Kassi Anelia A Y, Mahavadi Anil K, Clavijo Angelica, Caliz Daniela, Lee Stephanie W, Ahmed Aminul I, Yokobori Shoji, Hu Zhen, Spurlock Markus S, Wasserman Joseph M, Rivera Karla N, Nodal Samuel, Powell Henry R, Di Long, Torres Rolando, Leung Lai Yee, Rubiano Andres Mariano, Bullock Ross M, Gajavelli Shyam
Department of Neurological Surgery, The Miami Project to Cure Paralysis, University of Miami Miller School of Medicine, Miami, FL, United States.
Neurosurgery Service, INUB-MEDITECH Research Group, El Bosque University, Bogotá, CO, United States.
Front Neurol. 2019 Jan 17;9:1097. doi: 10.3389/fneur.2018.01097. eCollection 2018.
Traumatic brain injury (TBI) is the largest cause of death and disability of persons under 45 years old, worldwide. Independent of the distribution, outcomes such as disability are associated with huge societal costs. The heterogeneity of TBI and its complicated biological response have helped clarify the limitations of current pharmacological approaches to TBI management. Five decades of effort have made some strides in reducing TBI mortality but little progress has been made to mitigate TBI-induced disability. Lessons learned from the failure of numerous randomized clinical trials and the inability to scale up results from single center clinical trials with neuroprotective agents led to the formation of organizations such as the Neurological Emergencies Treatment Trials (NETT) Network, and international collaborative comparative effectiveness research (CER) to re-orient TBI clinical research. With initiatives such as TRACK-TBI, generating rich and comprehensive human datasets with demographic, clinical, genomic, proteomic, imaging, and detailed outcome data across multiple time points has become the focus of the field in the United States (US). In addition, government institutions such as the US Department of Defense are investing in groups such as Operation Brain Trauma Therapy (OBTT), a multicenter, pre-clinical drug-screening consortium to address the barriers in translation. The consensus from such efforts including "The Lancet Neurology Commission" and current literature is that unmitigated cell death processes, incomplete debris clearance, aberrant neurotoxic immune, and glia cell response induce progressive tissue loss and spatiotemporal magnification of primary TBI. Our analysis suggests that the focus of neuroprotection research needs to shift from protecting dying and injured neurons at acute time points to modulating the aberrant glial response in sub-acute and chronic time points. One unexpected agent with neuroprotective properties that shows promise is transplantation of neural stem cells. In this review we present (i) a short survey of TBI epidemiology and summary of current care, (ii) findings of past neuroprotective clinical trials and possible reasons for failure based upon insights from human and preclinical TBI pathophysiology studies, including our group's inflammation-centered approach, (iii) the unmet need of TBI and unproven treatments and lastly, (iv) present evidence to support the rationale for sub-acute neural stem cell therapy to mediate enduring neuroprotection.
创伤性脑损伤(TBI)是全球45岁以下人群死亡和残疾的主要原因。无论分布情况如何,诸如残疾等后果都会带来巨大的社会成本。TBI的异质性及其复杂的生物学反应,已凸显出当前TBI治疗药物方法的局限性。五十年来,在降低TBI死亡率方面取得了一些进展,但在减轻TBI所致残疾方面进展甚微。众多随机临床试验的失败以及无法扩大单中心神经保护剂临床试验结果所带来的教训,促使了诸如神经紧急治疗试验(NETT)网络等组织的形成,以及国际协作比较效果研究(CER)的开展,以重新定位TBI临床研究。通过诸如TRACK-TBI等项目,在美国生成包含人口统计学、临床、基因组、蛋白质组、影像学以及多个时间点详细结局数据的丰富而全面的人类数据集,已成为该领域的重点。此外,美国国防部等政府机构正在向诸如脑创伤治疗行动(OBTT)等组织投资,OBTT是一个多中心临床前药物筛选联盟,旨在解决转化过程中的障碍。包括《柳叶刀神经病学委员会》及当前文献在内的这些努力所达成的共识是,未得到缓解的细胞死亡过程、不完全的碎片清除、异常的神经毒性免疫反应以及胶质细胞反应,会导致原发性TBI的组织损失不断进展以及时空放大。我们的分析表明,神经保护研究的重点需要从在急性时间点保护濒死和受损神经元,转向在亚急性和慢性时间点调节异常的胶质细胞反应。一种具有神经保护特性且颇具前景的意外药物是神经干细胞移植。在本综述中,我们呈现:(i)对TBI流行病学的简要概述以及当前治疗的总结;(ii)过去神经保护临床试验的结果以及基于人类和临床前TBI病理生理学研究见解(包括我们团队以炎症为中心的方法)分析得出的可能失败原因;(iii)TBI未满足的需求以及未经证实的治疗方法;最后,(iv)提供证据支持亚急性神经干细胞治疗介导持久神经保护的理论依据。