Introduction: Frontotemporal dementia (FTD) includes a group of neurodegenerative diseases characterized clinically by behavioral disturbances and by neurodegeneration of brain anterior temporal and frontal lobes, leading to atrophy. Apart from symptomatic treatments, there is, at present, no disease-modifying cure for FTD.
Areas covered: Three main mutations are known as causes of familial FTD, and large consortia have studied carriers of mutations, also in preclinical Phases. As genetic cases are the only ones in which the pathology can be predicted in life, compounds developed so far are directed toward specific proteins or mutations. Herein, recently approved clinical trials will be summarized, including molecules, mechanisms of action and pharmacological testing.
Expert opinion: These studies are paving the way for the future. They will clarify whether single mutations should be addressed rather than common proteins depositing in the brain to move from genetic to sporadic FTD.
Keywords: Chromosome 9 open reading frame 72 (C9orf72); Frontotemporal dementia (FTD); Frontotemporal lobar degeneration (FTLD); Microtubule associated protein tau (MAPT); Progranulin (GRN); TAR DNA-binding protein 43 (TDP-43).