For the over 5 million Parkinson's disease patients in our country, the current medications and deep brain stimulation (DBS) techniques are still at the stage of "alleviating symptoms", unable to reverse the continuous loss of dopaminergic neurons. Patients often experience alternating "on" and "off" periods due to fluctuations in drug efficacy, and their quality of life is severely compromised.
In this context, an innovative therapy from the perspective of "root cause repair" - the "NCR201" therapy, which involves the derivation of dopaminergic neural precursor cells from human induced pluripotent stem cells (iPSCs), is currently undergoing clinical research at Zhongshan Hospital affiliated to Fudan University. The follow-up data of the first subject after transplantation for 3 months was recently disclosed, and several key indicators have shown clear improvements.
The motor function has significantly improved and the disease grade has been reduced.
▪ The MDS-UPDRS III (motor symptom score) showed a 30% decrease in the plateau phase compared to the baseline, and a 17% decrease in the initiation phase compared to the baseline.
▪ The Hoehn-Yahr staging (the main classification of Parkinson's disease based on the severity of motor symptoms, ranging from grade 0 to grade 5) decreased by one level from the preoperative level.
The "Good ON" time is extended every day, significantly improving the quality of life.
▪ The patient's "Good ON" time per day increased by 43%.
▪ The total score of the Parkinson's Disease Quality of Life Questionnaire (PDQ-39) improved by more than 50%, and the scores for depression and anxiety also showed significant improvement.
Professor Ding Jing, the director of the Neurology Department of Zhongshan Hospital Affiliated to Fudan University, stated:
The data from the 3-month follow-up of the first patient after transplantation indicated that both the patient's motor function and quality of life had significantly improved compared to before the treatment. This suggests that the iPSC therapy has a clear "cell replacement" mechanism. Early clinical trials have shown observable improvements in motor function and quality of life, and overall safety is controllable. This supports the continuation of subsequent clinical validation.
The core value of stem cell therapy for Parkinson's disease lies in the process of directing iPSCs to differentiate into dopaminergic neural precursor cells, and then performing targeted transplantation in the brain to replenish the missing dopaminergic neurons in Parkinson's disease. This approach aims to address the disease mechanism itself rather than merely providing symptomatic control.
Among the currently disclosed data, the first patient showed no surgical-related or perioperative serious complications, nor did any serious adverse events occur. The overall safety performance was good. Of course, the first case result still belongs to early single-case/small-sample data. Further confirmation of the stability and reproducibility of the therapeutic effect will still require more cases, control data, and longer follow-up.