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Acute promyelocytic leukemia as a paradigm for targeted therapy
1Department of Medicine, Northwestern University Feinberg School of Medicine, and Robert H Lurie Comprehensive Cancer Center, Chicago, IL 60611, USA.
Abstract:
Substantial progress has occurred in the treatment of acute promyelocytic leukemia (APL) because of improved understanding of the pathophysiology of the disease and identification of a molecular target. Novel agents such as all-trans retinoic acid (ATRA) (alone or combined with chemotherapy) and, more recently, arsenic trioxide have produced complete remission in most patients with newly diagnosed APL and/or relapsed or refractory disease, respectively. Use of these targeted therapies has resulted in evolution of the disease from one that was historically one of the most fatal subtypes of acute myeloid leukemia (AML) to one that appears curable in 70% to 80% of patients. The targeted approach to treatment of this disease can serve as a paradigm for the treatment of other leukemias.
Insights
New treatments like all-trans retinoic acid (ATRA) and arsenic trioxide have transformed acute promyelocytic leukemia (APL) care. These targeted therapies offer a high cure rate for APL, a once fatal leukemia subtype.
Area of Science:
- Hematology
- Oncology
- Molecular Biology
Background:
- Acute promyelocytic leukemia (APL) was historically a fatal subtype of acute myeloid leukemia (AML).
- Understanding APL pathophysiology and molecular targets has driven treatment advancements.
- Novel therapeutic agents have significantly improved patient outcomes.
Purpose of the Study:
- To review the progress in APL treatment.
- To highlight the efficacy of targeted therapies in APL.
- To present APL treatment as a potential paradigm for other leukemias.
Main Methods:
- Review of scientific literature on APL treatment.
- Analysis of the impact of all-trans retinoic acid (ATRA) and arsenic trioxide.
- Evaluation of complete remission rates and patient survival data.
Main Results:
- All-trans retinoic acid (ATRA) and arsenic trioxide induce complete remission in most APL patients.
- Targeted therapies have shifted APL from a highly fatal to a potentially curable disease.
- Estimated cure rates for APL now range from 70% to 80%.
Conclusions:
- Targeted therapies have revolutionized APL treatment, offering high cure rates.
- The successful APL treatment strategy can serve as a model for other leukemias.
- Continued research into molecular targets holds promise for future cancer therapies.
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