Premium
The advantages of frontotemporal degeneration drug development (part 2 of frontotemporal degeneration: The next therapeutic frontier)
Author(s) -
Boxer Adam L.,
Gold Michael,
Huey Edward,
Hu William T.,
Rosen Howard,
Kramer Joel,
Gao FenBiao,
Burton Edward A.,
Chow Tiffany,
Kao Aimee,
Leavitt Blair R.,
Lamb Bruce,
Grether Megan,
Knopman David,
Cairns Nigel J.,
Mackenzie Ian R.,
Mitic Laura,
Roberson Erik D.,
Van Kammen Daniel,
Cantillon Marc,
Zahs Kathleen,
Jackson George,
Salloway Stephen,
Morris John,
Tong Gary,
Feldman Howard,
Fillit Howard,
Dickinson Susan,
Khachaturian Zaven S.,
Sutherland Margaret,
Abushakra Susan,
Lewcock Joseph,
Farese Robert,
Kenet Robert O.,
LaFerla Frank,
Perrin Steve,
Whitaker Steve,
Honig Lawrence,
Mesulam Marsel M.,
Boeve Brad,
Grossman Murray,
Miller Bruce L.,
Cummings Jeffrey L.
Publication year - 2013
Publication title -
alzheimer's and dementia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 6.713
H-Index - 118
eISSN - 1552-5279
pISSN - 1552-5260
DOI - 10.1016/j.jalz.2012.03.003
Subject(s) - frontotemporal dementia , neuropsychology , drug development , psychology , disease , frontotemporal lobar degeneration , neuroscience , neuroimaging , medicine , drug , psychiatry , pathology , dementia , cognition
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.