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Molecular Profile | ALK R1275Q |
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 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 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 | 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 | Preclinical - Cell culture | Actionable | In a preclinical study, Lorbrena (lorlatinib) decreased viability of neuroblastoma cell lines harboring ALK R1275Q in culture (PMID: 38032104). | 38032104 |
PubMed Id | Reference Title | Details |
---|---|---|
(26554404) | The ALK/ROS1 Inhibitor PF-06463922 Overcomes Primary Resistance to Crizotinib in ALK-Driven Neuroblastoma. | Full reference... |
(37147298) | Circulating tumor DNA reveals mechanisms of lorlatinib resistance in patients with relapsed/refractory ALK-driven neuroblastoma. | Full reference... |
(37561984) | Anaplastic Lymphoma Kinase Inhibitors for Therapy of Neuroblastoma in Adults. | Full reference... |
(38032104) | SHP2 inhibition with TNO155 increases efficacy and overcomes resistance of ALK inhibitors in neuroblastoma. | Full reference... |
(39177282) | Long-Lasting Response to Lorlatinib in Patients with ALK-Driven Relapsed or Refractory Neuroblastoma Monitored with Circulating Tumor DNA Analysis. | Full reference... |