Professor Wen-Sung Lai's research team at National Taiwan University has successfully developed a new drug, RS-D7, by combining years of research, literature data, and artificial intelligence technology. RS-D7 works by inhibiting D-amino acid oxidase (DAO) to enhance NMDA receptor activity, effectively improving motor and cognitive deficits in patients with Multiple System Atrophy (MSA). This breakthrough drug has the potential to become a groundbreaking treatment for MSA.

Multiple System Atrophy: A Rare and Fatal Disease with Limited Treatment Options

MSA is a rare and fatal neurodegenerative disease that typically progresses rapidly within five years of onset, with most patients dying within nine years. Research shows that abnormal accumulation of alpha-synuclein (α-syn) protein in the brains of MSA patients leads to a reduction or decrease in the activity of NMDA receptors, affecting functions such as learning, memory, and movement. This results in autonomic dysfunction, cerebellar damage, Parkinson's-related symptoms, and even cognitive impairment. Current treatments for MSA are limited, only partially alleviating Parkinson's symptoms and lacking effective drugs for ataxia and cognitive deficits caused by cerebellar dysfunction.

Precision Regulation of NMDA Receptors to Improve MSA Symptoms Through Multiple Mechanisms

As a DAO inhibitor, RS-D7 effectively inhibits DAO activity, increasing the concentration of D-serine, a co-agonist of NMDA receptors. D-serine is an important neurotransmitter modulator that binds to NMDA receptors, enhancing their response to the main neurotransmitter, glutamate, and promoting signal transmission between neurons. By increasing D-serine concentration, RS-D7 can significantly enhance NMDA receptor function, improving the motor and cognitive deficits caused by reduced NMDA receptor activity in MSA patients. Additionally, chronic inflammation in the brains of MSA patients leads to neuronal damage and degeneration. RS-D7 not only enhances NMDA receptor activity but also inhibits brain inflammation, protecting neurons and slowing disease progression, offering comprehensive treatment benefits for MSA patients.

FDA Orphan Drug Designation and Upcoming Clinical Trials

Professor Wen-Sung Lai stated that RS-D7 has shown efficacy in improving motor coordination and cognitive abilities in MSA mice during preclinical animal studies, and its safety has been confirmed in proof-of-concept clinical trials. RS-D7 has already obtained multiple international patents and received FDA Orphan Drug Designation, with Phase I clinical trials set to begin soon. In the future, RS-D7 is expected to become the preferred new drug for treating MSA and may also be used to treat schizophrenia, Alzheimer's disease, and other neurodegenerative disorders, bringing new hope and improved quality of life to patients suffering from these conditions.

Resource (mandarin): 治療致命多重系統退化症MSA，台大研發RS-D7獲FDA孤兒藥資格！