Patient-derived xenograft models: Current status, challenges, and innovations in cancer research
Despite advancing therapeutic treatments, cancer remains the leading cause of death worldwide, with most of its patients developing drug resistance and recurrence after initial treatment. Therefore, incorporating preclinical models that mimic human cancer biology and drug responses is essential for...
Saved in:
| Main Authors: | , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
KeAi Communications Co., Ltd.
2025-09-01
|
| Series: | Genes and Diseases |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2352304225000091 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1849710171514732544 |
|---|---|
| author | Minqi Liu Xiaoping Yang |
| author_facet | Minqi Liu Xiaoping Yang |
| author_sort | Minqi Liu |
| collection | DOAJ |
| description | Despite advancing therapeutic treatments, cancer remains the leading cause of death worldwide, with most of its patients developing drug resistance and recurrence after initial treatment. Therefore, incorporating preclinical models that mimic human cancer biology and drug responses is essential for improving treatment efficacy and prognosis. Patient-derived xenograft (PDX) models, as a promising and reliable preclinical trial platform, retain key features of the original tumor such as gene expression profiles, histopathological features, drug responses, and molecular signatures more faithfully compared with traditional tumor cell line models and cell line-derived xenograft models. Their significant advantages have been the preferred choice in cancer research, especially demonstrating remarkable potential in drug development, clinical combination therapy, and precision medicine. However, the successful construction and effective application of PDX models still face several challenges. In this review, we summarize the details of constructing PDX models and the drivers affecting their success rates, which will provide some theoretical basis for subsequent model optimization. In the meantime, we delineate the strengths and weaknesses of various mature PDX models and other developing preclinical models, including PDX-derived models, organoids, and genetically engineered models. Moreover, we highlight the challenges of newly developed technologies on the PDX models. Finally, we emphasize the innovative usage of PDX models in a variety of cancer studies and offer insights into their prospects. |
| format | Article |
| id | doaj-art-aa9be44a69c4420180263315fbc6d99c |
| institution | DOAJ |
| issn | 2352-3042 |
| language | English |
| publishDate | 2025-09-01 |
| publisher | KeAi Communications Co., Ltd. |
| record_format | Article |
| series | Genes and Diseases |
| spelling | doaj-art-aa9be44a69c4420180263315fbc6d99c2025-08-20T03:15:02ZengKeAi Communications Co., Ltd.Genes and Diseases2352-30422025-09-0112510152010.1016/j.gendis.2025.101520Patient-derived xenograft models: Current status, challenges, and innovations in cancer researchMinqi Liu0Xiaoping Yang1Key Laboratory of Chemical Biology & Traditional Chinese Medicine Research of Ministry of Education, Key Laboratory of Study and Discovery of Small Targeted Molecules of Hunan Province, Engineering Research Center of Reproduction and Translational Medicine of Hunan Province, Key Laboratory of Protein Chemistry and Developmental Biology of Fish of Ministry of Education, Department of Pharmacy, School of Medicine, Hunan Normal University, Changsha, Hunan 410013, ChinaCorresponding author. School of Medicine, Hunan Normal University, Changsha, Hunan 410013, China.; Key Laboratory of Chemical Biology & Traditional Chinese Medicine Research of Ministry of Education, Key Laboratory of Study and Discovery of Small Targeted Molecules of Hunan Province, Engineering Research Center of Reproduction and Translational Medicine of Hunan Province, Key Laboratory of Protein Chemistry and Developmental Biology of Fish of Ministry of Education, Department of Pharmacy, School of Medicine, Hunan Normal University, Changsha, Hunan 410013, ChinaDespite advancing therapeutic treatments, cancer remains the leading cause of death worldwide, with most of its patients developing drug resistance and recurrence after initial treatment. Therefore, incorporating preclinical models that mimic human cancer biology and drug responses is essential for improving treatment efficacy and prognosis. Patient-derived xenograft (PDX) models, as a promising and reliable preclinical trial platform, retain key features of the original tumor such as gene expression profiles, histopathological features, drug responses, and molecular signatures more faithfully compared with traditional tumor cell line models and cell line-derived xenograft models. Their significant advantages have been the preferred choice in cancer research, especially demonstrating remarkable potential in drug development, clinical combination therapy, and precision medicine. However, the successful construction and effective application of PDX models still face several challenges. In this review, we summarize the details of constructing PDX models and the drivers affecting their success rates, which will provide some theoretical basis for subsequent model optimization. In the meantime, we delineate the strengths and weaknesses of various mature PDX models and other developing preclinical models, including PDX-derived models, organoids, and genetically engineered models. Moreover, we highlight the challenges of newly developed technologies on the PDX models. Finally, we emphasize the innovative usage of PDX models in a variety of cancer studies and offer insights into their prospects.http://www.sciencedirect.com/science/article/pii/S2352304225000091Cancer modelsCo-clinical trialsDrug screeningPDX modelsPrecision medicine |
| spellingShingle | Minqi Liu Xiaoping Yang Patient-derived xenograft models: Current status, challenges, and innovations in cancer research Genes and Diseases Cancer models Co-clinical trials Drug screening PDX models Precision medicine |
| title | Patient-derived xenograft models: Current status, challenges, and innovations in cancer research |
| title_full | Patient-derived xenograft models: Current status, challenges, and innovations in cancer research |
| title_fullStr | Patient-derived xenograft models: Current status, challenges, and innovations in cancer research |
| title_full_unstemmed | Patient-derived xenograft models: Current status, challenges, and innovations in cancer research |
| title_short | Patient-derived xenograft models: Current status, challenges, and innovations in cancer research |
| title_sort | patient derived xenograft models current status challenges and innovations in cancer research |
| topic | Cancer models Co-clinical trials Drug screening PDX models Precision medicine |
| url | http://www.sciencedirect.com/science/article/pii/S2352304225000091 |
| work_keys_str_mv | AT minqiliu patientderivedxenograftmodelscurrentstatuschallengesandinnovationsincancerresearch AT xiaopingyang patientderivedxenograftmodelscurrentstatuschallengesandinnovationsincancerresearch |