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Updated: Aug 19, 2025
Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
Exceptional response to neoadjuvant targeted therapy with the selective RET inhibitor selpercatinib in
Karin G Schrenk1, Wolfram Weschenfelder2, Christian Spiegel2
1Department of Hematology and Internal Oncology, Clinic of Internal Medicine II, University Hospital Jena, Am Klinikum 1, 07747, Jena, Germany. karin.schrenk@med.uni-jena.de.
Abstract:
With the increasing use of next-generation sequencing, highly effective targeted therapies have been emerging as treatment options for several cancer types. Recurrent gene-fusions have been recognized in sarcomas; however, options for targeted therapy remain scarce. Here, we describe a case of a sarcoma, associated with a RET::TRIM33-fusion gene with an exceptional response to a neoadjuvant therapy with the selective RET inhibitor selpercatinib. Resected tumor revealed subtotal histopathologic response. This is the first report of successful targeted therapy with selpercatinib in RET-fusion-associated sarcomas. As new targeted therapies are under development, similar treatment options may become available for sarcoma patients.
Insights
A novel targeted therapy using selpercatinib showed exceptional results in a patient with RET-fusion-associated sarcoma. This marks the first successful use of selpercatinib for this rare cancer, offering hope for future treatments.
Area of Science:
- Oncology
- Genetics
- Pharmacology
Background:
- Next-generation sequencing is driving the development of targeted cancer therapies.
- Recurrent gene fusions are implicated in sarcomas, but targeted treatment options are limited.
- The RET gene is a potential target in specific sarcoma subtypes.
Observation:
- A patient with sarcoma presented with a rare RET::TRIM33-fusion gene.
- The patient received neoadjuvant therapy with selpercatinib, a selective RET inhibitor.
- An exceptional clinical response was observed following the treatment.
Findings:
- The sarcoma exhibited a subtotal histopathologic response after selpercatinib therapy.
- This case represents the first successful application of selpercatinib in RET-fusion-associated sarcomas.
- Selpercatinib demonstrated significant efficacy in treating this specific genetic alteration in sarcoma.
Implications:
- This study highlights the potential of targeted therapies for rare sarcoma subtypes.
- Selpercatinib offers a promising new treatment avenue for patients with RET-fusion sarcomas.
- Further research into targeted therapies for sarcomas is warranted, potentially improving patient outcomes.
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