With the acceleration of global population aging, Parkinson's disease has become the second most common neurodegenerative disorder after Alzheimer's disease, affecting the health and quality of life of over ten million people worldwide.
According to statistics, the total number of patients worldwide has approached 10 million, while the total number of Parkinson's disease patients in China is approximately 3 million. Relevant institutions predict that by 2050, the number of Parkinson's disease patients in China will soar to 10.5 million, making it the "peak area" of global disease patients.
What is more worthy of attention is that in recent years, Parkinson's disease has shown a trend of affecting younger people. In March this year, a study published by an expert team from Beijing Tiantan Hospital in the journal BMJ showed that the prevalence of Parkinson's disease among people aged 20 to 40 will increase most rapidly between 2021 and 2050.
01 The traditional treatment Dilemma of Parkinson's Disease
The core pathological feature of Parkinson's disease is the progressive loss of dopaminergic neurons in the substantia nigra region of the brain, which leads to motor dysfunction such as resting tremors, muscle rigidity, and bradykinesia in patients.
For a long time, clinical treatment has mainly relied on drugs to replace dopamine or regulate neurotransmitters, but it has been difficult to reverse neuronal damage. Traditional surgical treatment has limitations such as limited application scope and easy attenuation of effect.
Dopaminergic therapy remains the core treatment option at present. Levodopa, as the gold standard drug, is converted into dopamine through the blood-brain barrier, but long-term use can lead to complications such as dyskinesia.
Non-dopaminergic drugs such as the NMDA receptor antagonist amantadine can improve dyskinesia, while adenosine A2A receptor antagonists can reduce the duration of the "critical phase". In surgical intervention, deep brain stimulation (DBS) can significantly improve motor symptoms in advanced patients, but it may cause side effects such as cognitive impairment.
02 Stem cell technology brings new hope
With the development of regenerative medicine, stem cell technology has brought a new direction for solving the treatment problems of Parkinson's disease. The rise of human induced pluripotent stem cell (iPSC) technology has brought breakthrough possibilities for the radical treatment of Parkinson 's disease, particularly.
Human Induced Pluripotent Stem Cells (iPSC for short) are stem cells that, through gene reprogramming technology, reverse common somatic cells such as those in skin and blood to a state with unlimited self-renewal ability and multi-directional differentiation potential.
Its core advantage lies in: high ethical compatibility (no need to rely on embryonic stem cells); Strong differentiation potential (can be directed to differentiate into dopaminergic nerve cells); It has great personalized potential (reducing the risk of immune rejection).
In the field of neurodegenerative diseases, IPscs are regarded as "ideal seeds for repairing damaged neural circuits". By differentiating directionally into missing functional neurons, they can directly replenish the cells lost due to lesions and improve the symptoms of the disease from the pathological root.
03 Clinical Research Progress and Breakthroughs
At present, significant progress has been made in the research on the treatment of Parkinson's disease based on iPSC technology. Stem cell technology has resolved the ethical bottleneck of fetal tissue transplantation.
The midbrain dopaminergic neurons differentiated from hPSC demonstrated functional integration in animal models. After the Kyoto University team transplanted HLA-matched IPSC-derived cells, the 18F-DOPA uptake of the patients increased by 44.7%, and the motor score improved by 20.4%.
The Phase I trial of MSK-DA01 (HESC-derived) also confirmed its safety.
NCR201 injection is currently a domestically developed dopaminergic neural precursor cell therapy product based on the directed differentiation of human induced pluripotent stem cells.
The technical approach is as follows: inducing the generation of IPscs from healthy donor somatic cells, precisely regulating the differentiation conditions to direct their differentiation into functionally active dopaminergic neural precursor cells, and then implanting them into the patient's substantia nigra region through minimally invasive transplantation techniques to repair the damaged neural circuits.
Early clinical research has achieved significant breakthroughs. PET/CT imaging examinations clearly show that there is a marked enhancement of dopaminergic functional signals in the transplanted area of the patient, confirming the survival and functional integration of the transplanted cells.
The first batch of Parkinson's disease patients who received treatment demonstrated remarkable clinical benefits during the six-month follow-up: the average daily GoodON time (the "start period" without troublesome movement disorders) increased by 89.5% compared to before treatment, significantly extending the patients' autonomous activity duration. Many patients achieved "complete disappearance of the critical stage". The maximum improvement in the MDS-UPDRSIII score during the critical period was 52.9%. The Hoehn-Yahr stage drops by up to 2 grades at most.
With the advancement of medicine, the treatment of Parkinson's disease is shifting from "symptom control" to "multi-target precise intervention", and further achieving "full-process management" to help patients restore a dignified life.
The development of stem cell therapy technology has brought new hope to patients with Parkinson's disease, especially the application of iPSC technology, which is expected to bring about a paradigm shift from symptom control to nerve repair.
For the general population, paying attention to the early symptoms of Parkinson's disease, maintaining a healthy lifestyle, avoiding environmental risk factors, and having regular physical examinations are important measures for the prevention and early detection of Parkinson's disease.