Drug intelligence / Profile preview

cyclophosphamide + melphalan + mitoxantrone

Development stage
Preclinical
Lead developer
Takeda
Modality
Small Molecules
Administration
Intravenous
01

Overview

This is a combination chemotherapy regimen consisting of three alkylating agents: cyclophosphamide, melphalan, and mitoxantrone. All three are small molecule antineoplastic drugs used primarily in the treatment of hematologic malignancies such as multiple myeloma and lymphoma. Cyclophosphamide and melphalan are nitrogen mustard derivatives that act by cross-linking DNA strands, thereby inhibiting DNA replication and transcription. Mitoxantrone is an anthracenedione that intercalates into DNA, causing strand breaks and inhibiting both DNA and RNA synthesis; it also inhibits topoisomerase II. This combination may be used as part of high-dose conditioning regimens prior to autologous stem cell transplantation or for salvage therapy in refractory cancers[1][2][7]. The regimen's toxicity profile includes significant hematological suppression (myelosuppression), mucositis, gastrointestinal symptoms, risk of infection, hemorrhagic cystitis (especially with cyclophosphamide), and potential cardiac toxicity (notably from mitoxantrone)[1][2].

02

Targets

TOP2A (DNA topoisomerase II)DNA

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