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Updated: Nov 11, 2025

Author Spotlight: Advancements in Molecular Biomarker Testing for Non-Squamous Non-Small Cell Lung Cancer
Published on: September 8, 2023
Therapeutic strategies in RET gene rearranged non-small cell lung cancer
Leylah M Drusbosky1, Estelamari Rodriguez2, Richa Dawar2
1Guardant 360, 505 Penobscot Drive, Redwood City, CA, 94063, USA.
Abstract:
The recent approvals by the Food and Drug Administration several tumor-agnostic drugs have resulted in a paradigm shift in cancer treatment from an organ/histology-specific strategy to biomarker-guided approaches. RET gene fusions are oncogenic drivers in multiple tumor types and are known to occur in 1-2% of non-squamous NSCLC patients. RET gene fusions give rise to chimeric, cytosolic proteins with constitutively active RET kinase domain. Standard therapeutic regimens provide limited benefit for NSCLC patients with RET fusion-positive tumors, and the outcomes with immunotherapy in the these patients are generally poor. Selpercatinib (LOXO-292) and pralsetinib (BLU-667) are potent and selective inhibitors that target RET alterations, including fusions and mutations, irrespective of the tissue of origin. Recently, the results from the LIBRETTO-001 and ARROW clinical trials demonstrated significant clinical benefits with selpercatinib and pralsetinib respectively, in NSCLC patients with RET gene fusions, with tolerable toxicity profiles. These studies also demonstrated that these RET-TKIs crossed the blood brain barrier with significant activity. As has been observed with other TKIs, the emergence of acquired resistance may limit long-term efficacy of these agents. Therefore, understanding the mechanisms of resistance is necessary for the development of strategies to overcome them.
Insights
Biomarker-guided cancer therapy is revolutionizing treatment. New drugs targeting RET gene fusions, like selpercatinib and pralsetinib, show significant benefits for non-small cell lung cancer (NSCLC) patients.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- Biomarker-guided therapy is shifting cancer treatment paradigms.
- RET gene fusions are key drivers in 1-2% of non-squamous NSCLC, leading to poor outcomes with standard treatments.
Purpose of the Study:
- To review the efficacy and safety of novel RET inhibitors, selpercatinib and pralsetinib.
- To highlight the clinical benefits and challenges of targeting RET alterations in NSCLC.
Main Methods:
- Analysis of clinical trial data (LIBRETTO-001 and ARROW) for selpercatinib and pralsetinib.
- Review of RET inhibitors' mechanism of action, efficacy, and toxicity profiles.
Main Results:
- Selpercatinib and pralsetinib demonstrated significant clinical benefits in NSCLC patients with RET gene fusions.
- These RET inhibitors showed tolerable toxicity and activity across different tumor types, crossing the blood-brain barrier.
Conclusions:
- Selpercatinib and pralsetinib represent effective targeted therapies for RET fusion-positive NSCLC.
- Understanding acquired resistance mechanisms is crucial for optimizing long-term efficacy of these RET-targeted therapies.
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