U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

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Mechlorethamine also known as mustine, brand name MUSTARGEN administered intravenously is the prototype anticancer chemotherapeutic drug, is indicated for the palliative treatment of Hodgkin's disease (Stages III and IV), lymphosarcoma, chronic myelocytic or chronic lymphocytic leukemia, polycythemia vera, mycosis fungoides, and bronchogenic carcinoma. In 2013 was approved orphan drug Valchlor (mechlorethamine) gel for the topical treatment of stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma (CTCL) in patients who have received prior skin-directed therapy. Mechlorethamine belongs to the group of nitrogen mustard alkylating agents. Alkylating agents work by three different mechanisms: attachment of alkyl groups to DNA bases, resulting in the DNA being fragmented by repair enzymes in their attempts to replace the alkylated bases, 2) DNA damage via the formation of cross-links (bonds between atoms in the DNA) which prevents DNA from being separated for synthesis or transcription, and 3) the induction of mispairing of the nucleotides leading to mutations all of which achieve the same end result - disruption of DNA function and cell death.