Unveiling a Novel Therapeutic Target to Slow Parkinson's Disease Progression

Researchers from Shanghai Huashan Hospital have announced a breakthrough in Parkinson's disease research by pinpointing a protein known as FAM171A2. This protein appears to function as a selective “recognition gate” on neuronal cell membranes, attracting pathological alpha-synuclein, which misfolds and ultimately leads to the death of nerve cells. The study, led by Yu Jintai and published in the journal Science, represents the result of five years of clinical and basic research that included genome-wide association analyses across large populations.

In their extensive study, the team discovered that FAM171A2's interaction with alpha-synuclein could be blocked using a molecule called Bemcentinib, identified from over 7,000 compounds screened by artificial intelligence. This approach may offer a means to intervene in the spreading of alpha-synuclein pathology, thereby potentially delaying the progression of Parkinson's disease, which currently relies on treatments that only manage symptoms rather than halt degeneration.

The global impact of Parkinson's is considerable, with numbers expected to nearly double by 2040. By providing a potential pathway to slow the disease’s progression, the study presents a significant advancement over existing therapies that focus primarily on symptom control. The team is now turning its attention to further preclinical development, exploring a range of therapeutic approaches including small molecule drugs, antibodies, and gene therapy, with plans to expand the compound search and eventually move the findings into clinical trials. This novel research not only deepens our understanding of Parkinson's pathology but also hints at future applications for other neurodegenerative conditions such as Alzheimer's disease and dementia.