German Centre for Neurodegenerative Diseases, Munich, Germany.
Memory Unit, Department of Neurology, Hospital Universitario 12 de Octubre, Madrid, Spain; Group of Neurogenerative Diseases, Hospital Universitario 12 de Octubre Research Institute (imas12), Madrid, Spain.
Lancet Neurol. 2023 Nov;22(11):1048-1060. doi: 10.1016/S1474-4422(23)00247-8.
Neurodegenerative disorders, including Alzheimer's disease, are associated with microgliosis. Microglia have long been considered to have detrimental roles in Alzheimer's disease. However, functional analyses of genes encoding risk factors that are linked to late-onset Alzheimer's disease, and that are enriched or exclusively expressed in microglia, have revealed unexpected protective functions. One of the major risk genes for Alzheimer's disease is TREM2. Risk variants of TREM2 are loss-of-function mutations affecting chemotaxis, phagocytosis, lipid and energy metabolism, and survival and proliferation. Agonistic anti-TREM2 antibodies have been developed to boost these protective functions in patients with intact TREM2 alleles. Several anti-TREM2 antibodies are in early clinical trials, and current efforts aim to achieve more efficient transport of these antibodies across the blood-brain barrier. PET imaging could be used to monitor target engagement. Data from animal models, and biomarker studies in patients, further support a rationale for boosting TREM2 functions during the preclinical stage of Alzheimer's disease.
神经退行性疾病,包括阿尔茨海默病,与小胶质细胞激活有关。小胶质细胞长期以来被认为在阿尔茨海默病中具有有害作用。然而,对与晚发性阿尔茨海默病相关的风险基因进行功能分析,这些基因编码的风险基因在小胶质细胞中富集或特异性表达,揭示了意想不到的保护作用。阿尔茨海默病的主要风险基因之一是 TREM2。TREM2 的风险变体是影响趋化性、吞噬作用、脂质和能量代谢以及存活和增殖的功能丧失突变。已经开发了激动剂抗 TREM2 抗体来增强具有完整 TREM2 等位基因的患者的这些保护作用。几种抗 TREM2 抗体正在进行早期临床试验,目前的努力旨在实现这些抗体更有效地穿过血脑屏障的转运。正电子发射断层扫描成像可用于监测靶标结合。动物模型的数据和患者的生物标志物研究进一步支持在阿尔茨海默病的临床前阶段增强 TREM2 功能的合理性。