The ongoing search for effective treatments for Alzheimer’s disease has focused on aggregates of amyloid-β peptides, which are the hallmark of the disease. However, efforts to inhibit one of the proteins responsible for producing amyloid-β, called BACE1, have led to several failed Phase 3 clinical trials.
But researchers aren’t giving up hope. A study published in Science Translational Medicine on November 18 reveals how variants in a gene called GGA3—which are a known risk factor for developing Alzheimer’s—alter BACE1 movement through brain cells in culture and in mice. This causes the buildup of BACE1 protein and creates axonal damage similar to that seen in the pre-symptomatic stages of Alzheimer’s disease. This could mean BACE1 inhibitors still have promise as a treatment if used much earlier in the disease process.
“I think the core message from this paper is that this could be one additional primary pathogenic development that precedes amyloid ...
