PHOENIX: On March 31 and April 1, 95 scientists from 23 companies, 19 academic institutions, as well as public and private funding and related organizations gathered in Washington, D.C., for the Frontotemporal Dementia Study Group’s fourth workshop. While the city’s cherry blossoms bloomed riotously outside, the scientists huddled in a hotel basement room with advocates, family members, and people with FTD. They took stock of how far the field has come in its quest to understand this heterogeneous set of diseases, and what tools they most urgently need to develop medications to treat or prevent them. Importantly, the group got an opportunity to quiz three leading regulatory scientists for advice on how to develop drugs for them (see Part 3 of this series). Disorders across the FTD spectrum encompass behavioural variant FTD, three different forms of primary progressive aphasia, progressive supranuclear palsy, corticobasal degeneration, and FTD with motor neuron disease. Together they afflict 50,000 to 60,000 people and their families in the United States. No approved treatments exist.
Overall, eight hours of meetings imparted four lessons:
- The basic science on FTDs is creating unprecedented momentum.
- The setbacks of Alzheimer’s drug development hold valuable lessons.
- Biomarkers are beginning to emerge, but much more work is needed.
- The Food and Drug Administration and European Medicines Agency urge rigorous science that links biomarker change to meaningful clinical outcomes…