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Updated: Apr 24, 2026
Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
Novel methods and approaches to acute lymphoblastic leukemia drug discovery
Michael C Wei1, Michael L Cleary
1Stanford University School of Medicine, Department of Pediatrics , 265 Campus Drive, Lokey Stem Cell Research Building, Stanford, CA 94305 , USA mcwei@stanford.edu.
Introduction:
Acute lymphoblastic leukemia (ALL) is a significant cause of cancer-related morbidity and mortality. Major advances in the understanding of the pathogenesis of ALL have uncovered new disease-associated biomarkers that can be targeted by biological and small-molecule therapeutics.
Areas Covered:
In this review, the authors examine novel approaches to target and drug discovery in ALL over the past 10 years. Cell surface antigens can be targeted by engineered mAbs and chimeric antigen receptor T cells. Detailed mechanistic studies in Philadelphia chromosome-positive ALL and ALL with mixed lineage leukemia rearrangements highlight current molecular approaches to target and drug discovery. Genomic technologies have uncovered genetic alterations that are potentially targetable. In addition, phenotypic screening can uncover unexpected targets. New targets in ALL include cell surface antigens, kinases, tumor suppressors, transcription factors, epigenetic regulators and metabolic enzymes.
Expert Opinion:
There are a number of effective approaches for discovering novel targets in ALL. Target validation is essential for further development of new therapeutics. Identifying select patient subsets with specific genetic vulnerabilities will be important in moving these therapeutics forward clinically.
Insights
Novel therapeutic targets for acute lymphoblastic leukemia (ALL) are emerging from advances in understanding its pathogenesis. Research focuses on cell surface antigens, kinases, and genetic vulnerabilities for targeted drug discovery in ALL.
Area of Science:
- Oncology
- Hematology
- Cancer Biology
Background:
- Acute lymphoblastic leukemia (ALL) remains a major cause of cancer mortality.
- Advances in understanding ALL pathogenesis reveal new biomarkers for targeted therapies.
Purpose of the Study:
- To review novel target and drug discovery approaches in ALL over the last decade.
- To highlight emerging therapeutic strategies for ALL.
Main Methods:
- Review of recent literature on ALL target discovery.
- Analysis of cell surface antigen targeting (mAbs, CAR T-cells).
- Examination of molecular approaches in specific ALL subtypes (Ph+, MLL rearrangements).
- Integration of genomic and phenotypic screening data.
Main Results:
- Identified novel targets including cell surface antigens, kinases, tumor suppressors, transcription factors, epigenetic regulators, and metabolic enzymes.
- Demonstrated the potential of engineered mAbs and CAR T-cells for antigen targeting.
- Highlighted the role of genomic technologies in uncovering targetable genetic alterations.
Conclusions:
- Effective strategies exist for discovering novel ALL targets.
- Target validation is critical for therapeutic development.
- Identifying patient subsets with genetic vulnerabilities is key for clinical advancement.
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