Drug intelligence / Profile preview

decitabine + azacitidine + mitoxantrone liposome + cytarabine + G-CSF

Development stage
Preclinical
Lead developer
Eisai
Modality
Small Molecules, Cytokines & Interferons → Recombinant Proteins and Enzymes, Liposomes → Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous, Subcutaneous, Intrathecal
01

Overview

This is a multi-agent combination regimen consisting of five drugs: - **Decitabine** and **azacitidine** are hypomethylating agents (DNA methyltransferase inhibitors) that induce DNA hypomethylation, leading to reactivation of silenced genes and promoting differentiation or apoptosis in malignant cells. - **Mitoxantrone liposome** is an anthracenedione antineoplastic agent formulated in liposomes to enhance delivery and reduce toxicity; it intercalates into DNA, inhibiting topoisomerase II and causing DNA strand breaks. - **Cytarabine** is a nucleoside analog that inhibits DNA synthesis by incorporation into DNA during replication, leading to cell death, especially in rapidly dividing cells. - **G-CSF (Granulocyte Colony-Stimulating Factor)** stimulates the bone marrow to produce neutrophils, often used as supportive care to mitigate chemotherapy-induced neutropenia. This combination has not been described as a single named regimen or product but represents an intensive therapeutic approach for acute myeloid leukemia (AML), particularly relapsed/refractory cases. The rationale for combining these agents includes epigenetic priming with hypomethylating agents before cytotoxic chemotherapy (cytarabine/mitoxantrone), with G-CSF added for hematopoietic support[1][2]. While similar regimens have been studied—such as decitabine or azacitidine followed by high-dose cytarabine plus mitoxantrone—there are no reports of all five drugs being used together in a single standardized protocol.

02

Targets

TOP2A (DNA topoisomerase II)CSF3R (Granulocyte colony-stimulating factor receptor)DNA polymerase familyDNADNMT1 (DNA (cytosine-5)-methyltransferase 1)TOP2B (DNA topoisomerase II beta)

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