Editors' ChoiceCancer

Hedgehog to the rescue

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Science Signaling  16 Jun 2015:
Vol. 8, Issue 381, pp. ec161
DOI: 10.1126/scisignal.aac7873

Acute myeloid leukemia is generally difficult to treat, and the presence of internal tandem duplication in a gene called FLT3 (FLT3-ITD) is associated with a particularly poor prognosis. Lim et al. discovered that patients with FLT3-ITD leukemia also have increased activity of the Hedgehog signaling pathway. Experiments in mouse models of FLT3-ITD confirmed the functional role of Hedgehog in the development of leukemia and showed that combined treatment targeting FLT3 and Hedgehog is effective as a therapeutic strategy in this setting.

Y. Lim, L. Gondek, L. Li, Q. Wang, H. Ma, E. Chang, D. L. Huso, S. Foerster, L. Marchionni, K. McGovern, D. N. Watkins, C. D. Peacock, M. Levis, B. D. Smith, A. A. Merchant, D. Small, W. Matsui, Integration of Hedgehog and mutant FLT3 signaling in myeloid leukemia. Sci. Transl. Med. 7, 291ra96 (2015). [Abstract]