The advantages of frontotemporal degeneration drug development (part 2 of frontotemporal degeneration: the next therapeutic frontier).
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.
Memory and Aging Center, Department of Neurology, University of California, San Francisco, CA, USA. aboxer@memory.ucsf.edu
Adam L
AL
Boxer
Michael
M
Gold
Edward
E
Huey
William T
WT
Hu
Howard
H
Rosen
Joel
J
Kramer
Fen-Biao
FB
Gao
Edward A
EA
Burton
Tiffany
T
Chow
Aimee
A
Kao
Blair R
BR
Leavitt
Bruce
B
Lamb
Megan
M
Grether
David
D
Knopman
Nigel J
NJ
Cairns
Ian R
IR
Mackenzie
Laura
L
Mitic
Erik D
ED
Roberson
Daniel
D
Van Kammen
Marc
M
Cantillon
Kathleen
K
Zahs
George
G
Jackson
Stephen
S
Salloway
John
J
Morris
Gary
G
Tong
Howard
H
Feldman
Howard
H
Fillit
Susan
S
Dickinson
Zaven S
ZS
Khachaturian
Margaret
M
Sutherland
Susan
S
Abushakra
Joseph
J
Lewcock
Robert
R
Farese
Robert O
RO
Kenet
Frank
F
Laferla
Steve
S
Perrin
Steve
S
Whitaker
Lawrence
L
Honig
Marsel M
MM
Mesulam
Brad
B
Boeve
Murray
M
Grossman
Bruce L
BL
Miller
Jeffrey L
JL
Cummings
D000818:Animals; D004195:Disease Models, Animal; D055808:Drug Discovery; D057174:Frontotemporal Lobar Degeneration / Q000188:drug therapy*; D006801:Humans; D018696:Neuroprotective Agents / Q000627:therapeutic use*
D018696:Neuroprotective Agents