Thursday, June 6, 2013
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G-CSF receptor (CSF3R;
mutations to guide diagnosis and treatment of atypical chronic myelogenous
leukemia (aCML) and chronic neutrophilic leukemia (CNL)
Patient sample studies
suggest mutations in CD114 could help diagnose aCML and CNL and guide
treatment decisions for the diseases. Activating mutations in CD114
were found in 16 of 27 patients with CNL or aCML, which is defined as BCR-ABL
tyrosine kinase-negative CML. In patient samples, those
with CD114 truncation mutations were sensitive to Sprycel
whereas those with CD114 membrane proximal mutations were sensitive to
In one patient with CNL who had CD114 membrane proximal mutations,
Jakafi normalized platelet counts and decreased white blood cell and
neutrophil counts. Next steps include clinical trials to evaluate the drugs
in patients who have CD114 mutations.
Corp. and Novartis
AG market Jakafi, a Janus
inhibitor, to treat myeloproliferative disorder.
Squibb Co. and Otsuka
Pharmaceutical Co. Ltd. market Sprycel, a small molecule
inhibitor of BCR-ABL tyrosine kinase and Src,
to treat acute lymphoblastic leukemia (ALL) and CML.
Published online June 6, 2013
Patent application filed;
available for licensing
Maxson, J.E. et al. N.
Engl. J. Med.; published online May 9, 2013;
Contact: Jeffrey W. Tyner, Oregon Health & Science
University, Portland, Ore.
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