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High Dose Melphalan
High-dose melphalan is an alkylating agent that crosslinks DNA strands, causing cell death, and is used in high doses followed by stem cell rescue to treat hematologic malignancies.
High-dose melphalan is an alkylating agent that crosslinks DNA strands, causing cell death, and is used in high doses followed by stem cell rescue to treat hematologic malignancies. Used for Multiple myeloma (with autologous stem cell transplantation), Light chain amyloidosis, Waldenström macroglobulinemia.
At a glance
| Generic name | High Dose Melphalan |
|---|---|
| Also known as | Alkeran |
| Sponsor | M.D. Anderson Cancer Center |
| Drug class | Alkylating agent |
| Target | DNA (non-specific alkylation) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
Melphalan is a nitrogen mustard alkylating agent that covalently binds to DNA, creating interstrand crosslinks that prevent DNA replication and transcription, leading to apoptosis. At high doses, it achieves greater tumor cell kill but requires autologous stem cell transplantation to rescue bone marrow function. This approach is particularly effective in multiple myeloma and other chemotherapy-sensitive hematologic cancers.
Approved indications
- Multiple myeloma (with autologous stem cell transplantation)
- Light chain amyloidosis
- Waldenström macroglobulinemia
Common side effects
- Myelosuppression (neutropenia, thrombocytopenia)
- Mucositis
- Nausea and vomiting
- Diarrhea
- Infection
- Secondary malignancy
- Infertility
Key clinical trials
- MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) (PHASE2)
- Auto Transplant High Dose Melphalan vs High Dose Melphalan+Bortezomib in Pts With Multiple Myeloma Age 65 Years or Older (PHASE2)
- Total Marrow and Lymphoid Irradiation, Fludarabine, and Melphalan Before Donor Stem Cell Transplant in Treating Participants With High-Risk Acute Leukemia or Myelodysplastic Syndrome (PHASE1)
- 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)
- Reduced Intensity Haploidentical BMT for High Risk Solid Tumors (PHASE2)
- Evaluation of the Safety and Efficacy of Treatment w/High Dose Melphalan Given Directly Into the Liver Followed by Treatment w/Approved Cancer Treatment or Approved Cancer Treatment Alone in Patients w/ Metastatic Colorectal Cancer w/Liver Dominant Disease (PHASE2)
- Evaluation of the Safety and Efficacy of Treatment w/High Dose Melphalan Given Directly Into the Liver Followed by Treatment w/Approved Cancer Treatment or Approved Cancer Treatment Alone in Patients w/ Metastatic Breast Cancer w/Liver Dominant Disease (PHASE2)
- Belantamab Mafodotin in Newly Diagnosed Transplant Eligible Multiple Myeloma Patients (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:
- High Dose Melphalan CI brief — competitive landscape report
- High Dose Melphalan updates RSS · CI watch RSS
- M.D. Anderson Cancer Center portfolio CI