Drug Trials in Frontotemporal Dementia: Can Industry, Academia, Families and Regulators Get on One Page?

Newswise — 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. For a three-part series on all things FTD, see Alzforum.

About UsFounded in 1996, Alzforum is a news and information resource website dedicated to helping researchers accelerate discovery and advance development of diagnostics and treatments for Alzheimer’s disease and related disorders.

Our site expands the traditional mode of scientific communication by reporting the latest scientific findings and industry news with insightful analysis that puts breaking news into context. We advance research by developing open-access databases of curated, highly specific scientific content to visualize and facilitate the exploration of complex data. Alzforum is a platform to disseminate the evolving knowledge around basic, translational, and clinical research in the field of AD.

Alzforum is supported by a team with backgrounds in science, journalism, information technology, design, and data science. Together with a distinguished Scientific Advisory Board, and the active participation of a global network of scientists, we strive to produce unbiased content to a rigorous editorial standard.

Alzforum is operated by the Biomedical Research Forum (BRF) LLC. BRF is a wholly owned subsidiary of FMR LLC. FMR LLC and its affiliates invest broadly in many companies, including life sciences and pharmaceutical companies. Alzforum does not endorse any specific product or scientific approach.