Associate Professor, Department of Molecular and Experimental Medicine, The Scripps Research Institute, La Jolla, CA 92037, USA.
Expert Rev Neurother. 2010 Mar;10(3):365-75. doi: 10.1586/ern.10.12.
Cladribine, a synthetic deoxyadenosine analog, is an oral immunomodulatory agent that produces targeted, sustained reduction of T and B lymphocytes. This mechanism of action provides the rationale for use in relapsing-remitting multiple sclerosis (MS) in a short-course annual dosing regimen. Based on the results of a pivotal Phase III study, therapy with cladribine tablets has the potential to become a licensed oral disease-modifying medication for relapsing forms of MS. This article will review the key points regarding MS and its pathogenesis, and discuss current unmet treatment needs. In particular the review provides an overview of emerging potential new oral MS therapies with a focus on the mechanism of action, chemistry, pharmacokinetics, pharmacodynamics, clinical efficacy and safety of cladribine tablets. Assessments and conclusions will include a speculative 5-year outlook.
克拉屈滨是一种合成的脱氧腺苷类似物，属于免疫调节药物，能够有针对性地、持续地减少 T 和 B 淋巴细胞。这种作用机制为其在复发缓解型多发性硬化症（MS）的短期年度治疗方案中提供了应用依据。基于一项关键的 III 期研究结果，克拉屈滨片剂疗法有可能成为一种获批的用于治疗复发型 MS 的口服疾病修正药物。本文将回顾 MS 及其发病机制的要点，并讨论当前未满足的治疗需求。特别地，该综述提供了对新兴潜在口服 MS 治疗方法的概述，重点介绍了克拉屈滨片剂的作用机制、化学、药代动力学、药效学、临床疗效和安全性。评估和结论将包括对未来 5 年的推测。
