Related Experiment Video
Updated: Dec 23, 2025
Zebrafish Model of Neuroblastoma Metastasis
Published on: March 14, 2021
Cabozantinib for relapsed neuroblastoma: Single institution case series
Michael P Perisa1, Michael Storey2, Keri A Streby3,4
1Pediatric Residency Program, Nationwide Children's Hospital, Columbus, Ohio.
Abstract:
Relapsed high-risk neuroblastoma has few effective therapies currently available or in development. Cabozantinib is an Food and Drug Administration approved multitargeted tyrosine kinase inhibitor for select adult malignancies with preclinical data suggesting efficacy against neuroblastoma. A safe and tolerable dose has been identified for children, but its efficacy remains unknown. We describe four children with relapsed metastatic neuroblastoma treated with cabozantinib. All four patients had extended disease control (two complete responsesfor >12 months, 2 stable disease >6 months) with manageable predictable toxicities requiring dose reduction in two patients. We discuss the potential for the use of cabozantinib in neuroblastoma.
Insights
Cabozantinib shows promise for treating relapsed high-risk neuroblastoma. This study found extended disease control in four pediatric patients with manageable side effects, suggesting potential as a new therapy.
Area of Science:
- Pediatric Oncology
- Pharmacology
- Cancer Therapeutics
Background:
- Relapsed high-risk neuroblastoma lacks effective treatment options.
- Cabozantinib, a tyrosine kinase inhibitor, shows preclinical promise for neuroblastoma.
- A safe pediatric dose of cabozantinib is established, but efficacy is unknown.
Observation:
- Four children with relapsed metastatic neuroblastoma received cabozantinib.
- Patients experienced manageable and predictable toxicities.
- Two patients required dose reduction due to toxicity.
Findings:
- All four patients achieved extended disease control.
- Two patients achieved complete response lasting over 12 months.
- Two patients achieved stable disease for over 6 months.
Implications:
- Cabozantinib demonstrates potential efficacy in pediatric neuroblastoma.
- Further investigation into cabozantinib for neuroblastoma is warranted.
- Cabozantinib may offer a new therapeutic avenue for relapsed neuroblastoma.
