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Updated: Aug 17, 2026

Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 19, 2013
Targeted therapy: for kids, too
1Division of Pediatrics, UT-MD Anderson Cancer Center, Houston, TX 77030, USA. sjcorey@mdanderson.org
Abstract:
Innovative hypothesis-driven clinical trials have achieved major successes over the past several decades in treating children and adolescents with cancer. DNA-damaging cytotoxic agents have cured children with cancer. While the mission is not yet accomplished, chemotherapy has been validated. None of these drugs were designed specifically for a pediatric disease. Continued progress will require new strategies. Now being tested for adult cancers, these strategies include gene therapy, immunotherapy, cancer prevention, and signal transduction inhibitor (STI) therapy. Of these, the most promising is STI therapy, also known as molecular therapeutics or targeted therapy. For this therapy to succeed, components of signal transduction (i.e., candidate drug targets) must be identified, the targets relevant to cancers, and the drugs available for trial. Because STI therapy is biologically driven and because therapy will be tailored depending on the molecular profile of a specific patient's tumor, clinical pediatric oncologists will need to acquire greater understanding of signaling pathways and their therapeutic relevance. With examples drawn from pediatric oncology, the critical steps in the pre-clinical development of targeted therapy are reviewed here.
Insights
Signal transduction inhibitor (STI) therapy, or targeted therapy, shows promise for treating pediatric cancers. This approach requires identifying specific molecular targets within tumors for tailored treatment strategies.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- Chemotherapy has historically cured pediatric cancers but was not developed for children.
- New strategies are needed to improve outcomes in pediatric oncology.
- Current advancements include gene therapy, immunotherapy, and signal transduction inhibitor (STI) therapy.
Purpose of the Study:
- To review the critical steps in the pre-clinical development of targeted therapy for pediatric cancers.
- To highlight the potential of STI therapy in pediatric oncology.
- To emphasize the need for pediatric oncologists to understand molecular signaling pathways.
Main Methods:
- Review of pre-clinical development steps for targeted therapy.
- Discussion of signal transduction pathways as drug targets.
- Examples from pediatric oncology cases.
Main Results:
- Signal transduction inhibitor (STI) therapy, also known as targeted therapy, is a promising strategy for pediatric cancers.
- Successful STI therapy requires identification of relevant molecular targets and development of suitable drugs.
- Tailoring therapy based on a patient's tumor molecular profile is crucial.
Conclusions:
- Targeted therapy offers a biologically driven approach to treating pediatric cancers.
- Pediatric oncologists require enhanced understanding of molecular signaling pathways.
- Further development of STI therapy is essential for advancing pediatric cancer treatment.
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