Shanghai hospital experts detect new target to slow Parkinson's disease
Medical experts from Shanghai's Huashan Hospital announced that their AI-assisted research has detected a new target for Parkinson's disease – a milestone in China's agenda to treat the illness.
They also found a chemical candidate with potential treatment effects, which could become the world's first medication to slow progression of the neurodegenerative ailment.
This discovery is expected to offer intervention at the very early stage of the disease and greatly postpone its deterioration before people start to suffer symptoms or only have slight disorders.
Combined with current medication, the new drug developed based on the discovery can also provide relief for patients already with motion difficulties.
Parkinson's disease, with high disability and mortality, is a common movement disorder of the nervous system that worsens over time. It is the second neurodegenerative disease following Alzheimer's disease. The number of patients suffering from Parkinson's are expected to rise from 7 million in 2015 to 13 million in 2040 and China is home to half of the patients.
Actually, the disease's symptoms start and develop slowly and the whole process from change of certain protein expression to showing significant symptoms can take decades.
Previous studies have found that pathological alpha-synuclein is a key protein for Parkinson's. The protein can gather together and cause the death of neurons. Moreover, the protein can spread like "seed" to invade nearby healthy neurons and guide more clumped forms of the protein and death of neurons. When the protein spreads to certain key areas of the brain, patients start showing symptoms.
Experts from Huashan Hospital conduct Parkinson's disease research in a laboratory.
"The current treatment only targets symptoms but fails to block or postpone the disease's progression," said Dr Yu Jintai of Huashan Hospital and a leading expert in the research, which used AI-assisted method to find that protein FAM171A2 is the key for the spread of pathological alpha-synuclein and disclose its mechanism for the first time in the world.
"We found people with Parkinson's disease have higher quantity of FAM171A2 in the brain and those with more FAM171A2 have more pathological alpha-synuclein. Follow-up studies also disclosed that FAM171A2 can identify pathological alpha-synuclein and bring it into neurons, leading to the death of neuron and alpha-synuclein's spread between neurons. This theory was confirmed by our experiments on animals."
Based on the discovery, Yu's team continued to use artificial intelligence to conduct drug screening, detecting one candidate from over 7,000 small molecular chemicals.
"This chemical can have the potential to be made into oral medicine to block the combination of FAM171A2 and pathological alpha-synuclein to achieve the aim of postponing disease development," he added.
The team has applied international patent on therapy development based on FAM171A2 intervention.
"We will keep looking for more effective chemicals targeting FAM171A2 to develop a new drug, antibody and genetic therapy for Parkinson's disease that could become the world's first innovative treatment effectively blocking the progression of the disease," Yu observed.
The discovery report was published in the world-leading journal Science on Friday.
The report of the discovery was published in the world-leading journal Science on Friday and the journal said the identification of FAM171A2 as a neuronal receptor for pathological alpha-synuclein aggregates is a 'holy-grail' in Parkinson's research as it holds the promise of therapies that can block the spread of pathogens and thereby the disease. Therein, the authors address an important and significant problem.
This is a highly interesting, novel, significant, and translational study, the journal claimed.
