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| Molecular Profile | ALK F1245I |
| Therapy | Lorlatinib |
| Indication/Tumor Type | neuroblastoma |
| Response Type | sensitive |
| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| ALK F1245I | neuroblastoma | sensitive | Lorlatinib | Case Reports/Case Series | Actionable | In a clinical study, Lorbrena (lorlatinib) treatment resulted in a partial response in a pediatric patient with neuroblastoma harboring ALK F1245I, inhibited viability of patient-derived cells in culture, decreased tumor growth and prolonged event-free survival in a patient-derived xenograft model (PMID: 37523146; NCT03336931). | 37523146 |
| ALK F1245I | neuroblastoma | sensitive | Lorlatinib | Case Reports/Case Series | Actionable | In a clinical case study, Lorbrena (lorlatinib) treatment resulted in a partial response in a pediatric patient with neuroblastoma harboring ALK F1245I, and in a preclinical study, induced response in a patient-derived xenograft (PDX) mouse model and zebrafish model (PMID: 40637158). | 40637158 |
| PubMed Id | Reference Title | Details |
|---|---|---|
| (37523146) | High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer. | Full reference... |
| (40637158) | Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy. | Full reference... |