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Precision treatment guided by patient-derived organoids-based drug testing for locally advanced thyroid cancer: a
Ziliang Guo1,2, Jiaye Liu1,2, Xinyue Zhang2,3
1Division of Thyroid Surgery, Department of General Surgery; Laboratory of Thyroid and Parathyroid Diseases, Frontiers Science Center for Disease-Related Molecular Network, West China Hospital, Sichuan University, Chengdu, 610000, China.
Purpose:
Locally advanced thyroid cancer (LATC) presents significant surgical challenges, with a high risk of incomplete resection and poor prognosis. Patient-derived organoids (PDOs) are a powerful tool to assess drug sensitivity at an individual level and to suggest new treatment options or re-challenge. This study aimed to evaluate the method's feasibility and efficacy as applied to patients with LATC.
Methods:
In this single arm, phase 2 study, we enrolled 75 patients with LATC. Biopsies from the primary tumor or metastatic site were cultured using organoid models. Sensitivity testing was performed by using PDOs with a panel of drugs with proven activity in phase II or III trials. At the discretion of the investigator considering toxicity, the drug with the highest relative activity was offered. The primary endpoint was the objective response rate (ORR).
Results:
Fifty-five patients received at least one dose of recommended drug and the primary endpoint, objective response was met in 18 patients with an overall ORR as 32.7% (95% CI 20.7-46.7). Based on the pre-defined subgroups of different histological subtypes, the ORR for patients with differentiated thyroid cancer, medullary thyroid cancer, anaplastic thyroid cancer were 32.6%, (95% CI 19.1-48.5), 16.7% (95% CI 0.4-64.1) and 50% (95% CI 11.8-88.2), respectively. The R0/R1 resection rate was 34.5% (19/55).
Conclusions:
This study is the first to validate the feasibility of PDOs and in vitro sensitivity testing for LATC. PDO-based neoadjuvant therapy holds promise in improving prognosis and providing surgical opportunities for these patients.
Trial Registration:
The study was registered at ClinicalTrials.gov (NCT06482086) on 06/25/2024.
Insights
Patient-derived organoids (PDOs) show promise for treating locally advanced thyroid cancer (LATC). This method demonstrated feasibility and efficacy, offering new therapeutic avenues for challenging LATC cases.
Area of Science:
- Oncology
- Translational Medicine
- Cancer Research
Background:
- Locally advanced thyroid cancer (LATC) poses significant surgical challenges, often leading to incomplete resection and poor prognoses.
- Patient-derived organoids (PDOs) offer a personalized approach to drug sensitivity testing, potentially guiding treatment strategies.
Purpose of the Study:
- To evaluate the feasibility and efficacy of using patient-derived organoids (PDOs) for drug sensitivity testing in patients with locally advanced thyroid cancer (LATC).
Main Methods:
- A single-arm, phase 2 study enrolled 75 patients with LATC.
- Tumor biopsies were used to establish organoid models for drug sensitivity testing.
- The drug with the highest relative activity was administered, with objective response rate (ORR) as the primary endpoint.
Main Results:
- Of 55 treated patients, 18 achieved an objective response, resulting in an overall ORR of 32.7%.
- Histological subtype ORRs varied: differentiated thyroid cancer (32.6%), medullary thyroid cancer (16.7%), and anaplastic thyroid cancer (50%).
- The R0/R1 resection rate was 34.5%.
Conclusions:
- This study validates the feasibility of PDOs and in vitro sensitivity testing for LATC.
- PDO-based neoadjuvant therapy shows potential for improving patient prognosis and enabling surgical resection in LATC.
