Stem-Cell Study Points to New Approach to Alzheimer's Disease

A study published in Stem Cell Reports suggests improving the transportation of cellular proteins in brain cells could lead to new treatments and prevention for Alzheimer's disease, reports ScienceDaily . The researchers used human brain cells cultured from stem cells to determine a compound that strengthens the function of the endosomal network significantly reduces the production of both amyloid beta and a precursor of the tau protein. The team collected skin cells from people with Alzheimer's and from people with no dementia symptoms. They then reprogrammed the skin cells to function as stem cells, thus producing neurons with the same genetic makeup as people from whom they had collected skin samples. The investigators tested a compound that had been shown in animal studies to stabilize and boost the function of a protein assembly called the retromer, which plays a key role in how endosomal "packages" are ferried in the endosomal network. The R33 compound enhanced the function of the retromer, considerably reducing production of both the amyloid beta and the form of tau protein that readily aggregates, phosphorylated-Tau. The team used the gene editing tool CRISPR to create cells that did not generate the necessary precursor of amyloid beta, yet the compound was still efficient at lowering phosphor-Tau. "The findings suggest that something upstream is affecting the production of amyloid beta and phosphorylated-Tau independently," says University of Washington School of Medicine Professor Jessica Young. "So one thing we're going to work on going forward will be using these cell lines to identify what this upstream defect might be and whether it, too, could be a target for new therapeutics to treat Alzheimer's."