FTD and ALS-translating mouse studies into clinical trials

Lars M. Ittner*, Glenda M. Halliday, Jillian J. Kril, Jürgen Götz, John R. Hodges, Matthew C. Kiernan

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

58 Citations (Scopus)


Frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) are related neurodegenerative disorders, which are characterized by a rapid decline in cognitive and motor functions, and short survival. Although the clinical and neuropathological characterization of these diseases has progressed-in part-through animal studies of pathogenetic mechanisms, the translation of findings from rodent models to clinical practice has generally not been successful. This article discusses the gap between preclinical animal studies in mice and clinical trials in patients with FTD or ALS. We outline how to better design preclinical studies, and present strategies to improve mouse models to overcome the translational shortfall. This new approach could help identify drugs that are more likely to achieve a therapeutic benefit for patients.

Original languageEnglish
Pages (from-to)360-366
Number of pages7
JournalNature Reviews Neurology
Issue number6
Publication statusPublished - 1 Jan 2015
Externally publishedYes


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