NTRK fusion as therapeutic target for treatment of central nervous system tumors — current data and perspectives

Oncology in Clinical Practice · Published 2025-11-14 · DOI 10.5603/ocp.107663

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Abstract

Neurotrophic tyrosine receptor kinase (NTRK) gene fusion represents a rare but highly significant oncogenicmolecular aberration with a predictive role in cancer therapy. Despite its low incidence, it is found in numeroustumors, including primary and metastatic central nervous system (CNS) tumors. It is of great clinical value due tothe rapidly growing market for targeted therapies for oncologic diseases. In recent years, the US Food and DrugAdministration (FDA) has approved several tropomyosin receptor kinase (TRK) inhibitors. The first of these waslarotrectinib (2018), followed by entrectinib (2019) and repotrectinib as a second-generation drug in 2023. Advancedclinical trials are currently underway for taletrectinib, which is showing promising results. These new drugs showthe ability to penetrate the blood-brain barrier, which is crucial in the treatment of CNS tumors. The efficacyof the therapies measured by the objective response rate (ORR) reaches approximately 80%, which representsa breakthrough in the treatment of patients with CNS tumors containing NTRK fusion. These therapies are revolutionizing molecular neurooncology, offering new therapeutic options for patients previouslydeprived of effective therapies, and setting the stage for further development of personalized medicinein oncology. It is extremely important to increase awareness and knowledge of NTRK fusion among researchersand clinicians, which can contribute to the intensification of clinical trials evaluating the efficacy and safetyof targeted therapies.

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Year
2025

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