四硫代钼酸胆碱
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临床资料 | |
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商品名 | Decuprate |
其他名称 | 四硫代钼酸双胆碱 双胆碱四硫代钼酸盐 ATN-224、WTX101 |
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识别信息 | |
CAS号 | 649749-10-0 ![]() |
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化学信息 | |
化学式 | C10H28MoN2O2S4 |
摩尔质量 | 432.542 g/mol |
3D模型(JSmol) | |
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四硫代钼酸胆碱(又称胆碱四硫代钼酸盐,商品名为Decuprate),是胆碱所形成的硫代钼酸盐(TTM, MoS42−)。作为威尔逊氏病的治疗方法,这是一种身体不能调节铜的罕见且潜在的致命疾病。威尔逊病是一种常染色体隐性遗传性疾病,有严重的肝、神经或精神症状的表现。若未经诊断和未经治疗,该疾病可能致命。据估计,全世界每3万人中约有1人患有威尔逊氏病,相当于欧盟约有1万5千人而美国约有1万1千人。[1]
已经在患有各种癌症的患者的临床试验中评估了四硫代钼酸胆碱,[2][3][4]并在美国和欧盟成为了孤儿药(即罕见药),作为威尔逊氏病的潜在治疗方法。[5][6]
四硫代钼酸胆碱是威尔逊治疗学公司的代号为WTX101的威尔逊氏病临床开发中的去铜疗法用药,该公司由HealthCap于2012年成立。
参考文献
- ^ Ala, A; Walker, A. P.; Ashkan, K; Dooley, J. S.; Schilsky, M. L. Wilson's disease. The Lancet. 2007, 369 (9559): 397–408. PMID 17276780. doi:10.1016/S0140-6736(07)60196-2.
- ^ Berenson JR, Boccia RV, Bashey A, Levine AM, Koc ON, Callahan JA, Mazar AP, Reich SD. Phase I Study of the [Cu, Zn] Superoxide Dismutase (SOD1) Inhibitor ATN-224 (Bis-Choline Tetrathiomolybdate) in Patients with Advanced Hematologic Malignancies Presentation at the Amer Soc Hematol 2006 Annual Meeting. Blood. 2006, 108. Abstract 2593.
- ^ Lin, J; Zahurak, M; Beer, T. M.; Ryan, C. J.; Wilding, G; Mathew, P; Morris, M; Callahan, J. A.; Gordon, G; Reich, S. D.; Carducci, M. A.; Antonarakis, E. S. A non-comparative randomized phase II study of 2 doses of ATN-224, a copper/zinc superoxide dismutase inhibitor, in patients with biochemically recurrent hormone-naïve prostate cancer. Urologic Oncology: Seminars and Original Investigations. 2013, 31 (5): 581–8. PMC 3227793
. PMID 21816640. doi:10.1016/j.urolonc.2011.04.009.
- ^ Lowndes, S. A.; Adams, A; Timms, A; Fisher, N; Smythe, J; Watt, S. M.; Joel, S; Donate, F; Hayward, C; Reich, S; Middleton, M; Mazar, A; Harris, A. L. Phase I study of copper-binding agent ATN-224 in patients with advanced solid tumors. Clinical Cancer Research. 2008, 14 (22): 7526–34. PMID 19010871. doi:10.1158/1078-0432.CCR-08-0315.
- ^ Public summary of opinion on orphan designation: Choline tetrathiomolybdate for the treatment of Wilson's disease (页面存档备份,存于互联网档案馆), EMA/COMP/795268/2012, ATN-224, 18 February 2013.
- ^ Orphan Drug Designations and Approvals: choline tetrathiomolybdate (页面存档备份,存于互联网档案馆), U.S. Food and Drug Administration, 25 August 2011