Alopecia Areata Center for Research Translation (AACORT)

总体AACORT摘要 哥伦比亚大学医学中心斑秃研究翻译中心 (CUMC)专注于开发斑秃(AA)新疗法的单一目标。的使命 AACORT将利用我们最近的发现，开始揭开再生障碍性贫血的遗传基础和事实 它的发病机制与先天免疫和获得性免疫的失调有关，并与它们协同作用 翻译研究的发现将导致这种疾病的新疗法，包括改变其用途 现有的药物。在过去的几年里，AACORT团队在认识上取得了革命性的进展 再生障碍性贫血的遗传学基础，以及利用机制驱动的靶向JAK启动一系列临床试验 旨在逆转这种疾病的抑制剂。虽然这类药物对指导 在再障的临床开发工作中，还有更多的工作要做，因为显然许多患者并不 对这些药物有反应。AACORT的目标是在我们已建立的翻译渠道的基础上 将我们的基础和临床前发现应用于临床环境。此AACORT以一个 中央研究项目，专注于斑秃的翻译科学，并处理 疾病的创新翻译方面，包括启动因素，如微生物组；临床前 研究集中在体外和体内的抗原发现和测试新的治疗方法；以及临床 旨在严格评估即将上市的新药的有效性和安全性的相关研究 再生障碍性贫血患者。该研究项目由翻译和临床资源核心(TRAC)支持， 和数据分析和翻译应用(DATA)核心，汇聚了一名优秀的干部 来自整个学院的才华横溢、尽职尽责、科学多样的教职员工。支持AACORT 由高度能干和经验丰富的管理核心提供其至关重要的组织 基础设施。AACORT的目标是跨越基本发现翻译应用的障碍 它们有可能彻底改变对这种迄今难以治愈且令人衰弱的疾病的管理。

Overall AACORT Abstract The Alopecia Areata Center of Research Translation (AACORT) at Columbia University Medical Center (CUMC) is focused on the singular goal of developing new treatments for alopecia areata (AA). The mission of the AACORT is to leverage our recent discoveries beginning to unravel the genetic basis of AA and the fact that its pathogenesis is linked to dysregulation of both innate and acquired immunity, and to synergize these findings into translational studies that will lead to novel therapies for this disease, including the repurposing of existing drugs. In the past few years, the AACORT team has made transformative progress in understanding the genetic basis of AA, as well as initiating a series of clinical trials employing mechanism-driven targeted JAK inhibitors aimed at reversing the disease. While this class of drugs is having a profound impact on guiding clinical development efforts in AA, there is much more work to be done, since clearly many patients do not respond to these drugs. The goal of this AACORT is to build upon our established translational pipeline to leverage our basic and preclinical discoveries into the clinical setting. This AACORT is centered around a central Research Project that focuses on the Translational Science of Alopecia Areata, and deals with innovative translational aspects of the disease, including initiating factors such as the microbiome; preclinical studies focused on antigen discovery and testing new therapeutic approaches in vitro and in vivo; and clinical correlative studies designed to rigorously assess the efficacy and safety of new drugs coming available for patients with AA. The Research Project is supported by the Translational and Clinical Resource Core (TRAC), and the Data Analysis and Translational Applications (DATA) Core, which bring together an outstanding cadre of talented, committed and scientifically diverse faculty from across the institution. The AACORT is supported by a highly capable and experienced Administrative Core that provides its vitally important organizational infrastructure. The goal of the AACORT is to surmount barriers to translational application of basic discoveries that have the potential to revolutionize the management of this heretofore intractable and debilitating disorder.