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Gene | ALK |
Variant | R1275Q |
Impact List | missense |
Protein Effect | gain of function |
Gene Variant Descriptions | ALK R1275Q lies within the protein kinase domain of the Alk protein (UniProt.org). R1275Q confers a gain of function to the Alk protein as demonstrated by transformation activity in cell culture and increased downstream signalling in in vitro assays (PMID: 21838707, PMID: 29533785). |
Associated Drug Resistance | |
Category Variants Paths |
ALK mutant ALK act mut ALK R1275Q ALK mutant ALK R1275X ALK R1275Q |
Transcript | NM_004304.5 |
gDNA | chr2:g.29209798C>T |
cDNA | c.3824G>A |
Protein | p.R1275Q |
Source Database | RefSeq |
Genome Build | GRCh38/hg38 |
Transcript | gDNA | cDNA | Protein | Source Database | Genome Build |
---|---|---|---|---|---|
NM_004304 | chr2:g.29209798C>T | c.3824G>A | p.R1275Q | RefSeq | GRCh38/hg38 |
NM_004304.5 | chr2:g.29209798C>T | c.3824G>A | p.R1275Q | RefSeq | GRCh38/hg38 |
NM_004304.4 | chr2:g.29209798C>T | c.3824G>A | p.R1275Q | RefSeq | GRCh38/hg38 |
Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
---|---|---|---|---|---|---|---|
ALK R1275Q | neuroblastoma | sensitive | Crizotinib + Cyclophosphamide + Topotecan | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Xalkori (crizotinib), Topotecan, and Cytoxan (cyclophosphamide) synergized to sustain tumor regression in human neuroblastoma cell line xenograft models harboring ALK R1275Q and wild-type TP53 (PMID: 26438783). | 26438783 |
ALK R1275Q | Advanced Solid Tumor | sensitive | Brigatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a transformed cell line expressing ALK R1275Q was sensitive to Alunbrig (brigatinib) in culture and in cell line xenograft models, resulting in cell growth inhibition (PMID: 27049722). | 27049722 |
ALK R1275Q | neuroblastoma | conflicting | Crizotinib | Case Reports/Case Series | Actionable | In a Phase I/II trial (ADVL0912), Xalkori (crizotinib) treatment resulted in an objective response of 15% (3/20) in pediatric patients with relapsed/refractory neuroblastoma harboring ALK activating mutations or amplifications, among the 12 patients harboring ALK R1275Q, 1 achieved a complete response, 2 achieved partial responses, and 2 achieved prolonged stable diseases (PMID: 33568345; NCT00939770). | 33568345 |
ALK R1275Q | neuroblastoma | conflicting | Crizotinib | Preclinical - Cell culture | Actionable | In a preclinical study, Xalkori (crizotinib) decreased viability of neuroblastoma cell lines harboring ALK R1275Q in culture (PMID: 38032104). | 38032104 |
ALK R1275Q | neuroblastoma | conflicting | Crizotinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Xalkori (crizotinib) did not inhibit growth of neuroblastoma cells over expressing ALK R1275Q in culture, and only delayed tumor growth in cell line xenograft models (PMID: 26554404). | 26554404 |
ALK R1275Q | neuroblastoma | conflicting | Crizotinib | Preclinical - Cell culture | Actionable | In a preclinical study, Xalkori (crizotinib) inhibited proliferation of neuroblastoma cells harboring ALK R1275Q in culture (PMID: 29907598). | 29907598 |
ALK R1275Q | neuroblastoma | conflicting | Crizotinib | Preclinical | Actionable | In a preclinical study, Xalkori (crizotinib) treatment resulted in decreased tumor weight in a Mycn-overexpressing neuroblastoma transgenic mouse model with ALK R1275Q (corresponds to R1279Q in mouse) and subsequent upregulation of Ret (PMID: 24811913). | 24811913 |
