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Mafosfamide is a synthetic oxazaphosphorine derivative and an alkylating agent with antineoplastic properties. It is structurally related to cyclophosphamide but, unlike cyclophosphamide, does not require hepatic activation to generate its active metabolite, 4-hydroxy-cyclophosphamide. Mafosfamide acts by alkylating DNA, forming DNA cross-links and inhibiting DNA synthesis, which leads to cell death. This mechanism makes it useful as a chemotherapeutic agent for various cancers. Mafosfamide has been investigated primarily for the treatment of neoplastic meningitis (including lymphomatous meningitis), lymphoma, leukemia, meningeal neoplasm, and brain and central nervous system tumors. Its development was led by Baxter Oncology in collaboration with the National Cancer Institute (USA), but clinical development has been discontinued[1][2][3][4][5][7].
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