A(nother) RAF mutation in LCH.
In this issue of Blood, Nelson et al describe a novel somatic ARAF mutation in a child with Langerhans cell histiocytosis (LCH) and demonstrate that the encoded protein has strong gain-of-function properties. Importantly, this mutant A-Raf molecule is sensitive to inhibition by vemurafenib, a potent and selective Raf kinase inhibitor that is Food and Drug Administration (FDA)-approved for the treatment of advanced melanoma. This work thus identifies a new driver mutation in LCH that is potentially actionable in the clinic.
UNIVERSITY OF CALIFORNIA, SAN FRANCISCO.
Kevin
K
Shannon
UNIVERSITY OF CALIFORNIA, SAN FRANCISCO.
Michelle
M
Hermiston
D000818:Animals; D006646:Histiocytosis, Langerhans-Cell / Q000201:enzymology* / Q000235:genetics*; D006801:Humans; D009154:Mutation; D048491:Proto-Oncogene Proteins A-raf / Q000235:genetics*
D048491:Proto-Oncogene Proteins A-raf