ALK R1275Q | neuroblastoma | sensitive | Lorlatinib | Case Reports/Case Series | Actionable | In a clinical case study, Lorbrena (lorlatinib) treatment resulted in 3 complete responses and 1 partial response in 4 pediatric patients with relapsed or refractory neuroblastoma harboring ALK R1275Q, with treatment ongoing for an average of 52 months (PMID: 39177282). | 39177282 |
ALK R1275Q | neuroblastoma | sensitive | Lorlatinib | Case Reports/Case Series | Actionable | In a clinical case study, Lorbrena (lorlatinib) treatment resulted in a partial response in 3 of 4 patients with neuroblastoma harboring ALK R1275Q (PMID: 37561984). | 37561984 |
ALK R1275Q | neuroblastoma | sensitive | Lorlatinib | Case Reports/Case Series | Actionable | In a Phase I trial, Lorbrena (lorlatinib) treatment resulted in a complete response in a neuroblastoma patient harboring a germline ALK R1275Q, who was on treatment for 18 months and remained in remission at 5 years (PMID: 37147298; NCT03107988). | 37147298 |
ALK R1275Q | neuroblastoma | sensitive | Lorlatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Lorbrena (lorlatinib) decreased viability of neuroblastoma cell lines harboring ALK R1275Q in culture (PMID: 38032104). | 38032104 |
ALK R1275Q | neuroblastoma | sensitive | Lorlatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Lorbrena (lorlatinib) inhibited Alk phosphorylation, resulted in growth inhibition of neuroblastoma cells over expressing ALK R1275Q in culture and induced rapid and sustained complete tumor regression in cell line xenograft models (PMID: 26554404). | 26554404 |
ALK R1275Q | Advanced Solid Tumor | sensitive | Lorlatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Lorbrena (lorlatinib) inhibited foci formation more efficiently than Xalkori (crizotinib) in transformed cells overexpressing ALK R1275Q in culture (PMID: 26554404). | 26554404 |
ALK R1275Q | neuroblastoma | sensitive | CEP-28122 | Preclinical - Cell culture | Actionable | In a preclinical study, CEP-28122 inhibited growth of neuroblastoma cells harboring ALK R1275Q in culture (PMID: 22203728). | 22203728 |
ALK R1275Q | neuroblastoma | sensitive | Ceritinib | Case Reports/Case Series | Actionable | In a Phase I trial, Zykadia (ceritinib) treatment resulted in 3 partial responses and 1 stable disease in 7 pediatric patients with neuroblastoma harboring ALK R1275Q (PMID: 34780709; NCT01742286). | 34780709 |
ALK R1275Q | neuroblastoma | sensitive | Ceritinib | Preclinical - Cell culture | Actionable | In a preclinical study, Zykadia (ceritinib) decreased viability of neuroblastoma cell lines harboring ALK R1275Q in culture (PMID: 38032104). | 38032104 |
ALK R1275Q | neuroblastoma | sensitive | Ceritinib | Preclinical - Cell culture | Actionable | In a preclinical study, Zykadia (ceritinib) inhibited proliferation of neuroblastoma cells harboring ALK R1275Q in culture (PMID: 29907598). | 29907598 |
ALK R1275Q | neuroblastoma | no benefit | Belizatinib | Case Reports/Case Series | Actionable | In a Phase I trial, a neuroblastoma patient harboring ALK R1275Q did not respond to treatment with Belizatinib (TSR-011) (PMID: 31217479; NCT02048488). | 31217479 |
ALK R1275Q | Advanced Solid Tumor | predicted - sensitive | TPX-0131 | Preclinical - Biochemical | Actionable | In a preclinical study, TPX-0131 inhibited kinase activity of ALK R1275Q in an in vitro assay (PMID: 34158340). | 34158340 |
ALK R1275Q | neuroblastoma | sensitive | Vandetanib | Preclinical | Actionable | In a preclinical study, Caprelsa (vandetanib) treatment resulted in decreased tumor weight in a Mycn-overexpressing neuroblastoma transgenic mouse model with ALK R1275Q (corresponds to R1279Q in mouse) and subsequent upregulation of Ret (PMID: 24811913). | 24811913 |
