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Expert Opin Drug Discov. 2017 Jul;12(7):659-671. doi: 10.1080/17460441.2017.1329294. Epub 2017 Jun 2.

New routes in frontotemporal dementia drug discovery.

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a Biotechnology Development Group , International Centre for Genetic Engineering and Biotechnology , Trieste , Italy.
b Centre for Neurodegenerative Disorders - Neurology Unit , University of Brescia , Brescia , Italy.


Research into the pathogenic mechanisms behind frontotemporal dementia (FTD) has yielded several new targets for therapeutic intervention; such targets include specific new pathways uncovered by mutations as well as targets involving the modulation, formation and degradation of protein aggregates. Areas covered: Herein, the authors outline the principal molecular causes underlying FTD to date and the research that has been performed in these areas with respect to an eventual corrective strategy. Expert opinion: While it is worthwhile targeting pathways affected by specific mutations with a causative loss of function linked to FTD, research still has to contend with issues including the remaining presence of protein aggregates or that treatments are rarely universally applicable. Aiming to recover function in a downstream target caused by the protein aggregates will likely be insufficient due to the large cascade of events affected. It is our belief that the clearance of these aggregates and the inhibition of protein misfolding are more appropriate and direct routes to an eventual therapy.


Aggregation; Antisense Oligonucleotides; C9orf72; FUS; Frontotemporal dementia; TDP-43; Therapeutic routes; progranulin; tau

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