Chinese researchers have identified a new therapeutic target for Parkinson’s disease, a neurodegenerative disorder with high disability and mortality rates, and have successfully discovered a promising small-molecule drug.
A research team from Huashan Hospital, affiliated with Fudan University in Shanghai, achieved this breakthrough, which could enable early intervention in Parkinson’s disease and offer hope for slowing its progression. The findings were published on Friday (Beijing time) in the renowned academic journal Science.
Parkinson’s disease, the second most common neurodegenerative disorder after Alzheimer’s, affects around 3 million people in China—roughly half of the global total. Patients often first experience a diminished sense of smell and sleep disturbances before developing more severe movement-related symptoms. As the disease advances, the effectiveness of existing medications decreases, significantly impacting patients' quality of life.
While current drugs and surgical treatments mainly alleviate symptoms without halting disease progression, further research into its underlying mechanisms and targeted therapies remains essential, scientists say.
Following five years of dedicated research, the team identified FAM171A2 as a crucial risk gene for neurodegeneration. Leveraging artificial intelligence, they screened over 7,000 approved compounds and identified bemcentinib, an anti-cancer agent currently in clinical trials, as a promising candidate.
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According to the researchers, the small-molecule drug effectively prevents the interaction between FAM171A2 and the pathogenic protein associated with Parkinson’s.
Team leader Yu Jintai stated that, based on an international patent, future efforts will focus on pre-clinical research and the development of small-molecule drugs, antibodies, and gene therapies for Parkinson’s disease, with the goal of advancing clinical trials and innovative treatments.
The elderly, particularly those over 60, are more susceptible to Parkinson’s. As China’s population continues to age, this medical breakthrough has generated optimism among academics, medical professionals, and patients, with expectations that it will lead to practical applications soon.
"The experimental results have allowed us to precisely understand the pathophysiological mechanisms of Parkinson’s disease, bringing us closer to overcoming a challenge long considered insurmountable," said Wang Jian, director of the neurology department at Huashan Hospital, a national medical center for neurological diseases.
A Science journal reviewer described the discovery as a “holy grail” in Parkinson’s research, emphasizing its potential to prevent the spread of pathological changes and, ultimately, the disease itself.
As neurological diseases like Parkinson’s and Alzheimer’s pose growing global public health and social challenges, Chinese experts are urging increased efforts in brain science research. They highlight the need for improved diagnostic tools, early intervention strategies, and novel treatments to enhance patients' quality of life.