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Myeloproliferative disorder

Calreticulin (CALR)

Genetic studies suggest neutralizing CALR mutations associated with myeloproliferative neoplasms could help treat the disease. Mutations in Janus kinase-2 (JAK-2) 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.

SciBX 7(3); doi:10.1038/scibx.2014.89
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.