SHANGHAI, Feb. 23 (Xinhua) -- Chinese scientists have discovered a novel therapeutic target for Parkinson's disease, a neurodegenerative condition with high disability and mortality rates, and have successfully identified a potentially effective small molecule drug.
The breakthrough, achieved by a team from Huashan Hospital affiliated with Fudan University in Shanghai, holds promise for early intervention in Parkinson's disease, offering patients renewed hope for delaying its progression. The research was published in the prestigious academic journal Science on Friday (Beijing time).
The battle against Parkinson's disease, the second most common neurodegenerative disorder after Alzheimer's, has been ongoing for years. There are approximately 3 million Parkinson's patients in China, roughly half of the global total.
People diagnosed with Parkinson's disease may initially experience a reduced sense of smell and sleep disorders, eventually developing more severe movement-related symptoms. As the disease progresses, the effectiveness of medications often declines, leading to a significant deterioration in quality of life.
Unfortunately, previous drugs and surgical therapies have only addressed the symptoms of Parkinson's disease, failing to prevent its progression. As a result, further in-depth research into the disease's pathophysiology and targeted treatments is crucial, according to the scientists.
After five years of persistent efforts, the team identified FAM171A2 as a key risk gene for neurodegeneration. Using artificial intelligence, they screened over 7,000 approved compounds and pinpointed bemcentinib, an anti-cancer agent currently in clinical trials, as a promising lead candidate.
The small molecule drug has been shown to effectively block the binding between FAM171A2 and the pathogenic protein, according to the research team.
Yu Jintai, leader of the team, said that, based on an international patent, they will focus on the pre-clinical research and development of small-molecule drugs, antibodies and gene therapies for Parkinson's disease in the coming years, with the aim of advancing clinical trials and applications to develop an innovative therapeutic system.
People over the age of 60 are more susceptible to Parkinson's disease. As China's population continues to age, this new medical breakthrough has sparked hope within both the academic and medical communities, as well as among patients, with the expectation that the research will have practical applications in the near future.
"The experimental results have helped us accurately figure out the pathophysiological mechanisms of Parkinson's disease, creating a real chance of overcoming a battle that has long seemed insurmountable," said Wang Jian, director of the neurology department at Huashan Hospital, which is also a national medical center for neurological diseases.
A referee for Science praised the discovery as a "holy grail" in Parkinson's research, highlighting its potential to lead to therapies that can block the spread of pathology and, consequently, the disease itself.
Neurological diseases, particularly those affecting the brain, such as Alzheimer's and Parkinson's, have become a major global public health and social challenge.
Chinese experts have called for greater efforts in brain science research, noting that it will be crucial for developing new treatment methods, enhancing early diagnosis, detection and intervention, and ultimately improving the quality of life for patients. ■
