The advantages of frontotemporal degeneration drug development (part 2 of frontotemporal degeneration: The next therapeutic frontier)

Adam L. Boxer, Michael Gold, Edward Huey, William T. Hu, Howard Rosen, Joel Kramer, Fen Biao Gao, Edward A. Burton, Tiffany Chow, Aimee Kao, Blair R. Leavitt, Bruce Lamb, Megan Grether, David Knopman, Nigel J. Cairns, Ian R. Mackenzie, Laura Mitic, Erik D. Roberson, Daniel Van Kammen, Marc CantillonKathleen Zahs, George Jackson, Stephen Salloway, John Morris, Gary Tong, Howard Feldman, Howard Fillit, Susan Dickinson, Zaven S. Khachaturian, Margaret Sutherland, Susan Abushakra, Joseph Lewcock, Robert Farese, Robert O. Kenet, Frank Laferla, Steve Perrin, Steve Whitaker, Lawrence Honig, Marsel M. Mesulam, Brad Boeve, Murray Grossman, Bruce L. Miller, Jeffrey L. Cummings

Research output: Contribution to journalReview articlepeer-review

43 Scopus citations


Frontotemporal degeneration (FTD) encompasses a spectrum of related neurodegenerative disorders with behavioral, language, and motor phenotypes for which there are currently no effective therapies. This is the second of two articles that summarize the presentations and discussions that occurred at two symposia in 2011 sponsored by the Frontotemporal Degeneration Treatment Study Group, a collaborative group of academic and industry researchers that is devoted to developing treatments for FTD. This article discusses the current status of FTD clinical research that is relevant to the conduct of clinical trials, and why FTD research may be an attractive pathway for developing therapies for neurodegenerative disorders. The clinical and molecular features of FTD, including rapid disease progression and relatively pure molecular pathology, suggest that there are advantages to developing drugs for FTD as compared with other dementias. FTD qualifies as orphan indication, providing additional advantages for drug development. Two recent sets of consensus diagnostic criteria will facilitate the identification of patients with FTD, and a variety of neuropsychological, functional, and behavioral scales have been shown to be sensitive to disease progression. Moreover, quantitative neuroimaging measurements demonstrate progressive brain atrophy in FTD at rates that may surpass Alzheimer's disease. Finally, the similarities between FTD and other neurodegenerative diseases with drug development efforts already underway suggest that FTD researchers will be able to draw on this experience to create a road map for FTD drug development. We conclude that FTD research has reached sufficient maturity to pursue clinical development of specific FTD therapies.

Original languageEnglish (US)
Pages (from-to)189-198
Number of pages10
JournalAlzheimer's and Dementia
Issue number2
StatePublished - Mar 2013


  • AD
  • Alzheimer's disease
  • Biomarker
  • Clinical trial
  • Corticobasal degeneration
  • Drug development
  • FTD
  • Frontotemporal degeneration
  • Progressive supranuclear palsy
  • Treatment


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