Akebia R&D, Akebia Therapeutics Inc., Cambridge, Massachusetts.
Department of Medicine, VA Pittsburgh Healthcare System/University of Pittsburgh, Pittsburgh, Pennsylvania.
Clin J Am Soc Nephrol. 2018 Jul 6;13(7):1113-1123. doi: 10.2215/CJN.06820617. Epub 2018 Mar 9.
AKI is a complex clinical condition associated with high mortality, morbidity, and health care costs. Despite improvements in methodology and design of clinical trials, and advances in understanding the underlying pathophysiology of rodent AKI, no pharmacologic agent exists for the prevention or treatment of AKI in humans. To address the barriers that affect successful clinical translation of drug targets identified and validated in preclinical animal models of AKI in this patient population, the National Institute of Diabetes and Digestive and Kidney Diseases convened the "AKI Outcomes: Overcoming Barriers in AKI" workshop on February 10-12, 2015. The workshop used a reverse translational medicine approach to identify steps necessary to achieve clinical success. During the workshop, breakout groups were charged first to design feasible, phase 2, proof-of-concept clinical trials for delayed transplant graft function, prevention of AKI (primary prevention), and treatment of AKI (secondary prevention and recovery). Breakout groups then were responsible for identification of preclinical animal models that would replicate the pathophysiology of the phase 2 proof-of-concept patient population, including primary and secondary end points. Breakout groups identified considerable gaps in knowledge regarding human AKI, our understanding of the pathophysiology of AKI in preclinical animal models, and the fidelity of cellular and molecular targets that have been evaluated preclinically to provide information regarding human AKI of various etiologies. The workshop concluded with attendees defining a new path forward to a better understanding of the etiology, pathology, and pathophysiology of human AKI.
急性肾损伤(AKI)是一种复杂的临床病症,与高死亡率、高发病率和高医疗成本相关。尽管在临床试验的方法和设计方面有所改进,对啮齿类动物 AKI 的潜在病理生理学的认识也有所提高,但仍没有药物可用于预防或治疗人类 AKI。为了解决影响将 AKI 临床前动物模型中鉴定和验证的药物靶点成功转化到临床的障碍,美国国立糖尿病、消化和肾脏疾病研究所于 2015 年 2 月 10 日至 12 日召开了“AKI 结局:克服 AKI 障碍”研讨会。该研讨会采用反向转化医学方法,确定实现临床成功所需的步骤。在研讨会上,分组的首要任务是设计可行的、第 2 阶段、概念验证临床试验,以解决移植后移植物功能延迟、预防 AKI(初级预防)和治疗 AKI(二级预防和恢复)。然后,分组负责确定能够复制第 2 阶段概念验证患者人群病理生理学的临床前动物模型,包括主要和次要终点。分组发现,在人类 AKI、临床前动物模型中 AKI 病理生理学的认识以及已在临床前评估的细胞和分子靶点的准确性方面,存在着大量的知识空白,这些靶点为各种病因的人类 AKI 提供了信息。研讨会结束时,与会者确定了一条新的前进道路,以更好地了解人类 AKI 的病因、病理和病理生理学。