The use of dianhydrogalactitol provides a novel therapeutic modality for the treatment of malignancies of the central nervous system in pediatric patients, including glioblastoma multiforme (GBM) high grade glioma, and medulloblastoma. Dianhydrogalactitol acts as an alkylating agent on DNA that creates N7 methylation and that can induce double-stranded breaks in DNA. Dianhydrogalactitol is effective in suppressing the growth of cancer stem cells and is active against tumors that are refractory to temozolomide, cisplatin, and tyrosine kinase inhibitors the drug acts independently of the MGMT repair mechanism. Dianhydrogalactitol can be used together with other anti-neoplastic agents (e.g. cisplatin) and can possess additive or super-additive effects.二去水半乳糖醇之用途為對小兒患者中樞神經系統之惡性病,包括多形性膠質母細胞瘤(GBM)高度惡性膠質瘤、與髓母細胞瘤,提供新穎之醫療模式。二去水半乳糖醇之作用為DNA之烷基化劑,造成N7甲基化,並可誘發DNA中雙股斷裂。二去水半乳糖醇可以有效壓制癌幹細胞生長,且有活性對抗已對帝盟多(temozolomide)、順鉑(cisplatin)、與酪胺酸激酶抑制劑產生頑抗性之腫瘤;該藥物之作用與MGMT修復機轉各自獨立。二去水半乳糖醇可與其他抗腫瘤劑(例如,順鉑)共同使用,且可具有加成性或超加成性效應。
