鞘内注射反义寡核苷酸的机制性药代动力学模型。
A mechanistic pharmacokinetic model for intrathecal administration of antisense oligonucleotides.
作者信息
Linninger Andreas A, Barua Dipak, Hang Yaming, Iadevaia Sergio, Vakilynejad Majid
机构信息
University of Illinois at Chicago, Chicago, IL, United States.
Takeda Pharmaceuticals, Cambridge, MA, United States.
出版信息
Front Physiol. 2023 Jun 2;14:1130925. doi: 10.3389/fphys.2023.1130925. eCollection 2023.
Intrathecal administration is an important mode for delivering biological agents targeting central nervous system (CNS) diseases. However, current clinical practices lack a sound theorical basis for a quantitative understanding of the variables and conditions that govern the delivery efficiency and specific tissue targeting especially in the brain. This work presents a distributed mechanistic pharmacokinetic model (DMPK) for predictive analysis of intrathecal drug delivery to CNS. The proposed DMPK model captures the spatiotemporal dispersion of antisense oligonucleotides (ASO) along the neuraxis over clinically relevant time scales of days and weeks as a function of infusion, physiological and molecular properties. We demonstrate its prediction capability using biodistribution data of antisense oligonucleotide (ASO) administration in non-human primates. The results are in close agreement with the observed ASO pharmacokinetics in all key compartments of the central nervous system. The model enables determination of optimal injection parameters such as intrathecal infusion volume and duration for maximum ASO delivery to the brain. Our quantitative model-guided analysis is suitable for identifying optimal parameter settings to target specific brain regions with therapeutic drugs such as ASOs.
鞘内给药是递送针对中枢神经系统(CNS)疾病的生物制剂的重要途径。然而,目前的临床实践缺乏一个完善的理论基础,难以对影响递送效率和特定组织靶向性(尤其是在大脑中的靶向性)的变量和条件进行定量理解。这项工作提出了一种分布式机制药代动力学模型(DMPK),用于鞘内药物向中枢神经系统递送的预测分析。所提出的DMPK模型能够在数天和数周的临床相关时间尺度上,根据输注、生理和分子特性,捕捉反义寡核苷酸(ASO)沿神经轴的时空扩散情况。我们利用非人类灵长类动物中反义寡核苷酸(ASO)给药的生物分布数据展示了其预测能力。结果与在中枢神经系统所有关键区室中观察到的ASO药代动力学密切一致。该模型能够确定最佳注射参数,如鞘内输注体积和持续时间,以实现ASO向大脑的最大递送。我们基于定量模型的分析适用于确定最佳参数设置,以便用ASO等治疗药物靶向特定脑区。