Treosulfan is an alkylating agent, which means it works by adding alkyl groups to DNA. This process damages the DNA of cells, preventing them from dividing and growing. In blood cancers such as acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), this action helps destroy abnormal blood cells and suppresses the immune system to prepare the body for a stem cell transplant.

Treosulfan is a prodrug, meaning it is converted in the body to active compounds that exert the cytotoxic (cell-killing) effects. It depletes hematopoietic stem cells (the cells that make new blood cells) and has both immunosuppressive and antitumor activities. This makes it particularly useful in conditioning regimens before allogeneic hematopoietic stem cell transplantation (alloHSCT), where the goal is to eliminate diseased cells and suppress the immune system to reduce the risk of transplant rejection.

Treosulfan

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Approved use in