FDA Patient Listening Session for MAPT FTD

Cure MAPT FTD: FDA Patient Listening Session

On May 23, 2025, members of the MAPT FTD patient community held a Patient Listening Session with the U.S. Food and Drug Administration (FDA). The MAPT FTD community requested this Patient Listening Session to educate and increase awareness within the Agency about the autosomal dominant MAPT genetic variations of FTD, by both providing a scientific overview and also sharing the lived experience of MAPT FTD patients and caregivers.  Additionally, the organization provided insight into the group’s perspectives on trial involvement, risk willingness, and considerations for clinically meaningful outcomes, while highlighting the challenges inherent in conducting studies using smaller populations in the rare disease space. 

While many MAPT family have actively participated as much as possible in related longitudinal research studies where appropriate, there are no forthcoming clinical trials specifically aimed at treating the MAPT variant.

In their patient testimony and in a formal letter submitted to the FDA, Cure MAPT FTD presented the following key findings and requests of the FDA:‍

1. Expand indication labelling: As applicable therapies are approved for Alzheimer’s Disease and other Tauopathies (e.g. Tau-targeting therapies, gene therapies, or therapies targeting neuroinflammation) allow sponsors to expand indication labelling to encompass MAPT FTD.
2. Use care partners: Encourage sponsors to facilitate clinical trial consent and participation by integrating care partners into the clinical trial process. 
3. Expand tau-targeting trials: Encourage sponsors to expand Tau-targeting clinical trials for Alzheimer’s disease and other Tauopathies to include MAPT FTD patients and asymptomatic mutation carriers. 
4. Reduce placebo: Allow use of natural history data as a comparator in clinical trials to determine treatment efficacy without requiring a placebo group or a large patient population. 
5. Include asymptomatic MAPT carriers: Encourage sponsors to include known asymptomatic MAPT mutation carriers in clinical trials and drug labeling, similar to how individuals with genetic Alzheimer’s Disease are being included.
6. High risk tolerance: MAPT FTD patients and asymptomatic carriers are desperate for a cure and are willing to assume moderate to high levels of risk.
7. High priority symptoms: In absence of a disease-modifying therapy, encourage sponsors to look for opportunities to treat high-priority symptoms.

Get the executive summary