Bien-Möller Sandra, Weidemeier Martin E, Radke Josefine, Baldauf Jörg, Engeli Stefan, Tzvetkov Mladen V, Schroeder Henry W S
Department of General Pharmacology, University Medicine Greifswald, Greifswald, Germany.
Department of Neurosurgery, University Medicine Greifswald, Greifswald, Germany.
BJC Rep. 2025 Apr 28;3(1):29. doi: 10.1038/s44276-025-00124-7.
Glioblastoma is the most aggressive primary brain tumor in adults. The prognosis is still very poor with a median survival time less than a year. A growing body of data supports the role for fatty acid oxidation (FAO) in the aggressive behavior of glioblastoma. We have previously shown that meldonium, an orally active compound that impairs FAO, caused significant growth reduction of glioblastoma in mice. Here, we report three cases of experimental meldonium-containing therapy in end-stage recurrent glioblastoma patients.
Three end-stage glioblastoma patients, who had second relapse tumor progression after standard of care therapy, received 500 mg meldonium twice a day on the top of the existing therapy regimen. Tolerability and treatment outcomes were monitored.
Meldonium was well tolerated by all three patients. One patient experienced long-term growth arrest and maintained clinically stable disease status, currently 24 months into treatment with meldonium. In contrast, the other two patients passed away.
The case reports presented here suggest good tolerability and the potential for meldonium to improve outcome in glioblastoma patients. Controlled clinical trials need to follow to evaluate systematically possible benefits from the integration of meldonium into standard glioblastoma treatment protocols.
胶质母细胞瘤是成人中最具侵袭性的原发性脑肿瘤。其预后仍然很差，中位生存时间不到一年。越来越多的数据支持脂肪酸氧化（FAO）在胶质母细胞瘤侵袭行为中的作用。我们之前已经表明，米多君是一种口服活性化合物，可损害FAO，它能显著降低小鼠胶质母细胞瘤的生长。在此，我们报告三例终末期复发性胶质母细胞瘤患者接受含米多君实验性治疗的病例。
三名终末期胶质母细胞瘤患者，在接受标准治疗后出现第二次复发肿瘤进展，在现有治疗方案基础上，每天两次服用500毫克米多君。监测耐受性和治疗结果。
三名患者对米多君耐受性良好。一名患者经历了长期生长停滞，并维持临床稳定的疾病状态，目前接受米多君治疗已达24个月。相比之下，另外两名患者去世。
此处呈现的病例报告表明米多君耐受性良好，且有可能改善胶质母细胞瘤患者的预后。需要开展对照临床试验，以系统评估将米多君纳入胶质母细胞瘤标准治疗方案可能带来的益处。
