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Safety and efficacy of lorlatinib against ALK-driven refractory or relapsed neuroblastoma
1Centre for Discovery in Cancer Research (CDCR), McMaster University, Hamilton, ON, Canada; Department of Biochemistry and Biomedical Sciences, McMaster University, Hamilton, ON, Canada; Michael G DeGroote School of Medicine, McMaster University, Hamilton, ON, Canada.
Abstract:
We spotlight the promising preliminary findings reported by Goldsmith et al. of a phase 1 first-in-child study showcasing the safety and efficacy of lorlatinib against treatment-refractory or relapsed ALK-driven neuroblastoma.1.
Insights
Lorlatinib shows promise in a phase 1 study for children with relapsed or refractory ALK-driven neuroblastoma, demonstrating safety and efficacy. Further research is warranted for this challenging pediatric cancer.
Area of Science:
- Pediatric Oncology
- Pharmacology
- Clinical Trials
Background:
- Neuroblastoma is a pediatric cancer often driven by anaplastic lymphoma kinase (ALK) gene alterations.
- Treatment-refractory or relapsed neuroblastoma has a poor prognosis.
- Targeted therapies are needed for ALK-driven neuroblastoma.
Purpose of the Study:
- To evaluate the safety and efficacy of lorlatinib in pediatric patients with relapsed or refractory ALK-driven neuroblastoma.
- To establish a recommended dose for further studies.
Main Methods:
- A phase 1, first-in-child study design.
- Enrollment of pediatric patients with confirmed ALK-driven neuroblastoma.
- Dose escalation to determine safety and tolerability.
Main Results:
- Preliminary findings indicate acceptable safety and tolerability of lorlatinib.
- Evidence of preliminary efficacy in a subset of patients.
- Identification of a potential dose for future trials.
Conclusions:
- Lorlatinib is a potential therapeutic option for pediatric patients with relapsed/refractory ALK-driven neuroblastoma.
- The phase 1 study provides a foundation for further investigation.
- Targeted inhibition of ALK may overcome treatment resistance.
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