Dementia Pipeline Update: Phase 2 and 3 Developments in Alzheimer Disease and Lewy Body Dementia

The Cambridgeshire and Peterborough NHS Foundation Trust has launched AD-SMART (Alzheimer Disease Systematic Multi-arm Adaptive Randomised Trial), a UK platform trial designed to accelerate therapeutic discovery in Alzheimer disease. The first trial of its kind to test multiple drugs simultaneously, it initially evaluates 2 repurposed agents, one approved for type 2 diabetes and one for attention-deficit/hyperactivity disorder. Participants will be randomized to either agent or placebo, with investigators assessing effects on cognitive symptoms and quality of life. An international expert panel and individuals with lived experience selected the agents and helped define success measures. Interim reviews will allow agents without benefit to be dropped and replaced, limiting exposure to ineffective treatment and shortening evaluation time. Because the drugs are already approved, successful agents could reach secondary approval faster than novel compounds. Up to 20 NHS sites are expected to open over 12 to 18 months, with a target enrollment of 1200 participants.