Parkinson病における創薬開発の現状

抄録

Parkinson disease (PD) is characterized by the progressive loss of dopaminergic neurons in the substantia nigra, leading to motor and non–motor symptoms. While symptomatic treatments exist, disease progression results in complications that impact patients' quality of life. Recent research has focused on developing disease–modifying therapies (DMTs) alongside symptomatic treatments. As of January 31, 2024, 136 clinical trials (phases 1–3) were registered, with 56% targeting symptom relief and 44% focusing on disease modification. DMTs target α–synuclein aggregation (e.g., prasinezumab), GBA1–related lysosomal dysfunction (e.g., ambroxol), and neurotrophic pathways (e.g., HGF/MET activation). Anti–inflammatory approaches, including GM–CSF modulation and NLRP3 inhibitors, are also under investigation. Mitochondrial dysfunction research has led to trials on glycolysis enhancement (e.g., terazosin) and ATP augmentation (e.g., febuxostat and inosine). A comprehensive approach combining symptomatic and neuroprotective strategies is essential. Future research should refine disease–modifying interventions and personalized treatments to improve patient outcomes.