It is an alkylating antitumor drug and is effective for multiple myeloma, breast cancer, ovarian cancer, chronic lymphocyte and granulocytic leukemia, malignant lymphoma and multiple myeloma. It has a good effect on the treatment of limb malignant tumors such as malignant melanoma, soft tissue sarcoma and osteosarcoma by arterial perfusion. The effect of racemes on testis seminoma is better. Preparations include tablets and injections.
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Melphalan, chemically known as L-phenylalanine mustard, is a potent alkylating agent widely utilized in modern oncology. Its molecular formula is C13H18Cl2N2O2, and it is identified by the CAS Registry Number 148-82-3. Structurally, this nitrogen mustard derivative consists of a phenylalanine backbone linked to two chloroethyl groups, which facilitates its mechanism of action within cancer cells. The primary therapeutic application of Melphalan lies in the treatment of multiple myeloma, where it serves as a cornerstone for high-dose chemotherapy regimens often preceding autologous stem cell transplantation. Additionally, it is frequently employed in the management of advanced ovarian cancer, particularly in combination with other cytotoxic agents, and has shown efficacy in treating metastatic melanoma.
The drug functions by cross-linking DNA strands through alkylation, thereby inhibiting DNA replication and transcription. This disruption triggers apoptosis in rapidly dividing malignant cells. Due to its systemic toxicity profile, administration is strictly controlled, typically via intravenous infusion or oral routes depending on the specific clinical protocol. Common adverse effects include severe bone marrow suppression, leading to leukopenia, thrombocytopenia, and anemia, as well as gastrointestinal disturbances such as nausea and vomiting. Renal and hepatic function must be closely monitored during therapy. Recent advancements have also explored intra-arterial delivery for isolated limb perfusion in regional melanoma cases, maximizing local concentration while minimizing systemic exposure. Despite the emergence of novel immunotherapies and targeted agents, Melphalan remains an indispensable component of standard-of-care protocols for hematologic malignancies. Its historical significance and enduring clinical utility underscore its role as a critical tool in the fight against cancer, balancing potent efficacy with manageable, albeit significant, side effect profiles under rigorous medical supervision.