Infantile Fibrosarcoma With NTRK3-ETV6 Fusion Successfully Treated With the Tropomyosin-Related Kinase Inhibitor LOXO-101

Infantile fibrosarcoma (IFS) is a rare pediatric cancer typically presenting in the first 2 years of life. Surgical resection is usually curative and chemotherapy is active against gross residual disease. However, when recurrences occur, therapeutic options are limited. We report a case of refractor...

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Bibliographic Details
Published in:Pediatric blood & cancer Vol. 63; no. 8; pp. 1468 - 1470
Main Authors: Nagasubramanian, Ramamoorthy, Wei, Julie, Gordon, Paul, Rastatter, Jeff C., Cox, Michael C., Pappo, Alberto
Format: Journal Article
Language:English
Published: United States Blackwell Publishing Ltd 01.08.2016
Wiley Subscription Services, Inc
John Wiley and Sons Inc
Subjects:
Brief Report
Brief Reports
ETS Translocation Variant 6 Protein
ETV6-NTRK3
Female
Fibrosarcoma - diagnostic imaging
Fibrosarcoma - drug therapy
Fibrosarcoma - genetics
Fibrosarcoma - surgery
gene fusion
Genes
Hematology
Humans
Infant
infantile fibrosarcoma
Kinases
LOXO-101
Magnetic Resonance Imaging
Oncology
Pediatrics
Proto-Oncogene Proteins c-ets - genetics
Pyrazoles - therapeutic use
Pyrimidines - therapeutic use
Receptor, trkC - antagonists & inhibitors
Receptor, trkC - genetics
Recombinant Fusion Proteins - genetics
Repressor Proteins - genetics
tropomyosin-related kinase (TRK)
LOXO-101
ETV6-NTRK3
infantile fibrosarcoma
tropomyosin-related kinase (TRK)
gene fusion
ISSN:1545-5009, 1545-5017, 1545-5017
Online Access:Get full text
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Description
Summary:Infantile fibrosarcoma (IFS) is a rare pediatric cancer typically presenting in the first 2 years of life. Surgical resection is usually curative and chemotherapy is active against gross residual disease. However, when recurrences occur, therapeutic options are limited. We report a case of refractory IFS with constitutive activation of the tropomyosin‐related kinase (TRK) signaling pathway from an ETS variant gene 6–neurotrophin 3 receptor gene (ETV6–NTRK3) gene fusion. The patient enrolled in a pediatric Phase 1 trial of LOXO‐101, an experimental, highly selective inhibitor of TRK. The patient experienced a rapid, radiographic response, demonstrating the potential for LOXO‐101 to provide benefit for IFS harboring NTRK gene fusions.
Bibliography:Loxo Oncology, Inc.
istex:FFB34EF41B208F6D04A3BFEB93672189A276053B
ArticleID:PBC26026
ark:/67375/WNG-F0M18RPN-5
Grant sponsor: Loxo Oncology, Inc.
Conflict of interest: Michael C. Cox, Pharm.D., MHSc, is an employee and stock holder of Loxo Oncology, Inc. All other authors have no conflict of interest to declare.
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ISSN:1545-5009
1545-5017
1545-5017
DOI:10.1002/pbc.26026