Establishing Therapeutic Efficacy in Frontotemporal Degeneration

描述(由申请者提供)：本提案请求R13会议拨款，以部分支持定于2013年11月14-16日在加利福尼亚州旧金山四季酒店举行的“建立家族性额颞部变性(FTD)的治疗疗效”会议。本次会议的目的是召集美国和国际领先的家族性FTD研究人员，以确定大规模家族性FTD疗效临床试验的可行性和进行这些试验的基本原则，包括制定试验登记、结果指标和治疗剂选择的共识标准。最近，我们对家族性FTD最常见致病基因(tau、prograin或C9ORF72)的了解取得了进展，导致靶向治疗进入早期临床试验和先导研究。这次会议是必要的，因为预计迫切需要进行大规模的疗效研究，这将需要协调多个国家的数百名个人，可能需要较长的试验期。预计将有大约75名具有不同背景的美国和国际领先临床科学家出席，其中包括女性、少数族裔和初级研究人员，因此来自所有主要FTD治疗中心的代表将出席这一里程碑式的会议。会议将确定世界各地现有患者群体的规模和构成，制定解决试验后勤问题的协议，决定特定试验类型的可行性，确定最佳试验结果衡量标准，并确定对现有治疗剂进行排名的程序。主要调查结果将在会议结束后6-12个月内总结并发表在同行评议的期刊上。

DESCRIPTION (provided by applicant): This proposal requests an R13 conference grant to partially support the "Establishing Therapeutic Efficacy for Familial Frontotemporal Degeneration (FTD)" conference, scheduled for November 14-16, 2013 at the Four Seasons Hotel in San Francisco, CA. The purpose of this meeting is to assemble leading American and international familial FTD researchers to determine the feasibility of large scale familial FTD efficacy clinical trials and basic principles for their conduct, including the development of consensus criteria for trial enrollment, outcome metrics, and therapeutic agent selection. Recent advances in our understanding of the most common disease-causative genes for familial FTD (tau, progranulin, or C9ORF72) have led to targeted therapeutics that are entering early stage clinical trial pilot studies. This meeting is needed in anticipation of an imminent need for large-scale efficacy studies that will require the coordination of hundreds of individuals across multipl countries, potentially for lengthy trial terms. Approximately 75 leading US and international clinical scientists with diverse backgrounds, including women, minorities and junior investigators are expected to attend, thus representatives from all major FTD treatment centers will be present at this landmark meeting. The conference will establish the size and composition of available patient populations world-wide, develop protocols to address logistical issues for trials decide on the feasibility of specific trial types, identify best trial outcome measures and identif processes to rank available therapeutic agents. Key findings will be summarized and published in a peer-reviewed journal within 6-12 months after the meeting.