Center for Neurological and Psychiatric Research and Drug Discovery, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Shanghai, 201203, China.
University of Chinese Academy of Sciences, Beijing, 100049, China.
Neurosci Bull. 2024 May;40(5):594-608. doi: 10.1007/s12264-023-01138-2. Epub 2023 Oct 28.
Epilepsy is a common neurological disorder that is primarily treated with antiseizure medications (ASMs). Although dozens of ASMs are available in the clinic, approximately 30% of epileptic patients have medically refractory seizures; other limitations in most traditional ASMs include poor tolerability and drug-drug interactions. Therefore, there is an urgent need to develop alternative ASMs. Levetiracetam (LEV) is a first-line ASM that is well tolerated, has promising efficacy, and has little drug-drug interaction. Although it is widely accepted that LEV acts through a unique therapeutic target synaptic vesicle protein (SV) 2A, the molecular basis of its action remains unknown. Even so, the next-generation SV2A ligands against epilepsy based on the structure of LEV have achieved clinical success. This review highlights the research and development (R&D) process of LEV and its analogs, brivaracetam and padsevonil, to provide ideas and experience for the R&D of novel ASMs.
癫痫是一种常见的神经系统疾病,主要通过抗癫痫药物(ASM)治疗。尽管临床上有数十种 ASM,但约 30%的癫痫患者存在药物难治性癫痫发作;大多数传统 ASM 的其他局限性包括耐受性差和药物相互作用。因此,迫切需要开发替代 ASM。左乙拉西坦(LEV)是一种一线 ASM,具有良好的耐受性、有前景的疗效,且药物相互作用较少。尽管人们普遍认为 LEV 通过独特的治疗靶点突触囊泡蛋白(SV)2A 发挥作用,但它的作用机制仍不清楚。即便如此,基于 LEV 结构的新一代针对癫痫的 SV2A 配体已取得临床成功。本综述重点介绍了 LEV 及其类似物、布瓦西坦和帕多伐尼的研发(R&D)过程,为新型 ASM 的研发提供了思路和经验。