Targeted Therapies for Acute Leukemias

Dear Colleagues,

Targeted therapies aim to interfere with the molecular pathways that drive the growth and survival of leukemia cells, while simultaneously mitigating any adverse effects on healthy cells. Several such agents have revolutionized treatment approaches and improved outcomes for many patients with acute leukemias. They have been used as single agents, in combination with conventional chemotherapy or stem cell transplantation to attain the most favorable results. Treatment choices are determined by various criteria, including the specific subtype of leukemia, genetic or molecular alterations, the age of the patient, and/or their previous responsiveness to therapeutic interventions.

Acute myeloid leukemia (AML) is the most common acute leukemia in adults. Recent advances have significantly impacted treatment options. Targeted agents approved and commonly used in AML are gemtuzumab ozogamicin, FLT3 inhibitors, bcl-2 inhibitors, IDH1/2 inhibitors and Hedgehog pathway inhibitors, both in first line and the relapsed/refractory (R/R) setting.

Acute lymphoblastic leukemia (ALL) is the most common acute leukemia in children. The targeted agents used in B-ALL are tyrosine kinase inhibitors when Philadelphia-positive, rituximab, blinatumomab, inotuzumab ozogamicin and, most recently, chimeric antigen receptor (CAR)-T cell therapies, such as tisagenlecleucel and axicabtagene ciloleucel that have shown promising results in treating R/R disease.

Nevertheless, questions remain about optimal sequencing, effective combinations, maintenance therapy post-remission, especially in AML, and treatment options post-CAR-T cell therapy failure in ALL. Moreover, certain AML subtypes, such as those with TP53 mutations or KMT2A rearrangements, still need better solutions. This Special Issue focuses on known or developing targeted treatments in acute leukemias to provide clinicians a comprehensive perspective on not only established, but also new and innovative findings in the field.

Dr. Eleftheria Hatzimichael
Guest Editor

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• acute leukemia
• acute myeloid leukemia
• acute lymphoblastic leukemia
• bi-specific antibodies
• FLT3 inhibitors
• bcl-2 inhibitors
• IDH1/2 inhibitors
• hedgehog pathway inhibitors
• CAR-T cell therapy