Invention Library

Anti-Bin1 Treatment of Alzheimer’s Disease

Human genetics studies have identified Bin1 as second only to ApoE as a risk factor for late-onset Alzheimer’s disease (LOAD). Neurology studies indicate that Bin1 binds and influences the turnover of tau as a likely mechanism in promoting LOAD risk. Lankenau Institute for Medical Research scientists developed a cell-penetrating Bin1 antibody that appears to promote tau turnover to inhibit its expression and cellular deposition. This therapeutic technology based on novel MOA may offer a route to attenuate AD driven by tau deposition.

The scaffold and signaling molecule Bin1 modifies stress and inflammatory responses of cells under stress. A cell-permeable anti-Bin1 antibody — developed by LIMR scientists as a strategy to blunt its pathogenic function in inflammatory bowel disease — was found to exert anti-tau effects in cell culture and animals when examined. With the emergence of elevated Bin1 expression as a risk factor in LOAD development, this experimental therapeutic may be effective. Indeed, given emerging evidence of gut-brain interactions in the development of neurodegenerative diseases, including LOAD, this therapeutic intersection may be relevant.

A survival benefit has been observed in early tests of anti-Bin1 administration in a tauopathy-based mouse model of AD. Accordingly, anti-Bin1 may offer a novel tractable target to limit the development or progression of AD pathophysiology in patients.