Abstract
BACKGROUND: 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. METHODS: 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. RESULTS: 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. CONCLUSIONS: 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.