FTD: Treatments for FTD

AD-Related Dementias Focus

Accelerate pre-clinical disease-modifying and symptomatic therapeutic development in FTD.

2029 RFA-NS-22-055: Functional Target Validation for Alzheimer's Disease-Related Dementias (ADRDs) (R61/R33 Clinical Trial Not Allowed) RFA-NS-22-059: Early-Stage Therapy Development for ADRD (R61/R33 Clinical Trial Not Allowed) RFA-NS-23-017: Optimization of Genome Editing Therapeutics for Alzheimer's Disease and Alzheimer's Disease-Related Dementias (AD/ADRD) (U01 - Clinical Trials Not Allowed) RFA-NS-24-009: Optimization of Genome Editing Therapeutics for Alzheimer's Disease-Related Dementias (ADRD) (U01 - Clinical Trials Not Allowed) (Reissue of RFA-NS-23-017) RFA-NS-24-010: Early-Stage Therapy Development for Alzheimer's Disease-Related Dementias (ADRD) (R61/R33 - Clinical Trial Not Allowed) (Reissue of RFA-NS-22-059) RFA-NS-24-037: Optimization of Genome Editing Therapeutics for Alzheimer's Disease-Related Dementias (ADRD) (U01 - Clinical Trials Not Allowed) RFA-NS-25-011: Functional Target Validation for Alzheimer's Disease-Related Dementias (R61/R33 Clinical Trial Not Allowed) 7.F In Progress DN 2022 ADRD Summit: Frontotemporal Dementia, Milestone 6, Priority 2 Drug Repurposing and Combination Therapy Development

  • At least three studies to identify and/or validate drivers of FTD that are potential targets for intervention.
  • At least one new FTD intervention with FDA approval to proceed with clinical trials.

Summary of Key Accomplishments:

NIH is accelerating development of treatments for FTD through targeted initiatives and investigator-initiated research. In 2024-2025, NIH announced funding initiatives to encourage applications for the translation of new genome editing technologies for the development of treatments and to support validation of new therapeutic targets for ADRD, including FTD. In addition, investigators supported through NIH’s Small Business programs are developing new drugs, platforms, and delivery systems for use in the treatment of FTD and other forms of dementia.

The key accomplishments summary is current as of September 2025.