Related Experiment Video
Updated: Aug 25, 2026

Comet Assay to Quantify DNA Damage in FLT3 Mutant-expressing 32D Cells after Exposure to Type I and Type II FLT3 Inhibitors
Published on: October 17, 2025
FLT3 inhibitors: a paradigm for the development of targeted therapeutics for paediatric cancer
1Department of Oncology, Sidney Kimmel Cancer Center at Johns Hopkins, Baltimore, MD 21231-1000, USA.
Abstract:
The area of molecularly-targeted cancer therapeutics is generating tremendous interest and excitement. While clinical investigation of these agents has been largely limited to adults, clinical trials for paediatric cancer patients with many of these agents are now underway. This paper reviews the current status of molecularly-targeted therapies for paediatric malignancies, with special attention given to one class of agents, inhibitors of the FLT3 receptor tyrosine kinase. FLT3 is expressed and activated in many human leukemias, including a significant percentage of pediatric AML and infant and childhood ALL, especially in the setting of MLL gene rearrangement. Activating mutations of FLT3 portend a poor prognosis in pediatric AML. Activated FLT3 can be effectively and selectively targeted by small molecule inhibitors, and these agents have shown promise in early phase clinical trials in adults with AML. Limited preclinical data with FLT3 inhibitors in MLL-rearranged ALL have also been reported. Challenges and future directions for the use of FLT3 inhibitors and other targeted agents in paediatric cancer are discussed.
Insights
Molecularly-targeted cancer therapies, including FLT3 inhibitors, show promise for pediatric leukemias like acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL). Further research is needed to overcome challenges in their pediatric application.
Area of Science:
- Oncology
- Pharmacology
Background:
- Molecularly-targeted cancer therapeutics are a rapidly advancing field.
- Clinical trials for pediatric cancer patients are increasingly incorporating these novel agents.
Purpose of the Study:
- To review the current status of molecularly-targeted therapies for pediatric malignancies.
- To focus on FLT3 receptor tyrosine kinase inhibitors for pediatric leukemias.
Main Methods:
- Review of current literature on molecularly-targeted therapies in pediatric cancers.
- Specific focus on FLT3 inhibitors and their role in pediatric acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL).
Main Results:
- FLT3 is a key target in pediatric leukemias, particularly AML and MLL-rearranged ALL.
- Activating FLT3 mutations are associated with poor prognosis in pediatric AML.
- FLT3 inhibitors show early promise in adult AML and have limited preclinical data in pediatric ALL.
Conclusions:
- FLT3 inhibitors represent a promising targeted therapy for pediatric leukemias.
- Challenges and future directions for targeted agents in pediatric oncology require further investigation.
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...
Tumor Immunotherapy
Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase
