Drug intelligence / Profile preview

busulfan + fludarabine + total body irradiation

Development stage
Unknown
Lead developer
GSK
Modality
Small Molecules
Administration
Intravenous, Oral, External Radiation
01

Overview

This is a combination conditioning regimen used primarily before allogeneic hematopoietic stem cell transplantation (HSCT), especially in patients with myeloid malignancies such as acute myeloid leukemia (AML). The regimen consists of two chemotherapeutic agents—busulfan and fludarabine—combined with total body irradiation (TBI). - **Busulfan** is an alkylating agent that crosslinks DNA, leading to cell death. - **Fludarabine** is a purine analog that inhibits DNA synthesis by interfering with DNA polymerase and ribonucleotide reductase. - **Total Body Irradiation** delivers ionizing radiation to the entire body, targeting both malignant cells and immune cells to facilitate engraftment and reduce relapse risk. This combination can be used as a reduced-intensity conditioning (RIC) or myeloablative regimen depending on the doses of TBI and chemotherapy administered. It aims to balance efficacy in eradicating malignant cells while minimizing toxicity for older or higher-risk patients[1][2]. The addition of TBI has been shown to reduce relapse rates compared to chemotherapy alone[2].

Other names
busulfan/fludarabine/TBIBu/Flu/TBIfludarabine/busulfan plus total body irradiation
02

Targets

POLA1 (DNA polymerase alpha)DNADNA-directed primase/polymerase protein (PrimPol)RNR (Ribonucleotide reductase)LIG1 (DNA ligase 1)

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