Ramot Yuval, Rosenstock Moti, Klinger Ety, Bursztyn Dizza, Nyska Abraham, Shinar Doron M
Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
Toxicol Pathol. 2012;40(1):40-54. doi: 10.1177/0192623311424169. Epub 2011 Nov 14.
Glatiramer acetate (GA), the active ingredient in Copaxone®, is a complex mixture of polypeptides used for the treatment of relapsing remitting multiple sclerosis. Glatiramoids are related mixtures that may differ in some characteristics of the prototype molecule. Our aim is to describe the long-term toxicity studies with protiramer (TV-5010), a new glatiramoid, in comparison with similar studies conducted with GA. The toxicity of twice-weekly subcutaneous injections of protiramer to Sprague-Dawley rats (twenty-six weeks) and cynomolgus monkeys (fifty-two weeks) was compared with similar studies done with daily subcutaneous injections of GA. Daily treatment with GA was safe and well tolerated, without systemic effects or death. Protiramer administration was not as well tolerated as GA and led to dose- and time-related mortalities, probably mediated through severe injection-site lesions both in rats and in monkeys. Bridging fibrosis in the liver and severe progressive nephropathy were seen in rats. A dose-related increase in eosinophils was observed in monkeys. The protiramer toxicity studies show that minor variations in the manufacturing of glatiramoids may lead to significant toxic effects. It is therefore essential that the safety of any new glatiramoid be studied in long-term preclinical studies before exposing humans.
醋酸格拉替雷(GA)是Copaxone®中的活性成分,是一种用于治疗复发缓解型多发性硬化症的多肽复杂混合物。格拉替雷类似物是相关混合物,在原型分子的某些特性上可能有所不同。我们的目的是描述新型格拉替雷类似物protiramer(TV - 5010)的长期毒性研究,并与用GA进行的类似研究进行比较。将protiramer每周两次皮下注射给Sprague-Dawley大鼠(26周)和食蟹猴(52周)的毒性与每日皮下注射GA的类似研究进行了比较。每日用GA治疗是安全的且耐受性良好,没有全身影响或死亡。Protiramer给药的耐受性不如GA,导致了与剂量和时间相关的死亡,可能是通过大鼠和猴子严重的注射部位损伤介导的。在大鼠中观察到肝脏桥接纤维化和严重的进行性肾病。在猴子中观察到嗜酸性粒细胞与剂量相关的增加。Protiramer毒性研究表明,格拉替雷类似物生产过程中的微小变化可能导致显著的毒性作用。因此,在将任何新型格拉替雷类似物用于人体之前,在长期临床前研究中研究其安全性至关重要。