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Drug Name Trade Name Synonyms Drug Classes Drug Description
Cyclophosphamide Cytoxan CPM Chemotherapy - Alkylating 18 Cytoxan (cyclophosphamide) is an alkylating agent, which inhibits DNA replication (NCI Drug Dictionary). Cytoxan (cyclophosphamide) is FDA approved in multiple hematological malignancies, breast cancer, neuroblastoma, ovarian cancer, and retinoblastoma (NCI Drug Dictionary).
Doxorubicin Adriamycin Adria|ADR|Doxorubicin hydrochloride|Hydroxydaunorubicin Chemotherapy - Anthracycline 13 TOPO2 inhibitor 5 Adriamycin (doxorubicin) is an anthracycline chemotherapeutic, in a non-liposomal formulation, which intercalates into DNA and inhibits topoisomerase II (PMID: 24367159). Doxorubicin is FDA approved for multiple cancer types (FDA.gov).
Lorlatinib Lorbrena PF-06463922|Lorviqua ALK Inhibitor 32 ROS1 Inhibitor 20 Lorbrena (lorlatinib) is a small molecule inhibitor of Alk and Ros1 fusion proteins, which may result in suppression of Alk and Ros1-mediated signaling thereby inhibiting cell growth and causing tumor regression (PMID: 26144315). Lorbrena (lorlatinib) is FDA approved for use in patients with ALK-positive non-small cell lung cancer (FDA.gov).
Vincristine Sulfate Oncovin 22-Oxovincaleukoblastine|vincristine Oncovin (vincristine) binds microtubules and prevents mitotic spindle formation, resulting in cell-cycle arrest (NCI Drug Dictionary).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
ALK F1174L neuroblastoma sensitive Cyclophosphamide + Doxorubicin + Lorlatinib + Vincristine Sulfate Preclinical Actionable In a preclinical study, the combination of Lorbrena (lorlatinib), Cytoxan (cyclophosphamide), Adriamycin (doxorubicin), and Oncovin (vincristine) resulted in an early tumor response, and improved survival in a genetically engineered mouse model of neuroblastoma harboring ALK F1174L (PMID: 36602782). 36602782
ALK amp neuroblastoma no benefit Cyclophosphamide + Doxorubicin + Lorlatinib + Vincristine Sulfate Preclinical - Pdx Actionable In a preclinical study, addition of Cytoxan (cyclophosphamide), Adriamycin (doxorubicin), and Oncovin (vincristine) to Lorbrena (lorlatinib) did not improve tumor growth inhibition in a patient-derived xenograft (PDX) model of neuroblastoma with ALK amplification and overexpression (PMID: 36602782). 36602782
ALK F1174C neuroblastoma predicted - sensitive Cyclophosphamide + Doxorubicin + Lorlatinib + Vincristine Sulfate Preclinical - Pdx Actionable In a preclinical study, the combination of Lorbrena (lorlatinib), Cytoxan (cyclophosphamide), Adriamycin (doxorubicin), and Oncovin (vincristine) resulted in modest inhibition of tumor growth in a patient-derived xenograft (PDX) model of neuroblastoma harboring ALK F1174C (PMID: 36602782). 36602782

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Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries


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