Treatment with intracerebroventricular (ICV)-delivered cerliponase alfa enzyme replacement therapy (ERT) in a Phase 1/2 study of 24 subjects with CLN2 disease resulted in a meaningful preservation of motor and language (ML) function and was well tolerated. Treatment was associated with anti-drug antibody (ADA) production in the cerebrospinal fluid (CSF) of 6/24 (25%) and in the serum of 19/24 (79%) of clinical trial subjects, respectively, over a mean exposure of 96.4 weeks (range 0.1–129 weeks). Neutralizing antibodies (NAb) were not detected in the CSF of any of the subjects. No events of anaphylaxis were reported. Neither the presence of serum ADA nor drug-specific immunoglobulin E was associated with the incidence or severity of hypersensitivity adverse events. Serum and CSF ADA titers did not correlate with change in ML score. Therefore, the development of an ADA response to cerliponase alfa is not predictive of an adverse safety profile or poor treatment outcome.

在一项针对24名患有CLN2疾病的受试者的1/2期研究中，采用脑室内（ICV）传递的神经脂酶阿尔法酶替代疗法（ERT）进行的治疗有效地保留了运动和语言（ML）功能，并且耐受性良好。在平均暴露量下，治疗与脑脊髓液（CSF）中6/24（25％）和19/24（79％）血清中抗药物抗体（ADA）的产生有关96.4周（范围0.1-129周）。在任何受试者的CSF中均未检测到中和抗体（NAb）。没有过敏反应的报道。血清ADA或药物特异性免疫球蛋白E的存在均与超敏反应不良事件的发生或严重程度无关。血清和CSF ADA滴度与ML评分的变化无关。所以，