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studies suggest neutralizing CALR mutations associated with
myeloproliferative neoplasms could help treat the disease. Mutations in Janus
and other genes cause the majority of myeloproliferative neoplasms, but
genetic causes for about 30%-45% of cases are unknown. In the first study,
exome sequencing identified frameshift mutations in CALR that altered
the C-terminal peptide in all six patients lacking known mutations. The CALR
mutations were confirmed in 67% of patients with thrombocythemia and 88% of
patients with myelofibrosis in a validation cohort. In the second study,
exome sequencing identified CALR mutations in 70%-84% of samples from
151 patients with myeloproliferative neoplasms that lacked JAK-2
mutations but not in patients with other cancers. In mouse B cells,
expression of the most common Calr mutant increased cell proliferation
compared with wild-type Calr expression. Next steps include designing mAbs
targeting the new C-terminal peptide sequence of mutant CALR. Authors from
the first study plan to start a company to develop anti-CALR antibodies.
Published online Jan. 23, 2014
For findings in first
study, patent application filed for diagnostic applications and for mutant
CALR as a therapeutic target; diagnostic applications available for licensing
Patent and licensing status unavailable for findings in second study
Klampfl, T. et al. N.
Eng. J. Med.; published online Dec. 10, 2013;
Contact: Robert Kralovics, Research Center for Molecular
Medicine of the Austrian Academy of Sciences, Vienna, Austria
Nangalia, J. et al. N. Eng. J. Med.; published online Dec. 10, 2013;
Contact: Anthony R. Green, Cambridge Institute for Medical
Research, Cambridge, U.K.
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