"Establishing Therapeutic Efficacy in Familial FTD" Conference Advances INternational Clinical Trials Network

SAN FRANCISCO, CA - 11/26/13. Conducting clinical trials for a rare disease like FTD will require an extensive international network of collaborative investigators. Prospective participants must be identified, clinical research infrastructures established, and participating physicians must agree upon the study parameters. To begin to build a network and better understand the worldwide distribution of individuals carrying genetic mutations that cause FTD, the Bluefield Project cosponsored a landmark conference entitled, “Establishing Therapeutic Efficacy in Familial Frontotemporal Degeneration” in San Francisco on November 14-16, 2013. Attended by over 140 clinical researchers from more than 20 countries, the conference represented a pivotal event in the effort to find a cure for FTD.

"This was a remarkable meeting of many of the leading clinical research centers studying FTD worldwide. There was a real sense of cooperation and collaboration as we start to translate the discoveries in the laboratories into treatments for this very serious disease," said conference co-organizer and Bluefield Project President Dr. Rodney Pearlman.

The meeting focused on FTD caused by mutations in progranulin, tau and C9ORF72 and was co-organized by Dr. Bruce Miller, Director of UCSF’s Memory and Aging Center, Dr. Adam Boxer, Director of UCSF’s Alzheimer’s and FTD Clinical Trials Programs, and Dr. Howard Rosen, Associate Professor of Neurology at UCSF. Invited speakers described advances in the understanding of the biological functions of proteins that cause FTD when mutated and current efforts to develop targeted therapies. Similarly, speakers detailed efforts to develop precise clinical rating scales to follow disease progression and rigorously determine if a drug changes the disease course. Also of great interest was a valuable discussion of the ethics of genetic testing and the dissemination of genetic data in the context of clinical trials.

Much of the conference focused on cataloging existing patient populations around the world in anticipation of a clinical trial. Meeting participants were asked to complete a general survey about their patient populations ahead of the meeting, and the results were reported in aggregate at the conference. Speakers from the UK, USA, Belgium, France, Germany, Scandinavia, Spain, Italy, Greece, Turkey, Israel, India, Korea, Japan, Australia, the Philippines and Brazil presented information on their patient cohorts. It was estimated that, worldwide, at least 500 families carry mutations in C9ORF72, 320 families in progranulin, and 50 families in tau. Speakers stressed the paramount importance of working closely with patients and their families as potential therapies are considered for study in the clinic.

Conference participants agreed to continue to discuss and formalize clinical trial network efforts, to meet again at the International FTD Meeting in Vancouver in 2014, and to work together to write a manuscript detailing the key outcomes from this conference.

The conference was sponsored by The Bluefield Project to Cure FTD, the Rainwater Charitable Foundation, the National Institute on Aging, the Association for Frontotemporal Degeneration and the Memory and Aging Center at the University of California, San Francisco.