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Response to Selpercatinib in a Patient With Recurrent Glioblastoma and RET Amplification
Cameron Czech1,2,3, Ashley Chen1,2,4, Katherine P Morgan1,2
1Department of Pharmacy, University of North Carolina Medical Center, Chapel Hill, North Carolina.
Abstract:
Glioblastoma (GBM) is a malignant central nervous system neoplasm that remains largely incurable. Limited treatment options currently exist after disease progression on standard-of-care first-line therapy. However, repurposing the use of approved therapies in patients with potentially targetable genomic alterations continues to be an emerging area of interest. This report presents the first description of a patient with isocitrate dehydrogenase wild-type GBM with an underlying RET amplification who demonstrated a near-complete response while receiving therapy with the RET inhibitor selpercatinib. The case highlights the excellent blood-brain barrier penetration of selpercatinib, as well as its potential role in the management of RET-amplified GBM. Larger biomarker-enriched studies are needed to confirm the results of this case report. Given the rare incidence of RET alterations in GBM, findings from this report can help guide and support optimal treatment strategies for patients with RET-altered GBM.
Insights
This case study shows a patient with glioblastoma (GBM) and RET amplification responded well to selpercatinib. This RET inhibitor may offer a new treatment option for specific GBM patients.
Area of Science:
- Neuro-oncology
- Genomic Medicine
- Drug Repurposing
Background:
- Glioblastoma (GBM) is an aggressive brain cancer with limited treatment options post-progression.
- Targeting specific genomic alterations in GBM is an emerging therapeutic strategy.
Observation:
- A patient with isocitrate dehydrogenase wild-type GBM and RET amplification was treated with selpercatinib.
- Selpercatinib demonstrated excellent blood-brain barrier penetration.
Findings:
- The patient achieved a near-complete response to selpercatinib therapy.
- This represents the first reported case of successful selpercatinib use in RET-amplified GBM.
Implications:
- Selpercatinib shows potential as a treatment for RET-amplified GBM.
- Biomarker-driven studies are needed to validate these findings in larger patient cohorts.
- This case informs potential treatment strategies for rare GBM subtypes.
