Abstract

Background: Ketogenic diet (KD) is one of the metabolic therapy which intents low-carbohydrate and high-fat diet, resulting in reduction of serum glucose level and induction of abundant ketone bodies. KD was established as a diet therapy for intractable epilepsy approximately in the 1920s. The metabolism of tumor cells manifests abnormal accentuation of anaerobic glycolysis regardless of oxygen concentration, resulting in assumption of quite abundant glucose for their living, proliferation. KD has been proposed as the diet for the patients with cancers including malignant brain tumors with expectancy of tumor inhibitory effect. Under the KD the normal cells are able to obtain the energy from ketones, while tumor cells are not because lacking critical ketolytic enzymes. Therefore the KD restricting glucose is supposed to selectively starve tumor cells and inhibit tumor growth. Objective: The purpose of this study is to investigate the safety and tumor inhibitory effect of KD for pediatric patients with malignant brain tumors. Methods: Written informed consent was obtained from the patient’s parents. KD treatment was conducted under our protocol approved by the Research Ethics Committee of the Institutional Review Board of Kumamoto University Hospital. Results: Two pediatric patients with infratentorial anaplastic ependymoma underwent KD. A 3-year-old boy underwent surgery followed by radio-chemotherapy. However the uncontrolled tumor progression was found with dural dissemination and palliative care with KD started. Surprisingly he had been still well without any major adverse event and no tumor progression was observed until 13 months later from starting palliative care with KD alone. He could spend the happy time in his home and could survive for 32 months from the date of diagnosis. The other is a 4-year-old girl that underwent surgery followed by radiation and KD. She continued KD without any major side effect for 8 months until she left our hospital. No tumor progression was found 7 months later after RT and she could spend the happy time in her home. Three month later after termination of KD taking, the progression of tumor was found with the dissemination in spinal cord and then she died 4 months later. Conclusions: KD was safe and provided good QOL to the pediatric patients with malignant brain tumors. KD may be an alternative treatment for pediatric patients with malignant brain tumors in palliative care.

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