Infantile Fibrosarcoma With NTRK3–ETV6 Fusion Successfully Treated With the Tropomyosin‐Related Kinase Inhibitor LOXO‐101 |
| |
Authors: | Ramamoorthy Nagasubramanian MD Julie Wei MD Paul Gordon MD Jeff C Rastatter MD Michael C Cox PharmD MHSc Alberto Pappo MD |
| |
Institution: | 1. Nemours Children's Hospital, Orlando, Florida;2. Department of Otolaryngology – Head and Neck Surgery, Northwestern University Feinberg School of Medicine, Chicago, Illinois;3. Loxo Oncology Inc, South San Francisco, California;4. St. Jude Children's Research Hospital, Memphis, Tennessee |
| |
Abstract: | 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. |
| |
Keywords: | ETV6– NTRK3 gene fusion infantile fibrosarcoma LOXO‐101 tropomyosin‐related kinase (TRK) |
|
|