Therapy and clinical trials in frontotemporal dementia: past, present, and future

J Neurochem. 2016 Aug;138 Suppl 1(Suppl 1):211-21. doi: 10.1111/jnc.13640. Epub 2016 Jun 15.


Frontotemporal dementia (FTD) is a common form of dementia with heterogeneous clinical presentations and distinct clinical syndromes. This article will review currently available therapies for FTD, its related disorders and their clinical evidence. It will also discuss recent advancements in FTD pathophysiology, treatment development, biomarker advancement and their relation to recently completed or currently ongoing clinical trials as well as future implications. Frontotemporal dementia (FTD) is a type of dementia with distinct clinical syndromes. Current treatments involve off-label use of medications for symptomatic management and cannot modify disease course. Advancements in FTD pathophysiology, genetics, and biomarkers have led to development of small molecules targeting the underlying pathology in hopes of achieving a disease-modifying effect. This article will review current therapies for FTD, discuss advancements in FTD pathophysiology, therapy development, biomarker advancement, their relation to recent clinical trials and future implications. This article is part of the Frontotemporal Dementia special issue.

Keywords: 10th International Conference on Frontotemporal Dementia; C9ORF72; TDP-43; behavioral variant frontotemporal dementia (bvFTD); clinical trials; frontotemporal dementia; primary progressive aphasia (PPA); progressive supranuclear palsy (PSP); tau; treatment.

Publication types

  • Review
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Biomarkers
  • Clinical Trials as Topic
  • Frontotemporal Dementia / diagnosis
  • Frontotemporal Dementia / psychology*
  • Frontotemporal Dementia / therapy*
  • Humans


  • Biomarkers