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Translational Strategies for Repotrectinib in Neuroblastoma
Tara J O'Donohue1, Glorymar Ibáñez1, Diego Ferreira Coutinho1
1Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, New York.
Molecular Cancer Therapeutics
|September 5, 2021
Summary
Repotrectinib shows antitumor activity in neuroblastoma models. Combining repotrectinib, a multikinase inhibitor, with chemotherapy may offer a promising new treatment for relapsed or refractory neuroblastoma.
Area of Science:
- Oncology
- Pharmacology
Background:
- Neuroblastoma pathogenesis involves ALK, TRK, JAK2/STAT, and Src/FAK signaling pathways.
- Limited clinical data exist on multikinase inhibitors for neuroblastoma treatment.
Purpose of the Study:
- To evaluate the preclinical efficacy of repotrectinib monotherapy and combination therapy in neuroblastoma.
- To assess repotrectinib's activity against relapsed/refractory neuroblastoma models.
Main Methods:
- In vitro sensitivity assays in neuroblastoma cell lines.
- In vivo studies using genotypically diverse patient-derived xenograft (PDX) models.
Main Results:
- Repotrectinib exhibited cytotoxic activity irrespective of ALK mutational status.
- Combination therapy enhanced antiproliferative effects in cell lines.
- Repotrectinib monotherapy and combination therapy demonstrated significant antitumor activity and prolonged event-free survival in PDX models.
Conclusions:
- Repotrectinib possesses antitumor activity in diverse neuroblastoma models.
- Combination of repotrectinib with chemotherapy presents a promising therapeutic strategy for clinical translation.
