Rigosertib in myelodysplastic syndromes (MDS)

References

• Ma X, Does M, Raza A, et al. Myelodysplastic syndromes. Cancer. 2007;109:1536–1542. doi:10.1002/cncr.22570.
   PubMed Web of Science ®Google Scholar
• Raza A, Gezer S, Mundle S, et al. Apoptosis in bone marrow biopsy samples involving stromal and hematopoietic cells in 50 patients with myelodysplastic syndromes. Blood. 1995;86(1):268–276.
   PubMed Web of Science ®Google Scholar
• Raza A, Galili N. Myelodysplastic syndromes. Nat Cancer Rev. 2012;12:850–859.
   Web of Science ®Google Scholar
• Greenberg P, Cox C, LeBeau MM, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997;89(6):2079–2088.
   PubMed Web of Science ®Google Scholar
• Greenberg P, Tuechler H, Schanz J, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012;120(12):2454–2465.
   PubMed Web of Science ®Google Scholar
• Raza A, Iverson N, Ali AM. Developments in the treatment of transfusion-dependent anemia in patients with myelodysplastic syndromes: epidemiology, etiology, genetics, and targeted therapies. Adv Genomics Genet. 2014;4:95–106. doi:10.2147/AGG.S54184.
   Google Scholar
• Athuluri-Divakar SK, Vasquez-Del Carpio R, Dutta K, et al. A small molecule RAS-mimetic disrupts RAS association with effector proteins to block signaling. Cell. 2016;165(3):643–655.
   PubMed Web of Science ®Google Scholar
• Padua RA, Carter G, Hughes D, et al. RAS mutations in preleukaemias. Haematol Blood Transfus. 1989;32:250–254.
   PubMedGoogle Scholar
• Makishima H, Cazzolli H, Szpurka H, et al. Mutations of e3 ubiquitin ligase cbl family members constitute a novel common pathogenic lesion in myeloid malignancies. J Clin Oncol. 2009;27(36):6109–6116.
   PubMed Web of Science ®Google Scholar
• Nolte F, Hofmann W-K. Molecular mechanisms involved in the progression of myelodysplastic syndrome. Future Oncol. 2010;6(3):445–455.
   PubMed Web of Science ®Google Scholar
• Bejar R, Stevenson K, Abdel-Wahab O, et al. Clinical effect of point mutations in myelodysplastic syndromes. N Engl J Med. 2011;364(26):2496–2506.
   PubMed Web of Science ®Google Scholar
• Itzykson R, Kosmider O, Fenaux P. Somatic mutations and epigenetic abnormalities in myelodysplastic syndromes. Best Pract Res Clin Haematol. 2013;26(4):355–364.
   PubMed Web of Science ®Google Scholar
• Cazzola M, Della Porta MG, Malcovati L. The genetic basis of myelodysplasia and its clinical relevance. Blood. 2013;122(25):4021–4034.
   PubMed Web of Science ®Google Scholar
• Bejar R, Stevenson KE, Caughey B, et al. Somatic mutations predict poor outcome in patients with myelodysplastic syndrome after hematopoietic stem-cell transplantation. J Clin Oncol. 2014;32(25):2691–2698.
   PubMed Web of Science ®Google Scholar
• Malcovati L, Papaemmanuil E, Ambaglio I, et al. Driver somatic mutations identify distinct disease entities within myeloid neoplasms with myelodysplasia. Blood. 2014;124(9):1513–1521.
   PubMed Web of Science ®Google Scholar
• Papaemmanuil E, Gerstung M, Malcovati L, et al. Clinical and biological implications of driver mutations in myelodysplastic syndromes. Blood. 2013;122(22):3616–3627.
   PubMed Web of Science ®Google Scholar
• Gröschel S, Sanders MA, Hoogenboezem R, et al. Mutational spectrum of myeloid malignancies with inv(3)/t(3;3) reveals a predominant involvement of RAS/RTK signaling pathways. Blood. 2015;125(1):133–139.
   PubMed Web of Science ®Google Scholar
• Xu F, He Q, Li X, et al. Rigosertib as a selective anti-tumor agent can ameliorate multiple dysregulated signaling transduction pathways in high-grade myelodysplastic syndrome. Sci Rep. 2014;4:7310.
   PubMed Web of Science ®Google Scholar
• Levitzki A, Mishani E. Tyrphostins and other tyrosine kinase inhibitors. Annu Rev Biochem. 2006;75:93–109.
   PubMed Web of Science ®Google Scholar
• Reddy MVR, Venkatapuram P, Mallireddigari MR, et al. Discovery of a clinical stage multi-kinase inhibitor sodium (E)-2-{2-methoxy-5-[(2ʹ,4ʹ,6ʹ-trimethoxystyrylsulfonyl)methyl]phenylamino}acetate (ON 01910.Na): synthesis, structure-activity relationship, and biological activity. J Med Chem. 2011;54:6254–6276.
   PubMed Web of Science ®Google Scholar
• Seetharam M, Fan AC, Tran M, et al. Treatment of higher risk myelodysplastic syndrome patients unresponsive to hypomethylating agents with ON 01910.Na. Leuk Res. 2012;36(1):98–103.
   PubMed Web of Science ®Google Scholar
• Safety assessment for IND safety reporting guidance for industry. 2015 Dec [cited 2016 Mar 21]. Available from: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM477584.pdf.
   Google Scholar
• Komrokji RS, Raza A, Lancet JE, et al. Phase I clinical trial of oral rigosertib in patients with myelodysplastic syndromes. Br J Haematol. 2013;162(4):517–524.
   PubMed Web of Science ®Google Scholar
• Raza A, Tycko B, Lee S, et al. Oral rigosertib (ON 01910.Na) treatment produces an encouraging rate of transfusion independence in lower risk myelodysplastic syndromes (MDS) patients; a genomic methylation profile is associated with responses. Blood. 2013;122(21):2745.
   Web of Science ®Google Scholar
• Sekeres MA, Cutler C. How we treat higher-risk myelodysplastic syndromes. Blood. 2014;123(6):829–836.
   PubMed Web of Science ®Google Scholar
• Prébet T, Gore SD, Esterni B, et al. Outcome of high-risk myelodysplastic syndrome after azacitidine treatment failure. J Clin Oncol. 2011;29(24):3322–3327.
   PubMed Web of Science ®Google Scholar
• Garcia-Manero G, Fenaux P, Al-Kali A, et al. Rigosertib versus best supportive care for patients with high-risk myelodysplastic syndromes after failure of hypomethylating drugs (ONTIME): a randomised, controlled, phase 3 trial. Lancet Oncol. 2016;17(4):496–508.
   PubMed Web of Science ®Google Scholar
• Sloand EM, Pfannes L, Chen G, et al. CD34 cells from patients with trisomy 8 myelodysplastic syndrome (MDS) express early apoptotic markers but avoid programmed cell death by up-regulation of antiapoptotic proteins. Blood. 2007;109:2399–2405.
   PubMed Web of Science ®Google Scholar
• Olnes MJ, Shenoy A, Weinstein B, et al. Directed therapy for patients with myelodysplastic syndromes (MDS) by suppression of cyclin D1 with ON 01910.Na. Leuk Res. 2012;36:982–989.
   PubMed Web of Science ®Google Scholar
• Silverman LR, Greenberg P, Raza A, et al. Clinical activity and safety of the dual pathway inhibitor rigosertib for higher risk myelodysplastic syndromes following DNA methyltransferase inhibitor therapy. Hematol Oncol. 2015;33(2):57–66.
   PubMed Web of Science ®Google Scholar