title: Frontotemporal degeneration, the next therapeutic frontier: molecules and animal models for frontotemporal degeneration drug development.
abstract: Frontotemporal degeneration (FTD) is a common cause of dementia for which there are currently no approved therapies. Over the past decade, there has been an explosion of knowledge about the biology and clinical features of FTD that has identified a number of promising therapeutic targets as well as animal models in which to develop drugs. The close association of some forms of FTD with neuropathological accumulation of tau protein or increased neuroinflammation due to progranulin protein deficiency suggests that a drug's success in treating FTD may predict efficacy in more common diseases such as Alzheimer's disease. A variety of regulatory incentives, clinical features of FTD such as rapid disease progression, and relatively pure molecular pathology suggest that there are advantages to developing drugs for FTD as compared with other more common neurodegenerative diseases such as Alzheimer's disease. In March 2011, the Frontotemporal Degeneration Treatment Study Group sponsored a conference entitled "FTD, the Next Therapeutic Frontier," which focused on preclinical aspects of FTD drug development. The goal of the meeting was to promote collaborations between academic researchers and biotechnology and pharmaceutical researchers to accelerate the development of new treatments for FTD. Here we report the key findings from the conference, including the rationale for FTD drug development; epidemiological, genetic, and neuropathological features of FTD; FTD animal models and how best to use them; and examples of successful drug development collaborations in other neurodegenerative diseases.
authors.affiliation: Memory and Aging Center, Department of Neurology, University of California, San Francisco, CA, USA. aboxer@memory.ucsf.edu
authors.forename: Adam L
authors.initials: AL
authors.lastname: Boxer
authors.forename: Michael
authors.initials: M
authors.lastname: Gold
authors.forename: Edward
authors.initials: E
authors.lastname: Huey
authors.forename: Fen-Biao
authors.initials: FB
authors.lastname: Gao
authors.forename: Edward A
authors.initials: EA
authors.lastname: Burton
authors.forename: Tiffany
authors.initials: T
authors.lastname: Chow
authors.forename: Aimee
authors.initials: A
authors.lastname: Kao
authors.forename: Blair R
authors.initials: BR
authors.lastname: Leavitt
authors.forename: Bruce
authors.initials: B
authors.lastname: Lamb
authors.forename: Megan
authors.initials: M
authors.lastname: Grether
authors.forename: David
authors.initials: D
authors.lastname: Knopman
authors.forename: Nigel J
authors.initials: NJ
authors.lastname: Cairns
authors.forename: Ian R
authors.initials: IR
authors.lastname: Mackenzie
authors.forename: Laura
authors.initials: L
authors.lastname: Mitic
authors.forename: Erik D
authors.initials: ED
authors.lastname: Roberson
authors.forename: Daniel
authors.initials: D
authors.lastname: Van Kammen
authors.forename: Marc
authors.initials: M
authors.lastname: Cantillon
authors.forename: Kathleen
authors.initials: K
authors.lastname: Zahs
authors.forename: Stephen
authors.initials: S
authors.lastname: Salloway
authors.forename: John
authors.initials: J
authors.lastname: Morris
authors.forename: Gary
authors.initials: G
authors.lastname: Tong
authors.forename: Howard
authors.initials: H
authors.lastname: Feldman
authors.forename: Howard
authors.initials: H
authors.lastname: Fillit
authors.forename: Susan
authors.initials: S
authors.lastname: Dickinson
authors.forename: Zaven
authors.initials: Z
authors.lastname: Khachaturian
authors.forename: Margaret
authors.initials: M
authors.lastname: Sutherland
authors.forename: Robert
authors.initials: R
authors.lastname: Farese
authors.forename: Bruce L
authors.initials: BL
authors.lastname: Miller
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Cummings
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D055808:Drug Discovery; D057174:Frontotemporal Lobar Degeneration / Q000188:drug therapy*; D006801:Humans; D018696:Neuroprotective Agents / Q000627:therapeutic use*
chemical_list: D018696:Neuroprotective Agents