Drug intelligence / Profile preview

busulfan

Development stage
Approved
Lead developer
GSK
Modality
Nucleic Acid-Directed Small Molecules → Small Molecules, Covalent Small Molecules → Small Molecules, Classical Binding Small Molecules → Small Molecules
Administration
Intravenous, Oral
01

Overview

Busulfan is a bifunctional alkylating agent and antineoplastic drug used primarily in the treatment of chronic myelogenous leukemia (CML) and as part of conditioning regimens prior to hematopoietic stem cell transplantation. It is also used for polycythaemia vera, essential thrombocythaemia, and myelofibrosis—conditions classified as myeloproliferative neoplasms[1][3][4][5]. Busulfan works by hydrolyzing to release methanesulfonate groups that generate reactive carbonium ions. These ions alkylate DNA, leading to crosslinking that disrupts DNA replication and RNA transcription, ultimately causing cell death through apoptosis[3][6]. Its cytotoxic effects are most pronounced on rapidly dividing cells such as those found in bone marrow. Busulfan has been in clinical use since the 1950s.

Brand names
BusulfexMyleran
Other names
busulphan
02

Targets

DNA

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