Use of Patient-Derived Organoids-Based Testing to Guide Neoadjuvant Treatment for Patients With Locally Advanced Thyroid Cancer
According to results from a phase 2 study, the use of patient-derived organoids and in vitro sensitivity testing shows promise in improving the prognosis for patients with locally advanced thyroid cancer.
Study authors wrote that surgery holds “a high risk of incomplete resection and poor prognosis" for patients with locally advanced thyroid cancer. The use of testing with patient-derived organoids can assess a patient’s individual drug sensitivity and suggest new treatment options or the opportunity to re-challenge.
This single arm, phase 2 study enrolled 75 patients with locally advanced thyroid cancer. Using patient-derived organoids, sensitivity testing was done for a panel of drugs with proven activity in phase 2 or 3 trials. The drug with the highest relative activity, at the investigator’s discretion considering toxicity, was offered. The primary end point of this study was objective response rate (ORR).
Of the 75 patients enrolled, 55 received at least 1 dose of the recommended drug. The primary end point was met, with an overall ORR of 32.7%. The ORR of patients with differentiated thyroid cancer was 32.6%, with medullary thyroid cancer was 16.7%, and with anaplastic thyroid canced was 50%. The R0/R1 resection rate was 34.5%
Study authors noted this is the first study “to validate the feasibility of [patient-derived organoids] and in vitro sensitivity testing for [locally advanced thyroid cancer].” They went on to note that patient-derived organoid-based neoadjuvant treatment “holds promise in improving prognosis and providing surgical opportunities for these patients.”
Source:
Guo Z, Liu J, Zhang X, et al. Precision treatment guided by patient-derived organoids-based drug testing for locally advanced thyroid cancer: A single arm, phase 2 study. Endocrine. Published online April 30, 2025. doi:10.1007/s12020-025-04240-9
