A century-old debate on protein aggregation and neurodegeneration enters the clinic.
The correlation between neurodegenerative disease and protein aggregation in the brain has long been recognized, but a causal relationship has not been unequivocally established, in part because a discrete pathogenic aggregate has not been identified. The complexity of these diseases and the dynamic nature of protein aggregation mean that, despite progress towards understanding aggregation, its relationship to disease is difficult to determine in the laboratory. Nevertheless, drug candidates that inhibit aggregation are now being tested in the clinic. These have the potential to slow the progression of Alzheimer's disease, Parkinson's disease and related disorders and could, if administered presymptomatically, drastically reduce the incidence of these diseases. The clinical trials could also settle the century-old debate about causality.
Lansbury
Peter T
PT
Department of Neurology, Harvard Medical School and Brigham and Women's Hospital, 65 Landsdowne Street, Cambridge, Massachusetts 02139, USA. lansbury@rics.bwh.harvard.edu
Lashuel
Hilal A
HA
D000544:Alzheimer Disease / Q000175:diagnosis / Q000188:drug therapy / Q000378:metabolism / Q000503:physiopathology; D000682:Amyloid / Q000187:drug effects / Q000378:metabolism; D000818:Animals; D006801:Humans; D008954:Models, Biological; D019636:Neurodegenerative Diseases / Q000175:diagnosis / Q000188:drug therapy* / Q000378:metabolism* / Q000503:physiopathology; D011489:Protein Denaturation / Q000187:drug effects
D000682:Amyloid