What happened at the Youngtimers Dec 2025 Patient Listening Session with the FDA?

On December 10, 2025, Youngtimers had the opportunity to meet with with the U.S. Food and Drug Administration (FDA) to share lived experiences of the familial Alzheimer’s disease (FAD) patient and caregiver community.

Staff from 20 FDA departments attended, along with nine community advocates, Dr. Randall Bateman of Washington University, and supporters from several other Alzheimer's organizations.

The meeting served as an opportunity to:

1. help the FDA understand what FAD is and how it affects families

2. share community survey data on treatment attitudes

3. showcase why mutation carriers need access to FDA-approved treatments before symptoms begin

Dr. Bateman opened with a science overview. He explained that mutations in PSEN1, PSEN2, and APP will almost certainly cause FAD, that brain changes begin 15 to 20 years before symptoms appear, and that early treatment has been shown to cut the risk of cognitive decline in FAD mutation carriers by about 50%.

Nine community members then spoke, including someone with symptoms of FAD, someone who is at-risk of carrying the mutation, mutation carriers who have not yet developed symptoms, caregivers, and widowed spouses. They described how FAD shapes careers, finances, family planning, and children's lives across multiple generations.

Dr. Lindsay Hohsfield presented findings from a community survey conducted in November 2025. Survey results showed that many are reluctant to get genetic testing in order to join a research trial, especially knowing they might be assigned to a placebo group and receive an inactive treatment rather than the real drug. However, people were more likely to pursue testing if it meant access to an approved treatment. Across the board, community members said the risks of treatment were acceptable given the near-certainty of disease progression without it. This included ARIA, a type of brain swelling or bleeding that can occur as a side effect of some Alzheimer's treatments.

The community's core request to the FDA was straightforward: allow confirmed FAD mutation carriers access to approved treatments based on their genetic status, rather than making them wait for symptoms to appear first.

The FDA's Office of Neuroscience responded positively, saying these perspectives would directly inform their regulatory work.

Want more detail about Youngtimers FDA listening session?
Read the full report (PDF).