Previously, the China team had established that a variant in the FAM171A2 gene increased the risk for Parkinson’s (and other neurodegenerative diseases). In this work, they identified a receptor in the FAM171A2 protein, this receptor rolls out the red carpet for α-synuclein fibrils, ushering them inside neurons and fast-tracking their spread. Boosting FAM171A2 protein levels in neurons ramped up α-synuclein fibrils uptake, while knocking it down suppressed it.
Using artificial intelligence, they screened over 7,000 approved compounds and pinpointed bemcentinib (BGB324), an anti-cancer agent currently in clinical trials, as a promising lead candidate that would inhibit the binding of FAM171A2 protein to alpha-synuclein fibrils.
Is a cure for Parkinson’s disease hiding inside us?
Trehalose as a promising therapeutic candidate for the treatment of Parkinson's disease - 2019
Shining a light on Protein Aggregation in Parkinson's Disease
