Department of Neurology, Duke University School of Medicine, Durham, NC, 27710, USA.
Department of Neurobiology, Duke University School of Medicine, Durham, NC, 27710, USA.
Neurotherapeutics. 2023 Oct;20(6):1496-1507. doi: 10.1007/s13311-023-01413-0. Epub 2023 Aug 17.
The lack of targeted therapies for traumatic brain injury (TBI) remains a compelling clinical unmet need. Although knowledge of the pathophysiologic cascades involved in TBI has expanded rapidly, the development of novel pharmacological therapies has remained largely stagnant. Difficulties in creating animal models that recapitulate the different facets of clinical TBI pathology and flaws in the design of clinical trials have contributed to the ongoing failures in neuroprotective drug development. Furthermore, multiple pathophysiological mechanisms initiated early after TBI that progress in the subacute and chronic setting may limit the potential of traditional approaches that target a specific cellular pathway for acute therapeutic intervention. We describe a reverse translational approach that focuses on translating endogenous mechanisms known to influence outcomes after TBI to develop druggable targets. In particular, numerous clinical observations have demonstrated an association between apolipoprotein E (apoE) polymorphism and functional recovery after brain injury. ApoE has been shown to mitigate the response to acute brain injury by exerting immunomodulatory properties that reduce secondary tissue injury as well as protecting neurons from excitotoxicity. CN-105 represents an apoE mimetic peptide that can effectively penetrate the CNS compartment and retains the neuroprotective properties of the intact protein.
创伤性脑损伤(TBI)缺乏靶向治疗仍然是一个迫切的临床未满足的需求。尽管人们对 TBI 中涉及的病理生理级联反应的了解迅速增加,但新型药理学治疗方法的发展基本上仍然停滞不前。创建能够重现临床 TBI 病理学不同方面的动物模型的困难以及临床试验设计中的缺陷导致神经保护药物开发的持续失败。此外,TBI 后早期启动的多种病理生理机制在亚急性和慢性期进展,可能限制了针对急性治疗干预特定细胞途径的传统方法的潜力。我们描述了一种反向转化方法,该方法专注于将已知影响 TBI 后结果的内源性机制转化为可成药的靶点。特别是,许多临床观察表明载脂蛋白 E(apoE)多态性与脑损伤后的功能恢复之间存在关联。apoE 通过发挥免疫调节特性来减轻对急性脑损伤的反应,从而减少继发性组织损伤并保护神经元免受兴奋毒性。CN-105 是一种 apoE 模拟肽,能够有效地穿透中枢神经系统(CNS)区室,并保留完整蛋白的神经保护特性。