Related Experiment Video
Updated: Jan 28, 2026

Establishment of Orthotopic Patient-derived Xenograft Models for Brain Tumors using a Stereotaxic Device
Published on: May 2, 2025
A patient-derived orthotopic xenograft (PDOX) nude-mouse model precisely identifies effective and ineffective
Zhiying Zhang1, Kaiwen Hu2, Tasuku Kiyuna3
1AntiCancer, Inc., 7917 Ostrow Street, San Diego, CA 92111, USA; Department of Surgery, University of California San Diego, CA 92103, USA; Department of Oncology, Dongfang Hospital, Beijing University of Chinese Medicine, Beijing, 100078, China.
Abstract:
Leiomyosarcoma is a rare and recalcitrant disease. Doxorubicin (DOX) is usually considered first-line treatment for this disease, but frequently is ineffective. In order to individualize therapy for this and other cancers, we have developed the patient-derived orthotopic xenograft (PDOX) mouse model. In the present study, we implanted a recurrent leiomyosarcoma from a resected tumor from the patient's thigh into the femoral muscle of nude mice. The following drugs were tested on the leiomyosarcoma PDOX model: DOX, the combination of gemcitabine (GEM) and docetaxel (DOC), trabectedin (TRA), temozolomide (TEM), pazopanib (PAZ) and olaratumab (OLA). Of these agents GEM/DOC, TRA and TEM were highly effective in the leiomyosarcoma PDOX model, the other agents, including first-line therapy DOX, were ineffective. Thus the leiomyosarcoma PDOX model could precisely distinguish effective and ineffective drugs, demonstrating the potential of the PDOX model for leiomyosarcoma treatment.
Insights
Patient-derived orthotopic xenograft (PDOX) models effectively identified effective treatments for leiomyosarcoma, a rare cancer. Gemcitabine/docetaxel, trabectedin, and temozolomide showed promise, unlike standard doxorubicin therapy.
Area of Science:
- Oncology
- Translational Cancer Research
- Preclinical Cancer Models
Background:
- Leiomyosarcoma is a rare and challenging cancer.
- Current first-line treatments like doxorubicin are often ineffective.
- Personalized cancer therapy requires accurate predictive models.
Purpose of the Study:
- To evaluate the efficacy of various chemotherapeutic agents in a patient-derived orthotopic xenograft (PDOX) model of leiomyosarcoma.
- To determine if the PDOX model can predict treatment response for leiomyosarcoma.
Main Methods:
- A recurrent leiomyosarcoma tumor was implanted into the femoral muscle of nude mice to create a PDOX model.
- The model was treated with doxorubicin, gemcitabine/docetaxel, trabectedin, temozolomide, pazopanib, and olaratumab.
- Treatment efficacy was assessed based on tumor response within the PDOX model.
Main Results:
- Gemcitabine/docetaxel, trabectedin, and temozolomide demonstrated high efficacy in the leiomyosarcoma PDOX model.
- Doxorubicin, pazopanib, and olaratumab were ineffective in this model.
- The PDOX model successfully differentiated between effective and ineffective treatments.
Conclusions:
- The patient-derived orthotopic xenograft model is a valuable tool for predicting treatment efficacy in leiomyosarcoma.
- This model can aid in individualizing therapy for patients with rare and recalcitrant leiomyosarcoma.
- Effective agents identified include gemcitabine/docetaxel, trabectedin, and temozolomide.
Related Concept Videos
Modeling in Therapy
Participant Modeling
Participant modeling involves therapists demonstrating calm and effective behaviors in...
Pharmacodynamics in Geriatric Patients: Effects of Age
Gene Therapy
Directing and Steric Effects in Disubstituted Benzene Derivatives
Uncertainty in Measurement: Accuracy and Precision
Accuracy and Precision

