Recent Progress in Biological and Molecular Mechanisms and Targeted Therapies of Leukemia
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pathology, Diagnostics, and Therapeutics".
Deadline for manuscript submissions: 20 February 2026 | Viewed by 338
Special Issue Editor
Special Issue Information
Dear Colleagues,
Recent advancements in the understanding of leukemia have significantly improved the development of targeted therapies. Acute myeloid leukemia (AML) has seen major progress in molecular profiling, leading to better classification and risk stratification of the disease. This has resulted in the identification of specific genetic mutations, which are now targeted by new therapies. The introduction of low-intensity induction therapies, such as hypomethylating agents combined with venetoclax, has substantially improved outcomes for patients, especially those with poor prognoses. Additionally, targeted oral therapies against driver mutations have expanded the treatment options available. Regarding Chronic Myeloid Leukemia (CML), the excellent progress induced by tyrosine kinase inhibitors (TKIs) has dramatically improved the prognosis for CML patients, allowing many to achieve long-term remission. However, the resistance to TKIs in some CML patients necessitates ongoing research into new therapeutic strategies and combination treatments with the aim to offer a safe TKIs discontinuation and to eradicate the leukemic residual disease. Acute Lymphoblastic Leukemia (ALL) and Chronic Lymphocytic Leukemia (CLL) have benefited from targeted therapies and immunotherapies. CAR-T cell therapies, which genetically modify the patient's immune cells to fight cancer, have shown promising results, especially for ALL. Monoclonal antibody therapies and immune checkpoint inhibitors are other options being studied and used for various types of leukemia. Despite these advancements, challenges remain, particularly in overcoming resistance mechanisms that arise during treatment. Understanding these resistance pathways is crucial for developing combinatorial and sequential therapeutic approaches to enhance patient survival.
List of the main relevant topics:
- Molecular Profiling and Genetic Mutations: Identification and targeting specific genetic mutations in leukemia.
- Low-Intensity Induction Therapies: Use of hypomethylating agents combined with venetoclax.
- Targeted Therapies: Development of therapies against driver mutations.
- TKIs: Impact on Chronic Myeloid Leukemia (CML) prognosis and challenges with resistance.
- CAR-T Cell Therapies: Genetic modification of immune cells for Acute Lymphoblastic Leukemia (ALL).
- Monoclonal Antibody Therapies: Targeting specific proteins on leukemia cells.
- Immune Checkpoint Inhibitors: New options for various types of leukemia.
- Resistance Mechanisms: Understanding and overcoming resistance pathways in treatment.
Dr. Anna Sicuranza
Guest Editor
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Keywords
- leukemia-targeted therapies
- AML
- CML
- venetoclax combination therapy
- tyrosine kinase inhibitors (TKIs)
- hypomethylating agents
- ALL
- CLL
- CAR-T cell therapy
- monoclonal antibodies
- immune checkpoint inhibitors
- resistance mechanisms
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