Vöglein Jonathan, Levin Johannes, Höglinger Günter
Neurologische Klinik und Poliklinik mit Friedrich-Baur-Institut, LMU Klinikum, Ludwig-Maximilians-Universität (LMU) München, Marchioninistr. 15, 81377, München, Deutschland.
Deutsches Zentrum für Neurodegenerative Erkrankungen e. V. (DZNE) München, München, Deutschland.
Nervenarzt. 2023 Oct;94(10):904-912. doi: 10.1007/s00115-023-01544-x. Epub 2023 Oct 6.
Hallmarks of neurodegenerative diseases such as Parkinson's disease and Alzheimer's disease are pathological protein aggregation, neuroinflammation, neurodegeneration and progressive symptoms. Due to the limited causal treatment options they represent a big challenge.
Overview of disease-modifying strategies in neurodegenerative diseases and outlook regarding future treatment development.
Literature search regarding treatment development in neurodegenerative diseases and integration of the results. Additionally, consideration of expert opinions.
The development of biomarkers and genetic parameters for the detection of causal pathologies of neurodegenerative diseases as an indispensable basis for the development of disease-modifying treatment is rapidly advancing. Targets for causal interventions are all steps in the pathophysiological cascade of neurodegenerative diseases. Therapeutic antibodies are most advanced in the development and are able to remove protein deposits from the brain and to reduce the clinical progression in Alzheimer's disease. A combination of biomarkers, genetic characteristics and clinical parameters could enable an individualized treatment.
The future of the treatment of neurodegenerative diseases focuses on disease modification using molecular-based approaches. Targeted interventions against protein aggregation, inflammation and genetic factors as well as a personalized stratification of treatment hold promise for more effective forms of treatment. Although challenges still remain, current research and clinical studies give optimism for the development of disease-modifying treatment for neurodegenerative diseases.
帕金森病和阿尔茨海默病等神经退行性疾病的特征是病理性蛋白质聚集、神经炎症、神经变性和进行性症状。由于病因治疗选择有限，它们构成了巨大挑战。
概述神经退行性疾病中疾病修饰策略以及未来治疗发展前景。
检索有关神经退行性疾病治疗发展的文献并整合结果。此外，考虑专家意见。
用于检测神经退行性疾病病因病理的生物标志物和遗传参数的开发正在迅速推进，这是开发疾病修饰治疗不可或缺的基础。病因干预的靶点是神经退行性疾病病理生理级联反应的所有步骤。治疗性抗体在开发中最为先进，能够清除大脑中的蛋白质沉积物并减缓阿尔茨海默病的临床进展。生物标志物、遗传特征和临床参数的组合能够实现个体化治疗。
神经退行性疾病治疗的未来重点在于使用基于分子的方法进行疾病修饰。针对蛋白质聚集、炎症和遗传因素的靶向干预以及个性化的治疗分层有望实现更有效的治疗形式。尽管挑战依然存在，但当前的研究和临床研究为神经退行性疾病疾病修饰治疗的发展带来了乐观前景。
