Related Experiment Video
Updated: Nov 6, 2025
Zebrafish Model of Neuroblastoma Metastasis
Published on: March 14, 2021
ALK Inhibitors in Neuroblastoma: A Sprint from Bench to Bedside
Johannes H Schulte1,2,3, Angelika Eggert4,2,3
1Department of Pediatric Oncology and Hematology, Charité Berlin, Germany. johannes.schulte@charite.de.
Abstract:
Activating mutations of the anaplastic lymphoma kinase (ALK) gene were identified in the pediatric tumor neuroblastoma, in 2008. Rapid translation of this finding into targeted neuroblastoma therapy was facilitated by the availability of ALK inhibitors developed for adult malignancies and an efficient preclinical and clinical research program.See related article by Foster et al., p. 3543.
Insights
Activating mutations in the anaplastic lymphoma kinase (ALK) gene were found in pediatric neuroblastoma. ALK inhibitors, initially for adult cancers, were quickly adapted for targeted neuroblastoma treatment.
Area of Science:
- Oncology
- Genetics
- Pediatric Cancer Research
Background:
- Activating mutations in the anaplastic lymphoma kinase (ALK) gene were identified in neuroblastoma in 2008.
- Neuroblastoma is a significant pediatric cancer.
Purpose of the Study:
- To investigate the role of ALK mutations in neuroblastoma.
- To explore the potential for targeted therapy in neuroblastoma.
Main Methods:
- Genetic analysis to identify ALK mutations.
- Utilizing ALK inhibitors developed for adult malignancies.
- Preclinical and clinical research programs.
Main Results:
- Discovery of activating ALK gene mutations in neuroblastoma.
- Successful adaptation of ALK inhibitors for neuroblastoma treatment.
Conclusions:
- Targeted therapy for neuroblastoma is feasible.
- ALK inhibitors represent a promising treatment avenue for pediatric neuroblastoma.
