TABLE 1.
Selected examples of therapeutic strategies assessed using PDX models with details of the cancer type, specific target(s), and mouse models used.
| Cancer type | Drug or combination | Target | Mouse | References |
|---|---|---|---|---|
| Acute myelogenous leukemia | CSL362 monoclonal antibody with cytarabine and daunorubicin | CD123 | NSG | 71 |
| Acute myelogenous leukemia | Brequinar | Dihydroorotate dehydrogenase | SCID | 72 |
| Acute myelogenous leukemia | R406 | Syk | NOG | 73 |
| Acute myelogenous leukemia | Selinexor (KPT‐330) | XPO1 | NSG | 74 |
| Multiple myeloma | BI‐505 antibody | ICAM‐1 | SCID | 75 |
| B cell acute lymphoblastic leukemias | CHZ868 (JAK2 inhibitor) and dexamethasone | JAK2 | NSG | 76 |
| BRAF mutant cancer | PD0325901 | MEK | Nude | 77 |
| Breast carcinoma | Dinaciclib | CDK | NOD/SCID | 78 |
| Breast carcinoma | FRAX597 (PAK2 inhibitor) and fulvestrant | PAK2 and ER | NSG | 41 |
| Breast carcinoma | ICG‐001 (Wnt inhibitor) and doxorubicin | Wnt signaling | NSG | 79 |
| Breast carcinoma | decitabine | DNA methyltransferases | NOD/SCID | 80 |
| Cholangiocarcinoma | Ponatinib, dovitinib, and BGJ398 | FGFR | NSG | 81 |
| Chordomas | Erlotinib and gefitinib | EGFR | Nude | 82 |
| Colon cancer | Cetuximab and pimasertib | EGFR/MEK | NOD/SCID | 83 |
| Colon cancer | FP3 | VEGF | Nude | 84 |
| Colon cancer | Cetuximab and bevacizumab | EGFR/VEGF | Nude | 85 |
| Colorectal cancer | DEL‐22379 | Erk | NOD/SCID | 86 |
| Colorectal cancer | Anti‐RSPO3 (antibody) | RSPO3 | Nude | 87 |
| Esophageal squamous cell carcinoma | Trastuzumab | Her2 | Nude and SCID | 88 |
| Gastric cancer | Luteolin | cMet | Nude | 89 |
| Gastric cancer | Trastuzumab and cetuximab | Her2/EGFR | Nude | 81 |
| Gastric cancer | AZD5363 | AKT | Nude | 90 |
| Lung adenocarcinoma | Cetuximab | EGFR | NOD/SCID | 91 |
| Lung squamous cell carcinoma | BGJ398 and cisplatin | AKT and ERK | NSG | 92 |
| Lung cancer | Erlotinib and thalidomide | EGFR, TNF, and NF‐kB | NOD/SCID | 93 |
| Lung cancer | BDA‐366 | Bcl2 BH4 domain | Nude | 94 |
| Lymphoma | Pyruvinium pamoate | Glutathione | NOG | 95 , 96 |
| Medulloblastoma | Fingolimod (FTY720) | – | Nude | 96 |
| Melanoma | Salmonella A1‐R | – | Nude | 97 |
| Melanoma | Karonudib (TH1579) | MTH1 | NOG | 98 |
| Melanoma | Vemurafenib and fatostatin | SREBP‐1 | Nude | 99 |
| Melanoma | TH287 and TH588 | MTH1 | NOG | 100 |
| Multiple cancers | CFI‐400945, inhibitor | PLK4 | NSG and SCID | 101 |
| Melanoma | CCT196969, CCT241F161 | pan‐RAF and SFKs | Nude | 102 |
| Multiple myeloma | P5091 | USP7 | SCID | 103 |
| Multiple myeloma and solid tumors | CB‐5083 | p97 | Nude and SCID‐Beige | 26 |
| Neuroblastoma | MLN8237 and ABT‐199 | Aurora kinase and BCL‐2 | SCID | 104 |
| Non‐Hodgkin lymphoma | Anti‐CD47 antibody and rituximab | CD47 | NSG | 105 |
| Chronic myelogenous leukemia | NSC23766 | Rac | NOD/SCID | 106 |
| Pancreatic adenocarcinoma | BKM120 | PI3K inhibitor | NSG | 107 |
| Pancreatic cancer | IGF1‐IONP‐Dox | IGF1R | Nude mice/SCID | 108 |
| Pancreatic cancer | Trametinib and dasatinib | MEK/Tyrosine kinase Src | NSG | 109 |
| Pancreatic cancer | Phenformin | Mitochondrial complex I | Nude | 110 |
| Prostate cancer | EPI‐001 | Androgen receptor NTD domain | NOD/SCID | 111 |
| Prostate cancer | Bicalutamide | Androgen | SCID | 112 |
| Renal cell carcinoma | PT2399 | HIF‐2 | Nude | 113 |
| Sarcoma | Salmonella A1‐R and doxorubicin | – | Nude | 114 |
| Small cell lung cancer | GSK2879552 | LSD1 | Nude | 115 |
| Pancreatic cancer | Trametinib and MRTX1133 | KRAS | Nude | 116 |
| Mesothelioma | Gemcitabine | _ | Nude | 117 |
| Pancreatic cancer | Gemcitabine, 5‐fluorouracil | _ | Nude | 118 |
| Mesonephric adenocarcinoma | Paclitaxel, cisplatin | _ | Nude | 119 |