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From Bench to Bedside: Novel Molecular Targets and Therapeutic Strategies in AML

A special issue of Cancers (ISSN 2072-6694). This special issue belongs to the section "Cancer Therapy".

Deadline for manuscript submissions: 20 May 2026 | Viewed by 260

Special Issue Editor


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Guest Editor
Department of Hematology-Oncology, Azienda Universitaria Ospedaliera Renato Dulbecco, 88100 Catanzaro, Italy
Interests: acute myeloid leukemia; chronic myeloid leukemia; myelodisplastic syndromes
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Acute myeloid leukemia (AML) is a genetically heterogeneous malignancy with historically poor outcomes, especially in older or unfit patients. The advent of targeted therapies—such as FLT3, IDH1/2, BCL2, and menin inhibitors—has transformed the therapeutic landscape, enabling precision medicine approaches tailored to specific molecular alterations. These agents, often used in combination with hypomethylating agents or intensive chemotherapy, have shown the potential to improve remission rates, survival, and quality of life. Despite these advances, challenges remain regarding resistance mechanisms, optimal sequencing, and integration with allogeneic transplantation or immunotherapy. This Special Issue will gather cutting-edge original research and comprehensive reviews on novel targets, biomarkers, and strategies to optimize patient selection, with the ultimate goal of personalizing and improving AML treatment outcomes.

Dr. Matteo Molica
Guest Editor

Manuscript Submission Information

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Keywords

  • acute myeloid leukemia
  • targeted therapy
  • precision medicine
  • FLT3 inhibitors
  • IDH1/2 inhibitors
  • BCL2 inhibition
  • menin inhibitors
  • drug resistance
  • minimal residual disease
  • combination therapy

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Published Papers (1 paper)

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Review

23 pages, 1227 KB  
Review
FLT3: A 35-Year Voyage from Discovery to the Next Generation of Targeted Therapy in AML
by Maria-Camelia Stancioaica, Daniel Coriu and Gabriel Ghiaur
Cancers 2025, 17(21), 3415; https://doi.org/10.3390/cancers17213415 - 23 Oct 2025
Viewed by 128
Abstract
FMS-like tyrosine kinase 3 (FLT3) is a crucial regulator of normal hematopoiesis, with high expression in hematopoietic stem and progenitor cells. Beyond its role in stem cell survival and proliferation, FLT3 signaling is essential for immune regulation, particularly dendritic cell differentiation and NK [...] Read more.
FMS-like tyrosine kinase 3 (FLT3) is a crucial regulator of normal hematopoiesis, with high expression in hematopoietic stem and progenitor cells. Beyond its role in stem cell survival and proliferation, FLT3 signaling is essential for immune regulation, particularly dendritic cell differentiation and NK cell expansion. In acute myeloid leukemia (AML), FLT3 mutations—most commonly internal tandem duplications (FLT3-ITD) and tyrosine kinase domain (FLT3-TKD) substitutions—are among the most frequent genetic alterations, driving constitutive activation of proliferative and antiapoptotic pathways and conferring adverse prognosis. The clinical development of FLT3 inhibitors has been a decades-long endeavor. Early multikinase agents established proof-of-concept but were hampered by off-target effects and incomplete efficacy. The subsequent generation of potent and selective inhibitors has transformed outcomes, culminating in FDA approvals of midostaurin, quizartinib, and gilteritinib. Together with allogeneic transplantation, these agents have reshaped the treatment paradigm for FLT3-mutant AML, converting a historically high-risk subset into one with realistic prospects for long-term survival. Despite these advances, challenges remain. Resistance emerges through cell-intrinsic mechanisms such as acquisition of secondary TKD or RAS pathway mutations, metabolic reprogramming, and antiapoptotic shifts, as well as cell-extrinsic mechanisms mediated by the bone marrow microenvironment, including cytokine support, stromal CYP3A4 metabolism, and retinoid inactivation. These pathways sustain measurable residual disease (MRD), the key predictor of relapse. Rational combination strategies and MRD-directed approaches are therefore essential to fully realize the curative potential of FLT3 inhibition. Full article
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