Brief Report: potent clinical and radiological response to larotrectinib in TRK fusion-driven high-grade glioma

David S. Ziegler*, Marie Wong, Chelsea Mayoh, Amit Kumar, Maria Tsoli, Emily Mould, Vanessa Tyrrell, Dong-Anh Khuong-Quang, Mark Pinese, Velimir Gayevskiy, Richard J. Cohn, Loretta M. S. Lau, Mark Reynolds, Michael C. Cox, Andrew Gifford, Michael Rodriguez, Mark J. Cowley, Paul G. Ekert, Glenn M. Marshall, Michelle Haber

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

45 Citations (Scopus)
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Abstract

Genes encoding TRK are oncogenic drivers in multiple tumour types including infantile fibrosarcoma, papillary thyroid cancer and high-grade gliomas (HGG). TRK fusions have a critical role in tumourigenesis in 40% of infant HGG. Here we report the first case of a TRK fusion-driven HGG treated with larotrectinib—the first selective pan-TRK inhibitor in clinical development. This 3-year-old girl had failed multiple therapies including chemotherapy and radiotherapy. Tumour profiling confirmed an ETV6–NTRK3 fusion. Treatment with larotrectinib led to rapid clinical improvement with near total resolution of primary and metastatic lesions on MRI imaging. This is the first report of a TRK fusion glioma successfully treated with a TRK inhibitor.

Original languageEnglish
Pages (from-to)693-696
Number of pages4
JournalBritish Journal of Cancer
Volume119
Issue number6
DOIs
Publication statusPublished - 11 Sep 2018
Externally publishedYes

Bibliographical note

Copyright the Author(s) 2018. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher.

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