Ransohoff Richard M
Neuroinflammation Research Center, Department of Neurosciences, The Lerner Research Institute, The Cleveland Clinic Foundation, United States.
J Neurol Sci. 2007 Aug 15;259(1-2):50-2. doi: 10.1016/j.jns.2006.04.011. Epub 2007 May 22.
The novel multiple sclerosis (MS) therapeutic natalizumab has taken neurologists and their MS patients on a roller-coaster ride: initial encouraging efficacy data led to expedited release in the United States, followed by suspension of dosing with the unexpected occurrence of progressive multifocal leukoencephalopathy (PML) in three clinical trial participants. The drug was re-released in 2006, in a restricted distribution format. Aside from PML, natalizumab treatment was not associated with opportunistic infections, suggesting the possibility that PML in these individuals was mechanism-based, and was not a consequence of generalized immunosuppression. This commentary proposes a hypothesis to account for PML in natalizumab-treated patients.
新型多发性硬化症(MS)治疗药物那他珠单抗让神经科医生及其MS患者经历了一番过山车般的起伏:最初令人鼓舞的疗效数据促使其在美国加速获批上市,随后却因三名临床试验参与者意外出现进行性多灶性白质脑病(PML)而暂停给药。该药物于2006年以受限的分发形式重新上市。除了PML,那他珠单抗治疗与机会性感染无关,这表明这些个体发生的PML可能是基于某种机制,而非全身性免疫抑制的结果。本评论提出了一个假说来解释那他珠单抗治疗患者中出现PML的原因。