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Busulfan (BU)
Busulfan is an alkylating agent that cross-links DNA, causing strand breaks and cell death, primarily used for myeloablation in hematopoietic stem cell transplantation.
Busulfan is an alkylating agent that cross-links DNA, causing strand breaks and cell death, primarily used for myeloablation in hematopoietic stem cell transplantation. Used for Conditioning agent for hematopoietic stem cell transplantation in chronic myeloid leukemia, Conditioning agent for hematopoietic stem cell transplantation in other hematologic malignancies and severe aplastic anemia.
At a glance
| Generic name | Busulfan (BU) |
|---|---|
| Sponsor | Nanfang Hospital, Southern Medical University |
| Drug class | Alkylating agent |
| Target | DNA (non-specific alkylation) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
Busulfan covalently binds to DNA through its reactive methanesulfonate groups, creating interstrand and intrastrand cross-links that prevent DNA replication and transcription. This leads to apoptosis of rapidly dividing cells, particularly hematopoietic cells. It is used as a conditioning agent to ablate the bone marrow before stem cell transplantation, allowing engraftment of donor cells.
Approved indications
- Conditioning agent for hematopoietic stem cell transplantation in chronic myeloid leukemia
- Conditioning agent for hematopoietic stem cell transplantation in other hematologic malignancies and severe aplastic anemia
Common side effects
- Myelosuppression
- Mucositis
- Hepatotoxicity (veno-occlusive disease)
- Nausea and vomiting
- Seizures
- Secondary malignancies
Key clinical trials
- US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (PHASE1, PHASE2)
- Ruxolitinib-Decitabine Intensified Conditioning Regimen for AML: A Randomized Trial (PHASE4)
- Study on the Efficacy and Safety of the TmBU Conditioning Regimen in High-risk or Relapsed/Refractory Acute Leukemia (PHASE2)
- A Platform Protocol to Investigate Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis in Patients With Hematologic Malignancies Undergoing Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation (PHASE2)
- Allo HSCT for High Risk Hemoglobinopathies (PHASE2)
- Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders (PHASE2)
- Induction Therapy Including 131 I-MIBG and Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma Undergoing Stem Cell Transplant, Radiation Therapy, and Maintenance Therapy With Isotretinoin (PHASE1)
- Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Busulfan (BU) CI brief — competitive landscape report
- Busulfan (BU) updates RSS · CI watch RSS
- Nanfang Hospital, Southern Medical University portfolio CI