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AD-Related Dementias Focus
Develop an array of FTD biomarkers for diagnosis, prediction, disease monitoring, target engagement, and patient stratification for clinical trials.
2027 PAR-18-661: Pathway and Target Identification for Alzheimer's Disease Related Dementias (ADRDs) RFA-NS-19-014: Center without Walls for PET Ligand Development for Alzheimer's disease related dementias (ADRDs) (U19) RFA-AG-15-010: Interdisciplinary Research to Understand the Vascular Contributions to Alzheimer's Disease (R01) RFA-NS-21-003: Center without Walls for Mechanisms of Neurodegeneration in Frontotemporal Dementia (FTD) (U54 Clinical Trial Not Allowed) 9.Q In Progress Develop FTD biomarkers for diagnosis and disease progression. DN 2016 ADRD Summit: Frontotemporal Lobar Degeneration (FTD) Focus Area 2: Clinical science, Recommendation 2 2022 ADRD Summit: Frontotemporal Lobar Degeneration (FTD), Milestone 2, Priority 2 News: Blood test method may predict Alzheimer’s protein deposits in brain Blood marker shows potential for tracking frontotemporal dementia Workshop: Advancing Digital Endpoints & Digital Health Technologies for Neurological and Neuromuscular Disorders Biomarkers Center without Walls for PET Ligand Development for Alzheimer's disease related dementias (ADRDs) (U19) NINDS BioSEND National Centralized Repository for Alzheimer’s Disease and Related Dementias (NCRAD) CREATE Biorepository ALLFTD Center Without Walls for Imaging Proteinopathies with PET (CW2IP2) Select projects funded in FY21 Mechanisms of Gait and Balance Impairment in Progressive Supranuclear Palsy
- At least two new FTD biomarkers that are validated for clinical trials, including in at least two populations that experience health disparities.
Summary of Key Accomplishments
NIH-funded researchers have recently made dramatic progress in identifying novel biomarkers that could be used to diagnose and monitor patients with frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS; a related condition involving degeneration of motor neurons). Unique biomarkers were found to specifically correspond to the dysfunction of the TDP-43 protein that underlies most ALS and about half of FTD cases. These findings could facilitate rapid diagnosis and enhance the power of clinical trials for new therapeutics.
Several advances have the potential to expand access to diagnosis, disease monitoring, and clinical trial participation in underserved communities. Relatively simple and inexpensive blood tests may be able to distinguish FTD from other dementias (such as Alzheimer's), which will be critical for future targeted clinical trials. Additionally, supplemental studies to the large ALLFTD cohort include the use of mobile apps as well as other non-invasive measurements and trackers that have the promise of monitoring the progression of FTD in a highly sensitive manner directly from an individual's home, reducing or eliminating the need for frequent travel to a clinical research site.
Further work on FTD biomarker development continues through two large consortia. Researchers recently found that neurofilament light chain (NfL), a protein released when nerve cells are damaged, has potential to be a biomarker for FTD. A public-private biomarkers consortium led by the Foundation for NIH (FNIH) is currently evaluating NfL as a biomarker for neurodegeneration in familial forms of FTD. Additionally, an NIH-led proteinopathy consortium has defined the atomic structure of various forms of proteins like tau that are aggregated in FTD and identifying promising targets for PET imaging development.
The key accomplishments summary is current as of July 2024.