Earlier this month in Washington, D.C., 95 scientists from 23 companies, 19 academic institutions and two regulatory agencies met with funders, advocates and patients and caregivers. The buzz was all about learning from the mistakes and setbacks 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 apace, but what the fields needs most urgently now to launch more and good trials is a toolbox of biomarkers to subtype FTD disorders and measure target engagement. For their part, the regulators want creative, rigorous science that tries to couple biomarker change to meaningful outcomes, but assured the scientists that no disease is too rare for them to be keenly interested and approve drugs for it. Read Gabrielle Strobel’s series.
At a workshop of the Frontotemporal Dementia Treatment Study Group, advocates and regulatory scientists urged leaders from industry and academia to forge a collaborative approach while the field is still young.
Hoping for better luck in clinical trials than their Alzheimer’s colleagues had in the past decade, FTD researchers are now chasing biomarkers. It’s slim pickings so far, but neurofilament, tau PET, and MRI are showing promise.
Citing “fantastic opportunity,” FDA and EMA call for rigorous science. Agency scientists tell FTD Treatment Study Group: Explore individualized outcomes, and connect biomarkers to meaningful improvement.