WASHINGTON, D.C.: Earlier this month in Washington, D.C., 95 scientists from 23 companies, 19 academic institutions and the U.S. Food and Drug and European Medicines Agencies met with funders, advocates, patients, and caregivers for some frank conversations about frontotemporal dementia. This collection of disorders starts in late mid-life with personality changes, language, or movement problems, and invariably leads to dementia and death. No drugs are approved for these diseases. The buzz at this unusual meeting was about learning from the mistakes of drug development in Alzheimer’s disease, and getting a collective act together while the FTD field is still young.
On what did the group agree? Basic science and longitudinal human studies are advancing at record speed, but to launch rigorous drug trials the field urgently needs a toolbox of biomarkers. Why? Biomarkers enable them to subtype FTD disorders and to measure that a candidate drug has reached and hit its intended target. For their part, the regulators called for creative science that tries to couple a drug-induced change in the biomarker to meaningful outcomes. At the same time, the regulators assured the scientists that they take FTD seriously and that no disease is too rare for them to approve drugs for it…