Selective Janus associated kinase 1 inhibition as a therapeutic target in myelofibrosis

References

• Kralovics R, Passamonti F, Buser AS, et al. A gain-of-function mutation of JAK2 in myeloproliferative disorders. N Engl J Med 2005;352:1779–1790.
   PubMed Web of Science ®Google Scholar
• Pikman Y, Lee BH, Mercher T, et al. MPLW515L is a novel somatic activating mutation in myelofibrosis with myeloid metaplasia. PLoS Med 2006;3:e270.
   PubMed Web of Science ®Google Scholar
• Oh ST, Simonds EF, Jones C, et al. Novel mutations in the inhibitory adaptor protein LNK drive JAK-STAT signaling in patients with myeloproliferative neoplasms. Blood 2010;116:988–992.
   PubMed Web of Science ®Google Scholar
• Grand FH, Hidalgo-Curtis CE, Ernst T, et al. Frequent CBL mutations associated with 11q acquired uniparental disomy in myeloproliferative neoplasms. Blood 2009;113:6182–6192.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Gotlib J, et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med 2012;366:799–807.
   PubMed Web of Science ®Google Scholar
• Harrison C, Kiladjian JJ, Al-Ali HK, et al. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med 2012;366:787–798.
   PubMed Web of Science ®Google Scholar
• Kremyanskaya M, Mascarenhas J, Rampal R, et al. Development of extramedullary sites of leukaemia during ruxolitinib therapy for myelofibrosis. Br J Haematol 2014;167:144–6.
   Google Scholar
• Gotlib J. JAK inhibition in the myeloproliferative neoplasms: lessons learned from the bench and bedside. Hematology Am Soc Hematol Educ Prog 2013:529–537.
   Google Scholar
• Kremer JM, Bloom BJ, Breedveld FC, et al. The safety and efficacy of a JAK inhibitor in patients with active rheumatoid arthritis: results of a double-blind, placebo-controlled phase IIa trial of three dosage levels of CP-690,550 versus placebo. Arthritis Rheum 2009;60:1895–1905.
   PubMed Web of Science ®Google Scholar
• Shuai K, Liu B. Regulation of JAK-STAT signalling in the immune system. Nat Rev Immunol 2003;3:900–911.
   PubMed Web of Science ®Google Scholar
• Baker SJ, Rane SG, Reddy EP. Hematopoietic cytokine receptor signaling. Oncogene 2007;26:6724–6737.
   PubMed Web of Science ®Google Scholar
• Yamaoka K, Saharinen P, Pesu M, et al. The Janus kinases (Jaks). Genome Biol 2004;5:253.
   PubMed Web of Science ®Google Scholar
• Staerk J, Kallin A, Demoulin JB, et al. JAK1 and Tyk2 activation by the homologous polycythemia vera JAK2 V617F mutation: cross-talk with IGF1 receptor. J Biol Chem 2005;280:41893–41899.
   PubMed Web of Science ®Google Scholar
• Watowich SS, Wu H, Socolovsky M, et al. Cytokine receptor signal transduction and the control of hematopoietic cell development. Annu Rev Cell Dev Biol 1996;12:91–128.
   PubMed Web of Science ®Google Scholar
• Rochman Y, Spolski R, Leonard WJ. New insights into the regulation of T cells by gamma© family cytokines. Nat Rev Immunol 2009;9:480–490.
   PubMed Web of Science ®Google Scholar
• Murray PJ. The JAK-STAT signaling pathway: input and output integration. J Immunol 2007;178:2623–2629.
   PubMed Web of Science ®Google Scholar
• Haan C, Rolvering C, Raulf F, et al. Jak1 has a dominant role over Jak3 in signal transduction through gammac-containing cytokine receptors. Chem Biol 2011;18:314–323.
   PubMed Web of Science ®Google Scholar
• Rodig SJ, Meraz MA, White JM, et al. Disruption of the Jak1 gene demonstrates obligatory and nonredundant roles of the Jaks in cytokine-induced biologic responses. Cell 1998;93:373–383.
   PubMed Web of Science ®Google Scholar
• Van Rompaey L, Galien R, van der Aar EM, et al. Preclinical characterization of GLPG0634, a selective inhibitor of JAK1, for the treatment of inflammatory diseases. J Immunol 2013;191:3568–3577.
   PubMed Web of Science ®Google Scholar
• Newcomb DC, Boswell MG, Zhou W, et al. Human TH17 cells express a functional IL-13 receptor and IL-13 attenuates IL-17A production. J Allergy Clin Immunol 2011;127:1006–1013 e1–e4.
