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Metabolic reprogramming represents a targetable mechanism to overcome acquired resistance to venetoclax in acute myeloid leukemia

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Author(s):
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de Queiroz, Gustavo Nery ; Lima, Keli ; Cipelli, Marcella ; Tomaz, Victoria ; Cortez, Luiz Gustavo Ferreira ; Silvad, Marina de Franca Basto ; Guedes, Rafael Lucas Muniz ; Campregher, Paulo Vidal ; Rego, Eduardo Magalhaes ; Camara, Niels Olsen Saraiva ; Costa-Lotufo, Leticia Veras ; Machado-Neto, Joao Agostinho
Total Authors: 12
Document type: Journal article
Source: BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE; v. 1872, n. 1, p. 12-pg., 2025-10-02.
Abstract

Acute myeloid leukemia (AML) often develops resistance to the BCL2 inhibitor venetoclax through metabolic reprogramming. This study established acquired venetoclax-resistant AML models (MV4-11VR and MOLM13VR) to explore resistance mechanisms and therapeutic strategies. Cell viability and apoptosis assays revealed robust acquired resistance to venetoclax upon intermittent drug exposure. Metabolic profiling revealed distinct adaptations: MV4-11VR cells favored glycolysis, while MOLM-13VR cells increased oxidative phosphorylation. Proteomic analysis supported these findings, showing pathway enrichment for carbohydrate metabolism in MV4-11VR and aerobic energy production in MOLM-13VR. Despite these differences, both models shared hyperactivation of the PI3K/AKT/mTOR pathway, as shown by RPS6 hyperphosphorylation. Apoptotic regulation also diverged between the cellular models in relation to modulated BCL2-related genes and activation of the MAPK signaling pathway. Targeting these metabolic changes with metformin (a mitochondrial complex I inhibitor) or KPT-9274 (a NAMPT inhibitor) re-sensitized resistant cells to venetoclax. Combination treatments showed strong synergy and near-complete cell elimination. These results highlight metabolic reprogramming as a heterogeneous but targetable resistance mechanism and support combining metabolic inhibitors with BCL2 blockade to treat refractory AML. (AU)

FAPESP's process: 23/07482-2 - Sensing extra and intracellular stressors by renal and immune cells: new insights into signal reception and transduction, and their relevance for understanding renal diseases
Grantee:Niels Olsen Saraiva Câmara
Support Opportunities: Research Projects - Thematic Grants
FAPESP's process: 23/01331-2 - Investigation of potential molecular targets in models of acute myeloid leukemias with acquired resistance to venetoclax.
Grantee:Gustavo Nery de Queiroz
Support Opportunities: Scholarships in Brazil - Master
FAPESP's process: 23/08735-1 - ProspecMar network: prospecting marine natural resources
Grantee:Leticia Veras Costa Lotufo
Support Opportunities: BIOTA-FAPESP Program - Thematic Grants
FAPESP's process: 23/12246-6 - Evaluation of the antineoplastic effects of the multikinase inhibitor AD80 in hematologic malignancies with constitutive activation of tyrosine-kinase pathways
Grantee:João Agostinho Machado Neto
Support Opportunities: Regular Research Grants
FAPESP's process: 21/11606-3 - Investigation of the antineoplastic effects of novel PIP4K2 and HDAC inhibitors in hematologic malignancies
Grantee:João Agostinho Machado Neto
Support Opportunities: Regular Research Grants
FAPESP's process: 24/02796-1 - Prospecting biomarkers of response to antineoplastic agents and molecular and clinical-laboratory correlation in acute lymphoblastic leukemia using an ex vivo platform
Grantee:Keli Cristina de Lima
Support Opportunities: Scholarships in Brazil - Post-Doctoral