Picture this: Your husband gets fired from his college professorship. He had written his student's final essays himself and graded his work as theirs; they turned him in. Soon after, he buys a sports car he can ill afford and you beg his neurologists to get the dealer to take it back. Or this: Seized with chest pain, you drop onto the floor and urge your spouse to call 911. Unmoved, he replies, "Oh. What's for dinner?" Such vignettes of executive and emotional dysfunction hint at why frontotemporal degeneration is a crushing disease, particularly for caregivers. They also animated a meeting that the FTD Treatment Study Group (FTSG) convened 4 June in Washington, D.C., where FTD clinician-researchers from North America and Europe met with experts from regulatory agencies, industry, family representatives, and public and private funders.
The questions of the day were: Given the explosive progress science has made in the past six years on the genetics and molecular pathology of this spectrum of dementing disorders, how close is the field today to running therapeutic trials that stand to be successful? If the field is not there yet, what is still missing? And what do scientists still have to do attrract the interest, funds, and expertise of the biotech and pharmaceutical industry?
Hosted jointly by the Association for Frontotemporal Degeneration (AFTD) and the National Institute of Neurologic Disorders and Stroke, the workshop drew 67 scientists from academic, industry, the U.S. Food and Drug Administration (FDA), and public and private funding organizations.