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Pilot Study of a Next-Generation Sequencing-Based Targeted Anticancer Therapy in Refractory Solid Tumors at a Korean
Hyung Soon Park1,2, Sun Min Lim3, Sora Kim4
1Department of Pharmacology and Brain Korea 21 Plus Project for Medical Sciences, Yonsei University College of Medicine, Seoul, Korea.
Abstract:
We evaluated the preliminary efficacy and feasibility of a next-generation sequencing (NGS)-based targeted anticancer therapy in refractory solid tumors at a Korean institution. Thirty-six patients with advanced cancer underwent molecular profiling with NGS with the intent of clinical application of available matched targeted agents. Formalin-fixed paraffin-embedded (FFPE) tumors were sequenced using the Comprehensive Cancer Panel (CCP) or FoundationOne in the Clinical Laboratory Improvement Amendments-certified laboratory in the USA. Response evaluations were performed according to RECIST v1.1. Four specimens did not pass the DNA quality test and 32 specimens were successfully sequenced with CCP (n = 31) and FoundationOne (n = 1). Of the 32 sequenced patients, 10 (31.3%) were ≤40 years. Twelve patients (37.5%) had received ≥3 types of prior systemic therapies. Of 24 patients with actionable mutations, five were given genotype-matched drugs corresponding to actionable mutations: everolimus to PIK3CA mutation in parotid carcinosarcoma (partial response) and tracheal squamous cell carcinoma (stable disease; 21% reduction), sorafenib to PDGFRA mutation in auditory canal adenocarcinoma (partial response), sorafenib to BRAF mutation in microcytic adnexal carcinoma (progressive disease), and afatinib to ERBB2 mutation in esophageal adenocarcinoma (progressive disease). Nineteen of 24 patients with actionable mutations could not undergo targeted therapy based on genomic testing because of declining performance status (10/24, 41.7%), stable disease with previous treatment (5/24, 20.8%), and lack of access to targeted medication (4/24, 16.7%). NGS-based targeted therapy may be a good option in selected patients with refractory solid tumors. To pursue this strategy in Korea, lack of access to clinical-grade NGS assays and a limited number of genotype-matched targeted medications needs to be addressed and resolved.
Insights
Next-generation sequencing (NGS) for targeted cancer therapy shows promise in refractory solid tumors. Challenges in Korea include access to clinical-grade NGS and targeted medications, requiring further development for broader application.
Area of Science:
- Oncology
- Genomics
- Precision Medicine
Background:
- Refractory solid tumors present significant treatment challenges.
- Molecular profiling using next-generation sequencing (NGS) offers potential for personalized anticancer therapies.
- Evaluating the efficacy and feasibility of NGS-based targeted therapy is crucial for advanced cancer patients.
Purpose of the Study:
- To assess the preliminary efficacy and feasibility of NGS-based targeted anticancer therapy in Korean patients with refractory solid tumors.
- To identify actionable mutations and evaluate the clinical application of matched targeted agents.
Main Methods:
- Thirty-six patients with advanced solid tumors underwent molecular profiling using NGS (Comprehensive Cancer Panel or FoundationOne).
- Formalin-fixed paraffin-embedded (FFPE) tumor specimens were sequenced.
- Response evaluation was performed using RECIST v1.1 criteria.
Main Results:
- Thirty-two specimens were successfully sequenced, revealing actionable mutations in 24 patients.
- Five patients received genotype-matched targeted therapy, with partial responses observed in two cases (parotid carcinosarcoma, auditory canal adenocarcinoma).
- Barriers to targeted therapy included declining performance status (41.7%), stable disease (20.8%), and lack of medication access (16.7%).
Conclusions:
- NGS-based targeted therapy is a viable option for select patients with refractory solid tumors.
- Implementation in Korea requires addressing limitations in clinical-grade NGS assay access and targeted medication availability.
- Further research and infrastructure development are needed to optimize this precision medicine approach.
