institute, Boston, Mass.
e-mail:
william_kaelin@dfci.harvard.edu