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Cancers
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From Bench to Bedside: Novel Molecular Targets and Therapeutic Strategies...
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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 2194

Special Issue Editor

Dr. Matteo Molica

E-Mail Website
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

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as communications are invited. For planned papers, a title and short abstract (about 250 words) can be sent to the Editorial Office for assessment.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Cancers is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

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 (2 papers)

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Review

12 pages, 965 KB  
Review
Harnessing Venetoclax in NPM1-Mutated AML: A Path to Sustained Remission and Beyond
by Matteo Molica, Claudia Simio, Laura De Fazio, Caterina Alati, Massimo Martino and Marco Rossi
Cancers 2025, 17(23), 3733; https://doi.org/10.3390/cancers17233733 - 21 Nov 2025
Viewed by 399
Abstract
Background: Acute myeloid leukemia (AML) harboring NPM1 mutations constitutes a biologically and clinically distinct subtype, characterized by marked sensitivity to inhibition of the anti-apoptotic protein BCL-2. The introduction of venetoclax, a selective BCL-2 inhibitor, in combination with hypomethylating agents (HMAs), has reshaped [...] Read more.
Background: Acute myeloid leukemia (AML) harboring NPM1 mutations constitutes a biologically and clinically distinct subtype, characterized by marked sensitivity to inhibition of the anti-apoptotic protein BCL-2. The introduction of venetoclax, a selective BCL-2 inhibitor, in combination with hypomethylating agents (HMAs), has reshaped the therapeutic paradigm, particularly for patients deemed unfit for intensive chemotherapy. Materials and Methods: This review comprehensively analyzes the available scientific evidence—including prospective clinical trials, retrospective cohorts, and real-world studies—to summarize current knowledge on the efficacy, safety, and therapeutic role of venetoclax-based regimens in NPM1-mutated AML. Results and Discussion: Accumulating data demonstrate that venetoclax combined with HMAs achieves high rates of deep molecular remission and significantly improves overall survival in patients with NPM1-mutated AML. Despite these advances, important questions remain regarding the optimal duration of therapy, as well as timing and criteria for treatment discontinuation. Minimal residual disease monitoring is emerging as a pivotal tool to guide therapeutic decisions and enable personalized treatment strategies. Conclusions: Venetoclax-based regimens represent a major advancement in the treatment of NPM1-mutated AML, promoting a shift toward more targeted and less toxic therapeutic approaches. Nonetheless, prospective randomized trials are required to establish standardized clinical algorithms and to refine maintenance and discontinuation strategies, with the ultimate goal of improving patient quality of life and long-term outcomes. Full article
(This article belongs to the Special Issue From Bench to Bedside: Novel Molecular Targets and Therapeutic Strategies in AML)
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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 1648
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
(This article belongs to the Special Issue From Bench to Bedside: Novel Molecular Targets and Therapeutic Strategies in AML)
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