Related Experiment Video
Updated: May 23, 2026

Pooled shRNA Library Screening to Identify Factors that Modulate a Drug Resistance Phenotype
Published on: June 17, 2022
Novel therapeutic strategies for AML in 2012
1Department of Medicine, Leukemia Service, Memorial Sloan-Kettering Cancer Center, Weill Cornell Medical College, 1275 York Ave. Box 380, New York, NY 10065, USA. Tallmanm@mskcc.org
Abstract:
During the last four decades, much progress has been made in understanding the molecular pathogenesis of acute myeloid leukemia and in identifying prognostic factors predictive of outcome. However, progress in therapy has been much slower. Since the initial description of the combination of an anthracycline and cytarabine for induction, few major advances have changed the standard of care. Furthermore, these few advances apply to younger patients and those with inherently more favorable disease biology. Intensification of post-remission cytarabine improves the cure rate of patients in first complete remission (CR). Daunorubicin dose intensification improves outcome in younger patients. Finally, allogeneic hematopoietic cell transplantation is an effective strategy for many patients in first CR. The discovery of drugs with novel mechanisms of action which are directly at specific molecular targets is among the most exciting areas of research and holds great promise for the development of effective treatment.
Insights
Therapeutic advances in acute myeloid leukemia (AML) have lagged behind molecular understanding. Current treatments like chemotherapy and stem cell transplants offer some success, but novel targeted therapies are needed.
Area of Science:
- Hematology
- Oncology
- Molecular Biology
Background:
- Significant progress in understanding acute myeloid leukemia (AML) molecular pathogenesis and prognostic factors over four decades.
- Therapeutic advancements in AML have been considerably slower compared to molecular insights.
- The standard induction therapy (anthracycline and cytarabine) has seen limited major changes, with advances primarily benefiting younger patients or those with favorable biology.
Purpose of the Study:
- To review the progress in acute myeloid leukemia (AML) therapy.
- To highlight the limitations of current treatment strategies.
- To emphasize the potential of novel molecularly targeted therapies.
Main Methods:
- Review of established and emerging treatment modalities for acute myeloid leukemia (AML).
- Analysis of the impact of dose intensification and allogeneic hematopoietic cell transplantation on patient outcomes.
- Exploration of research into novel drugs targeting specific molecular pathways in AML.
Main Results:
- Intensification of post-remission cytarabine improves cure rates in patients achieving first complete remission (CR).
- Daunorubicin dose intensification demonstrates improved outcomes in younger AML patients.
- Allogeneic hematopoietic cell transplantation is a viable strategy for many patients in first CR.
Conclusions:
- Despite progress in understanding AML, therapeutic advancements remain limited.
- Current effective strategies include post-remission chemotherapy intensification, dose-adjusted daunorubicin, and allogeneic stem cell transplantation.
- Development of novel drugs targeting specific molecular pathways represents a promising frontier for future AML treatment.
Related Concept Videos
Targeted Cancer Therapies
There are several types of targeted therapies against specific...
Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
Treatment Resistant Cancers
Cancer Therapies
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...