Wang Shizun, Zhang Dan, Wang Chunlong, Liu Yuqin
Department of Pathology, Cell Resource Center, Institute of Basic Medical Sciences, School of Basic Medicine, Chinese Academy of Medical Sciences (CAMS), Peking Union Medical College (PUMC), Beijing, China.
Acta Neuropathol Commun. 2025 Jun 28;13(1):139. doi: 10.1186/s40478-025-02051-7.
Medulloblastoma is a common embryonic malignant tumor in children. Patients with Group 3 medulloblastoma exhibit the poorest prognosis among all subgroups, and approximately 20% of these patients carry an amplification of MYC. Metabolic reprogramming, a hallmark of cancer, includes the pentose phosphate pathway (PPP) as a branch of glucose metabolism, providing cells with ribose-5-phosphate (R5P) and nicotinamide adenine dinucleotide phosphate (NADPH). The role of PPP in medulloblastoma remains unclear. In this study, we utilized transcriptomic data to identify that high expression of transketolase (TKT) correlates with worse overall survival (OS) in Group 3 patients. We found that TKT promotes proliferation of Group 3 medulloblastoma cell line cells both in vitro and in vivo. Additionally, TKT enhances R5P synthesis, increasing the proportion of S-phase cells and promoting proliferation. TKT also facilitates NADPH synthesis, which reduces intracellular reactive oxygen species (ROS) levels, inhibits ROS-induced cell death, and strengthens cellular resistance to ROS-induced injury. Subsequently, we demonstrated that inhibition of MYC leads to decreased TKT protein levels, and MYC promotes cell proliferation and suppresses cell death via TKT. Chromatin immunoprecipitation-quantitative real-time polymerase chain reaction (ChIP-qPCR) confirmed that employing the antibody targeting MYC enables the immunoprecipitation of DNA localized to the promoter region of TKT. Using luciferase assay and western blot, we verified that MYC and specificity protein 1 (SP1) co-regulate the transcription of TKT and consequently elevates TKT protein levels. Collectively, our study reports that MYC facilitates the proliferation of Group 3 medulloblastoma cells and mitigates ROS-induced damage through TKT, suggesting TKT as a potential therapeutic target for MYC-driven Group 3 medulloblastoma.
髓母细胞瘤是儿童常见的胚胎性恶性肿瘤。3组髓母细胞瘤患者在所有亚组中预后最差,约20%的此类患者存在MYC扩增。代谢重编程是癌症的一个标志,包括作为葡萄糖代谢分支的磷酸戊糖途径(PPP),为细胞提供5-磷酸核糖(R5P)和烟酰胺腺嘌呤二核苷酸磷酸(NADPH)。PPP在髓母细胞瘤中的作用仍不清楚。在本研究中,我们利用转录组数据确定转酮醇酶(TKT)的高表达与3组患者较差的总生存期(OS)相关。我们发现TKT在体外和体内均促进3组髓母细胞瘤细胞系细胞的增殖。此外,TKT增强R5P合成,增加S期细胞比例并促进增殖。TKT还促进NADPH合成,降低细胞内活性氧(ROS)水平,抑制ROS诱导的细胞死亡,并增强细胞对ROS诱导损伤的抗性。随后,我们证明抑制MYC会导致TKT蛋白水平降低,且MYC通过TKT促进细胞增殖并抑制细胞死亡。染色质免疫沉淀-定量实时聚合酶链反应(ChIP-qPCR)证实,使用靶向MYC的抗体能够免疫沉淀定位于TKT启动子区域的DNA。通过荧光素酶测定和蛋白质印迹,我们验证了MYC和特异性蛋白1(SP1)共同调节TKT的转录,从而提高TKT蛋白水平。总体而言,我们的研究报告称,MYC通过TKT促进3组髓母细胞瘤细胞的增殖并减轻ROS诱导的损伤,提示TKT作为MYC驱动的3组髓母细胞瘤的潜在治疗靶点。