Ensuring access to Tau-Targeting therapies for MAPT frontotemporal dementia: patient community priorities for regulators and industry

Abstract

MAPT Frontotemporal Dementia (FTD) is a rare and devastating neurodegenerative disease caused by genetic mutations leading to tau aggregation. This document outlines seven key requests that Cure MAPT FTD, a nonprofit patient advocacy organization, made on May 23, 2025 in a Patient Listening Session to the U.S. Food and Drug Administration (FDA) regarding clinical trial design and the approval of tau-targeting therapies for MAPT FTD patients. The recommendations were informed by a community survey of 44 patients, asymptomatic mutation carriers, and care partners spanning 30 U.S. states, the United Kingdom, and Canada, and reflect lived experience translated into actionable regulatory and trial-design priorities. As global advocates, we emphasize the need for expanding clinical trial eligibility, incorporating care partners in trial processes, and considering natural history data for all rare genetic forms of dementia and other rare diseases with small patient populations. These recommendations aim to accelerate access to therapies for MAPT FTD patients and for asymptomatic mutation carriers who will inevitably face this disease in the future, providing hope for families affected by this condition.