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Developing Anticancer Drugs in Orphan Molecular Entities — A Paradigm under Construction
Developing Anticancer Drugs in Orphan Molecular Entities — A Paradigm under Construction
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Developing Anticancer Drugs in Orphan Molecular Entities — A Paradigm under Construction
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Developing Anticancer Drugs in Orphan Molecular Entities — A Paradigm under Construction
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Developing Anticancer Drugs in Orphan Molecular Entities — A Paradigm under Construction
Developing Anticancer Drugs in Orphan Molecular Entities — A Paradigm under Construction
Journal Article

Developing Anticancer Drugs in Orphan Molecular Entities — A Paradigm under Construction

2018
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Overview
Genomic characterization of cancers has shown that some oncogenic alterations occur at very low frequency and are shared across tumor types. For example, NTRK translocations mediate malignant transformation and are observed in less than 1% of cancers and in more than 20 cancer types. 1 The genes NTRK1 , NTRK2 , and NTRK3 encode for the tropomyosin receptor kinase (TRK) proteins TRKA, TRKB, and TRKC, respectively. In this issue of the Journal , Drilon et al. 2 report a pooled analysis of three prospective clinical trials testing larotrectinib, a TRK inhibitor, in 55 patients. The overall response rate was 80%, and 71% of the responses . . .