Chinese researchers find new target to treat Parkinson's disease

Chinese researchers at Huashan Hospital, an affiliate of Fudan University in Shanghai, have become the world's first to discover FAM171A2, a new therapeutic target for treating Parkinson's disease, according to a research published in the journal Science on Friday.

Led by Yu Jintai, deputy head of Huashan Hospital's neurology department, the team has also identified a candidate drug that can slow the progression of Parkinson's disease based on this new finding. This discovery was made after five years of research.

The research was also conducted under the auspices of the National Center for Neurological Diseases and the National Key Laboratory of Brain Function and Brain Diseases.

Parkinson's disease is the second most common neurodegenerative disease after Alzheimer's and seriously affects patients' daily lives. Traditional drugs and surgical treatments only address the symptoms of Parkinson's disease and do not block its progression.

The number of people with the disease may reach 13 million by 2040, with Chinese patients accounting for nearly half.

Pathological alpha-synuclein is a key pathogenic protein in the disease. Its aggregation can disrupt the normal function of neurons and lead to their death. It can also spread, damaging adjacent healthy neurons. When it affects different regions of the brain, it can cause motor symptoms such as bradykinesia, resting tremor and rigidity, as well as cognitive impairments like memory decline.

The latest breakthrough identified FAM171A2 as the crucial receptor mediating the transmission of pathological alpha-synuclein. Based on the finding, new drugs could be developed to slow the progression of the disease.

Using artificial intelligence technologies, the team found that Bemcentinib can be a candidate after screening over 7,000 compounds. Experiments have shown that this molecule can hinder the binding of FAM171A2 protein and pathological alpha-synuclein, and suppress the uptake of pathogenic protein fibrils by dopaminergic neurons.

The team applied for an international patent for treating Parkinson's disease by targeting FAM171A2. Next, they will carry out preclinical research, hoping to develop effective treatment for patients with Parkinson's disease.