ALK R1275Q | Advanced Solid Tumor | sensitive | Repotrectinib | Preclinical - Cell culture | Actionable | In a preclinical study, Augtyro (repotrectinib) decreased Alk phosphorylation and neurite outgrowth in cells expressing ALK R1275Q in culture (PMID: 31852910). | 31852910 |
ALK R1275Q | neuroblastoma | predicted - sensitive | Ensartinib | Preclinical - Cell culture | Actionable | In a preclinical study, Ensartinib (X-396) treatment resulted in inhibition of cell proliferation in a neuroblastoma cell line harboring ALK R1275Q in culture (PMID: 34482287). | 34482287 |
ALK R1275Q | neuroblastoma | predicted - sensitive | Repotrectinib | Preclinical - Cell culture | Actionable | In a preclinical study, Augtyro (repotrectinib) treatment resulted in inhibition of cell proliferation in a neuroblastoma cell line harboring ALK R1275Q in culture (PMID: 34482287). | 34482287 |
ALK R1275Q | Advanced Solid Tumor | predicted - sensitive | Conteltinib | Preclinical - Biochemical | Actionable | In a preclinical study, Conteltinib (CT-707) inhibited ALK R1275Q activity in an in vitro assay (PMID: 36424628). | 36424628 |
ALK R1275Q | neuroblastoma | sensitive | Ceritinib + Ribociclib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Zykadia (ceritinib) and Kisqali (ribociclib) synergistically inhibited proliferation and Alk phosphorylation and induced cell cycle arrest in neuroblastoma cell lines harboring ALK R1275Q in culture (PMID: 27986745). | 27986745 |
ALK R1275Q | neuroblastoma | sensitive | TNO155 | Preclinical - Cell culture | Actionable | In a preclinical study, TNO155 decreased viability of neuroblastoma cell lines harboring ALK R1275Q in culture (PMID: 38032104). | 38032104 |
ALK R1275Q | neuroblastoma | sensitive | Ceritinib + TNO155 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with the combination of TNO155 and Zykadia (ceritinib) synergistically decreased viability of neuroblastoma cell lines harboring ALK R1275Q in culture (PMID: 38032104). | 38032104 |
ALK R1275Q | neuroblastoma | sensitive | Lorlatinib + TNO155 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with the combination of TNO155 and Lorbrena (lorlatinib) synergistically decreased viability of neuroblastoma cell lines harboring ALK R1275Q in culture (PMID: 38032104). | 38032104 |
ALK R1275Q | neuroblastoma | sensitive | Crizotinib + TNO155 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with the combination of TNO155 and Xalkori (crizotinib) synergistically decreased viability of neuroblastoma cell lines harboring ALK R1275Q in culture (PMID: 38032104). | 38032104 |
ALK R1275Q | neuroblastoma | sensitive | Crizotinib + SHP099 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with the combination of SHP099 and Xalkori (crizotinib) synergistically decreased viability of neuroblastoma cell lines harboring ALK R1275Q in culture (PMID: 38032104). | 38032104 |
ALK R1275Q | neuroblastoma | sensitive | Ceritinib + SHP099 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with the combination of SHP099 and Zykadia (ceritinib) synergistically decreased viability of neuroblastoma cell lines harboring ALK R1275Q in culture (PMID: 38032104). | 38032104 |
ALK R1275Q | neuroblastoma | sensitive | Lorlatinib + SHP099 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with the combination of SHP099 and Lorbrena (lorlatinib) synergistically decreased viability of neuroblastoma cell lines harboring ALK R1275Q in culture (PMID: 38032104). | 38032104 |
ALK R1275Q | neuroblastoma | sensitive | SHP099 | Preclinical - Cell culture | Actionable | In a preclinical study, SHP099 decreased viability of neuroblastoma cell lines harboring ALK R1275Q in culture (PMID: 38032104). | 38032104 |