For the first time ever, researchers have been able to develop "Alzheimer's-in-dish" using an innovative laboratory culture system.
Using the system they were able to reproduce the full course of events underlying the development of Alzheimer's disease and investigators from the Genetics and Aging Research Unit at Massachusetts General Hospital (MGH) now provide the first clear evidence supporting the hypothesis that deposition of beta-amyloid plaques in the brain is the first step in a cascade leading to the devastating neurodegenerative disease.
They also identified the essential role in that process of an enzyme, inhibition of which could be a therapeutic target.
Cultured neurons from human patients with Alzheimer's exhibit elevated levels of the toxic form of amyloid found in plaques and the abnormal version of the tau protein that makes up tangles, but not actual plaques and tangles.
The team used a gel-based, three-dimensional culture system to grow human neural stem cells that carried variants in two genes, the amyloid precursor protein and presenilin 1, known to underlie early-onset familial Alzheimer's disease (FAD).
Rudolph Tanzi, PhD, said that this new system, which could be adapted to other neurodegenerative disorders, should revolutionize drug discovery in terms of speed, costs and physiologic relevance to disease.