   PubMed Web of Science ®Google Scholar
• Vanhouttte F, Mazur M, Van der Aa A, et al. Selective JAK1 inhibition in the treatment of rheumatoid arthritis: proof of concept with GLPG0634. Arthritis Rheum 2012;64(Suppl. 10): Abstract 2489.
   Google Scholar
• Namour F, Galien R, Gheyle R, et al. Once daily high dose regimens of GLPG0634 in healthy volunteers are safe and provide continuous inhibition of JAK1 but not JAK2. Arthritis Rheum. 2012;64(Suppl. 10): Abstract 1331.
   Google Scholar
• Verstovsek S, Kantarjian H, Mesa RA, et al. Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med 2010;363:1117–1127.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Passamonti F, Rambaldi A, et al. A phase 2 study of ruxolitinib, an oral JAK1 and JAK2 Inhibitor, in patients with advanced polycythemia vera who are refractory or intolerant to hydroxyurea. Cancer 2014;120:513–520.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa R, Salama ME, et al. Phase I study Of LY2784544, a JAK2 selective inhibitor, in patients with myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET). Blood 2013;122(Suppl. 1): Abstract 665.
   Google Scholar
• Komrokji RS, Wadleigh M, Seymour JF, et al. Results of a phase 2 study of pacritinib (SB1518), a novel oral JAK2 inhibitor, in patients with primary, post-polycythemia vera, and post-essential thrombocythemia myelofibrosis. Blood 2011;118(Suppl. 1): Abstract 282.
   PubMed Web of Science ®Google Scholar
• Ma L, Clayton JR, Walgren RA, et al. Discovery and characterization of LY2784544, a small-molecule tyrosine kinase inhibitor of JAK2V617F. Blood Cancer J 2013;3:e109.
   PubMed Web of Science ®Google Scholar
• Quintas-Cardama A, Vaddi K, Liu P, et al. Preclinical characterization of the selective JAK1/2 inhibitor INCB018424: therapeutic implications for the treatment of myeloproliferative neoplasms. Blood 2010;115:3109–3117.
   PubMed Web of Science ®Google Scholar
• Bissonnette R, Menter A, Fidelus-Gort R, et al. INCB018424 topical JAK1/JAK2 inhibitor: a double-blind, placebo-controlled study exploring the safety, tolerability, and efficacy of a 28-day course of escalating doses of an oral JAK 1 inhibitor (INCB039110) in subjects with stable, chronic plaque psoriasis. Presented at the European Academy of Dermatology and Venereology, Istanbul, Turkey, 3 October 2013.
   Google Scholar
• Mascarenhas J, Talpaz M, Gupta V, et al. An open-label, phase II study of the JAK1 inhibitor INCB039110 in patients with myelofibrosis. Blood 2013;122(Suppl. 1): Abstract 663.
   Google Scholar
• Mascarenhas JO, Orazi A, Bhalla KN, et al. Advances in myelofibrosis: a clinical case approach. Haematologica 2013;98: 1499–1509.
   PubMed Web of Science ®Google Scholar
• Mullighan CG. The molecular genetic makeup of acute lymphoblastic leukemia. Hematology Am Soc Hematol Educ Program 2012;2012:389–96.
   PubMed Web of Science ®Google Scholar
• Zenatti PP, Ribeiro D, Li W, et al. Oncogenic IL7R gain-of-function mutations in childhood T-cell acute lymphoblastic leukemia. Nat Genet 2011;43:932–939.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Vaidya R, Caramazza D, et al. Circulating interleukin (IL)-8, IL-2R, IL-12, and IL-15 levels are independently prognostic in primary myelofibrosis: a comprehensive cytokine profiling study. J Clin Oncol 2011;29:1356–1363.
   PubMed Web of Science ®Google Scholar
• Ghoreschi K, Laurence A, O’Shea JJ. Janus kinases in immune cell signaling. Immunol Rev 2009;228:273–287.
   PubMed Web of Science ®Google Scholar
• Hart S, Goh KC, Novotny-Diermayr V, et al. SB1518, a novel macrocyclic pyrimidine-based JAK2 inhibitor for the treatment of myeloid and lymphoid malignancies. Leukemia 2011;25:1751–1759.
   PubMed Web of Science ®Google Scholar
• Tyner JW, Bumm TG, Deininger J, et al. CYT387, a novel JAK2 inhibitor, induces hematologic responses and normalizes inflammatory cytokines in murine myeloproliferative neoplasms. Blood 2010;115:5232–5240.
   PubMed Web of Science ®Google Scholar