Table 3.
Potential therapeutic agents and their targets in different cancer types in PDX models.
| Tumor type | Animal strain | Implantation site | Gene target | Drug | Application | Reference | |
|---|---|---|---|---|---|---|---|
| Lung cancer | EGFR-mutant NSCLC | NSG mice | Subcutaneous, subrenal capsule | HER3 | Osimertinib, HER3-DXd | Evaluation of combination therapy with osimertinib and HER3-DXd | Haikala et al202 |
| SCLC | NSG mice | Subcutaneous | UBA1 | TAK-243 | Evaluation of TAK-243 as mono- and combination therapy in SCLC | Majeed et al203 | |
| Gastric cancer | Balb/c-nu mice | Subcutaneous | JAK2, STAT3 | CYT997 | Confirmation that CYT997 may be a potential anti-tumor drug | Cao et al204 | |
| NOD/SCID mice | Subcutaneous | CCAT5 | si-CCAT5, oxaliplatin | Uncovering the mechanism of STAT3 signaling regulated by wnt signaling | Liu et al.205 | ||
| Colorectal cancer | NOD/SCID mice | Subcutaneous | PIM1, FGFR1 | HCI-48 | Describing the anti-tumor effects of HCI-48 on the dual targeting of PIM1 and FGFR1 | Yin et al206 | |
| NOD/SCID mice | Subcutaneous | GART | Pemetrexed | Evidence for the function of GART and the role of the GART/RUVBL1/β-catenin signaling axis in promoting colorectal cancer stemness | Tang et al207 | ||
| Esophageal carcinoma | ESCC | NOD/SCID mice | Subcutaneous | eEF2 | Toosendanin | Revealed eEF2 as a potential therapeutic target for ESCC | Jia et al208 |
| Liver cancer | Hepatoblastoma | Nude mice | Subcutaneous | ALCD | Olaparib | Explained the regulatory mechanism of ALCD in hepatoblastoma | Johnston et al209 |
| HCC | NOD/SCID mice | Liver | cDCBLD2, TOP2A | Sorafenib | Provides a potential strategy for targeting cDCBLD2 or TOP2A to overcome sorafenib resistance in patients with HCC | Ruan et al210 | |
| Pancreatic cancer | PDAC | Balb/c-nu mice | Orthotopic, intrasplenic | CD73 | Diclofenac | Diclofenac may be an effective treatment for metastatic PDAC | Liu et al211 |
| Breast cancer | Breast cancer | NSG mice | Mammarian fat pad | FGFRs | AZD4547, BLU9931 | The potential of specific FGFRs as precision therapeutic targets was identified | Chew et al212 |
| ER-negative postmenopausal breast cancer | NSG mice | Mammarian fat pad | RANK | Denosumab | RANK protein expression is an independent biomarker of poor prognosis in patients with estrogen receptor-negative postmenopausal breast cancer | Ciscar et al213 | |
| Ovarian cancer | HGSOC | NOD/SCID mice | Subcutaneous | IGFBP2 | Gold nanoparticles | Reported key signaling axes for gold nanoparticles' therapeutic role | Hossen et al214 |
| TP53 mutant ovarian cancer | Nude mice | Subcutaneous | IRE1α | AZD1775 | Mechanism of UPR signaling network IRE1α in TP53 mutant ovarian cancer | Xiao et al215 | |
| Cervical cancer | Cervical cancer | NOD/SCID mice | Subcutaneous | ZNF275 | Triciribine, cisplatin | ZNF275 is revealed to be a potential predictor of cervical cancer treatment | Ye et al216 |
| Neuroblastoma | High-risk neuroblastoma | NMRI nude mice, NSG mice | Subcutaneous, adrenal glands | KSP | ARRY-520 | KSP inhibition may be a promising treatment strategy for neuroblastoma | Hansson et al217 |
| Osteosarcoma | Chemotherapy-resistant and metastatic osteosarcoma | NSG mice | Subcutaneous | β-catenin/ALDH1 | Tegavivint | Evaluate tegavivint in chemotherapy-resistant and metastatic osteosarcoma in chemotherapy-resistant and metastatic osteosarcoma | Nomura et al218 |
| Prostate cancer | NEPC | SCID mice | Subcutaneous | MYCN, CDK5, RB1, E2F1 | Enzalutamide, olaparib, dinaciclib | Elucidating the mechanism of action for PARP inhibition in the treatment of NEPC | Liu et al219 |
| Bladder cancer | NSG mice | Subcutaneous | ErbB3 | Seribantumab | ErbB3 phosphorylation may be a potential therapeutic strategy for bladder cancer | Steele et al220 | |
| Renal cell carcinoma | NSG mice | Subcutaneous | ERK | Cabozantinib, sapanisertib | The potential of the combination therapeutic approach of cabozantinib and sapanisertib was emphasized | Wu et al221 | |
| HNC | HNSCC | Nude mice | Subcutaneous | RAC1, RAC3 | EHOP-016, cetuximab | Uncovering biomarkers and mechanisms to overcome acquired cetuximab resistance | Yao et al222 |
| Gliomas | Glioblastoma | Balb/c-nu mice | Mouse brain | PTRF | Temozolomide | Revealed PTRF as a biomarker for the prognosis of glioblastoma patients after temozolomide treatment | Yang et al223 |
Note: ESCC, esophageal squamous cell carcinoma; SCLC, small cell lung cancer; HCC, hepatocellular carcinoma; PDAC, pancreatic ductal adenocarcinoma; HGSOC, high-grade serous ovarian cancer; IGFBP2, insulin growth factor binding protein 2; IRE1α, inositol-required enzyme 1α; NEPC, neuroendocrine prostate cancer; HNC, head and neck cancer; HNSCC, head and neck squamous cell carcinomas; HER3, human epidermal growth factor receptor 3; UBA1, ubiquitin-like modifier activating enzyme 1; Jak2, Janus kinase 2; STAT3, signal transducer and activator of transcription 3; CCAT5, colon cancer-associated transcript 5; FGFR1, fibroblast growth factor receptor 1; PIM1, moloney-murine leukemia 1; GART, glycinamide ribonucleotide transformylase; RUVBL1, RuvB like AAA ATPase 1; eEF2, eukaryotic elongation factor-2; AICD, amyloid precursor protein intra-cellular domain; TOP2A, type IIA topoisomerase; DCBLD2, discoidin, CUB, and LCCL domain-containing protein 2; CD73, cluster of differentiation 73; RANK, receptor activator of nuclear factor-κB; ZNF275, zinc finger protein 275; KSP, kidney-specific cadherin; ALDH1, aldehyde dehydrogenase 1; CDK5, cyclin-dependent kinase 5; RB1, retinoblastoma protein 1; E2F1, E2F transcription factor 1; ERBB3, Erb-b2 receptor tyrosine kinase 3; ERK, extracellular signal-regulated kinase; RAC1/3, Ras-related C3 botulinum toxin substrate 1/3; PTRF, polymerase I and transcript release factor.