Research Institute for Diseases of Old Age Juntendo, University Graduate School of Medicine, Tokyo, Japan.
Department of Neurology, Juntendo University Faculty of Medicine, Tokyo, Japan.
Autophagy. 2024 Dec;20(12):2839-2840. doi: 10.1080/15548627.2024.2413295. Epub 2024 Oct 14.
Macroautophagy/autophagy maintains cellular homeostasis by degrading cytoplasmic components and its disruption is linked to Parkinson disease (PD), which is characterized by dopamine depletion and the accumulation of SNCA/α-synuclein aggregates in neurons. Therefore, activation of autophagy is considered a therapeutic strategy for PD; however, autophagy inducers have not yet been developed as therapeutic drugs because they are involved in a wide range of signaling pathways. Here, we focused on the lysosomal clustering around the microtubule-organizing center (MTOC) that can regulate the process of autophagosome-lysosome fusion, the final step of autophagy, and examined how lysosomal clustering affects protein degradation through autophagy. Our study identified six compounds from a high-content screen of 1,200 clinically approved drugs that induce both lysosomal clustering and autophagy. Notably, albendazole reduced SNCA aggregates in a PD model by lysosomal clustering and autophagy. These findings suggest that targeting lysosomal clustering could offer new therapeutic insights for PD.
自噬通过降解细胞质成分来维持细胞内环境的稳定,其功能障碍与帕金森病(PD)有关,PD 的特征是多巴胺耗竭和 SNCA/α-突触核蛋白聚集物在神经元中的积累。因此,激活自噬被认为是 PD 的一种治疗策略;然而,自噬诱导剂尚未被开发为治疗药物,因为它们涉及广泛的信号通路。在这里,我们专注于围绕微管组织中心(MTOC)的溶酶体聚集,它可以调节自噬体-溶酶体融合的过程,这是自噬的最后一步,并研究了溶酶体聚集如何通过自噬影响蛋白质降解。我们的研究从 1200 种临床批准药物的高内涵筛选中鉴定出 6 种既能诱导溶酶体聚集又能诱导自噬的化合物。值得注意的是,阿苯达唑通过溶酶体聚集和自噬减少 PD 模型中的 SNCA 聚集物。这些发现表明,靶向溶酶体聚集可能为 PD 提供新的治疗思路。