Molecular Profile Detail

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Profile Name NPM1 - ALK
Gene Variant Detail

NPM1 - ALK (gain of function)

Relevant Treatment Approaches ALK Inhibitor

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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown alveolar soft part sarcoma predicted - sensitive ALK Inhibitor Crizotinib Phase II Actionable In a Phase II trial, Xalkori (crizotinib) treatment resulted in partial response (PR) in 2.5% (1/40) and stable disease (SD) in 87.5% (35/40) of patients with TFE3-rearranged, MET-positive alveolar soft part sarcoma, with a 1-year progression-free survival (PFS) rate of 37.5%, and resulted in PR in 25% (1/4) and SD in 75% (3/4) of patients without TFE3 rearrangement and MET expression, with a 1-year PFS rate of 50% (PMID: 29216400; NCT01524926). 29216400
Unknown unknown Advanced Solid Tumor not applicable ALK Inhibitor Crizotinib + Dasatinib Phase I Actionable In a Phase I trial, Xalkori (crizotinib) therapy, in combination with Sprycel (dasatinib), in patients with advanced solid tumors resulted in limited efficacy, including one patient with a partial response and three patients with stable disease for at least six months or more (PMID: 29047029). 29047029
Unknown unknown triple-receptor negative breast cancer not applicable ALK Inhibitor Crizotinib + Navitoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of Navitoclax (ABT-263) and Xalkori (crizotinib) resulted in a synergistic effect and inhibited the growth of triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
Unknown unknown Advanced Solid Tumor not applicable ALK Inhibitor Panulisib Preclinical - Cell line xenograft Actionable In a preclinical study, Panulisib (P7170) inhibited growth of several solid tumor cell lines in culture and in cell line xenograft models (PMID: 25700704). 25700704
Unknown unknown acute lymphoblastic leukemia no benefit ALK Inhibitor BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in no change in event-free survival in 100% (7/7) of cell ine xenograft models of acute lymphocytic leukemia (PMID: 21298745). 21298745
Unknown unknown rhabdomyosarcoma not applicable ALK Inhibitor BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 33% (2/6) of cell line xenograft models of rhabdomyosarcoma (PMID: 21298745). 21298745
Unknown unknown renal Wilms' tumor not applicable ALK Inhibitor BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 100% (3/3) of cell line xenograft models of Wilms tumor (PMID: 21298745). 21298745
Unknown unknown neuroblastoma not applicable ALK Inhibitor BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 67% (4/6) of cell line xenograft models of neuroblastoma (PMID: 21298745). 21298745
Unknown unknown osteosarcoma not applicable ALK Inhibitor BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 67% (4/6) of cell line xenograft models of osteosarcoma (PMID: 21298745). 21298745
Unknown unknown rhabdoid cancer not applicable ALK Inhibitor BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 100% (3/3) of cell line xenograft models of rhabdoid tumor (PMID: 21298745). 21298745
Unknown unknown Ewing sarcoma not applicable ALK Inhibitor BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 50% (2/4) of cell line xenograft models of Ewing sarcoma (PMID: 21298745). 21298745
Unknown unknown renal cell carcinoma no benefit ALK Inhibitor Axitinib + Dalantercept Phase II Actionable In a Phase II trial (DART), the combination of Dalantercept (ACE-041) and Inlyta (axitinib) did not significantly improve median progression-free survival (6.8 vs 5.6 months, HR=1.11, p=0.670), median overall survival (HR=1.39, p=0.349), or objective response rate (11/38, 19.0% vs 15/61, 24.6%) compared to placebo plus Inlyta (axitinib) in patients with advanced renal cell carcinoma (PMID: 30951193; NCT01727336). 30951193
Unknown unknown ovary epithelial cancer no benefit ALK Inhibitor Dalantercept Phase II Actionable In a Phase II trial, Dalantercept (ACE-041) demonstrated safety, but did not result in objective response in patients with persistent or recurrent epithelial ovarian cancer, with stable disease as best response in 36.7% (11/30) of patients, a median progression-free survival of 1.7 months, and a median overall survival of 19.8 months (PMID: 30041929). 30041929
Unknown unknown hepatocellular carcinoma no benefit ALK Inhibitor Dalantercept + Sorafenib Phase I Actionable In a Phase Ib trial, addition of Dalantercept (ACE-041) to Nexavar (sorafenib) was well tolerated, but did not improve the efficacy of Nexavar (sorafenib) in patients with advanced hepatocellular carcinoma, with overall survival ranged from 1.9 to 23.3 months and stable disease as best overall response in 53.3% (11/21) of the patients (PMID: 30352941; NCT02024087). 30352941
Unknown unknown hepatocellular carcinoma not applicable ALK Inhibitor Cabozantinib + CT-707 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Cometriq (Cabometyx, cabozantinib) and CT-707 resulted in synergism in hepatocellular carcinoma cells, demonstrating increased apoptosis and inhibition of colony formation in culture and decreased tumor weight in xenograft models (PMID: 27638856). 27638856
Unknown unknown hepatocellular carcinoma not applicable ALK Inhibitor CT-707 + PHA-665752 Preclinical - Cell culture Actionable In a preclinical study, the combination of PHA-665752 and CT-707 resulted in synergism in hepatocellular carcinoma cells in culture, demonstrating near complete cell death (PMID: 27638856). 27638856
Clinical Trial Phase Therapies Title Recruitment Status