Drug 'Chaperone' Fends Off Alzheimer's in Mice by Preventing Toxic Protein Clumping

Researchers at Temple University's Lewis Katz School of Medicine report in Molecular Neurodegeneration that they have developed a new drug for preventing amyloid beta and tau from clumping in the brain, according to FierceBiotech . The drug is a so-called "chaperone" because it is engineered to increase concentrations of a molecule, VPS35, critical to cells' ability to sort and move proteins. VPS35 separates dysfunctional proteins and shunts them out of cellular compartments called endosomes for purging. Previous research indicated that levels of VPS35 decline in Alzheimer's disease, and tied that reduction with the formation of tau tangles within neurons and external amyloid plaques. The drug chaperone, TPT-172, restored levels of VPS35 in mouse models along with synaptic functions. Mice treated with the drug exhibited improved memory and behavior versus animals engineered for Alzheimer's which received no treatment. "Because our most recent investigation was a preventative study, we want to know now whether this therapy could also work as a treatment for patients already diagnosed with Alzheimer's disease," said Temple Professor Domenico Praticò.