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.
The Establishing Therapeutic Efficacy in Familial Frontotemporal Degeneration (FTD) meeting will bring together an international group of investigators to begin planning for therapeutic trials in familial FTD. The meeting will be focused on creating an international clinical trials network and developing consensus criteria for how trials will be conducted and evaluated.