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etoposide + cytarabine + gemtuzumab + L-asparaginase + mercaptopurine + methotrexate + mitoxantrone + prednisone + vincristine

Development stage
Preclinical
Lead developer
Bristol Myers Squibb
Modality
Small Molecules, Cytotoxic ADCs → Antibody-Drug Conjugates (ADCs) → Antibody Conjugates → Antibody-Based Therapeutics, Therapeutic Enzymes → Recombinant Proteins and Enzymes
Administration
Intravenous, Oral
01

Overview

This agent is a multi-drug chemotherapy combination that includes **etoposide, cytarabine, gemtuzumab, L-asparaginase, mercaptopurine, methotrexate, mitoxantrone, prednisone, and vincristine**. Each component acts through distinct mechanisms to target malignant hematologic cells, especially in acute leukemias. Etoposide is a topoisomerase II inhibitor, cytarabine is a nucleoside analog inhibiting DNA synthesis, gemtuzumab is an anti-CD33 antibody-drug conjugate, L-asparaginase depletes asparagine essential for leukemic cell survival, mercaptopurine and methotrexate are antimetabolites, mitoxantrone is a DNA intercalating agent and topoisomerase II inhibitor, prednisone is a glucocorticoid, and vincristine inhibits microtubule assembly. Such intensive regimens are typically evaluated or used in refractory, relapsed, or high-risk acute lymphoblastic leukemia (ALL) or acute myeloid leukemia (AML) and related hematologic malignancies. This specific nine-drug protocol is not a standard named regimen, but its components are often found in advanced or clinical-trial settings for aggressive or recurrent disease[1][2][3][4][5][6].

Other names
St. Jude AML05 regimenAML05 protocolAML-05 protocolAML 05 protocol
02

Targets

DNA polymerase familyCD33 (Myeloid cell surface antigen CD33)DHFR (Dihydrofolate reductase)DNATOP2A (DNA topoisomerase II)

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