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mTOR is the cell's master growth controller, deciding whether to build proteins and divide. Rapamycin-like drugs clamp it down in kidney, breast and neuroendocrine cancers and in rare tumours driven by TSC gene loss. This dossier gathers the 3 products (2 approved), 7 trials, 3 pathways and 0 resistance routes in the corpus that involve it, with external identifiers so it can be joined to UniProt, ChEMBL, Open Targets and the rest of biology.
Rapalogues bind FKBP12 and allosterically inhibit mTORC1; feedback activation of AKT and incomplete 4E-BP1 inhibition explain modest single-agent activity. Stomatitis, hyperglycaemia, hyperlipidaemia and non-infectious pneumonitis are class effects.
| Cancer | Prevalence | Measure | Note | Source |
|---|---|---|---|---|
| Renal cell carcinoma | 8.2% | Targeted NGS, MTOR mutation in 184 everolimus-treated RCC patients (RECORD-3) | Voss 2019 (Clin Cancer Res); TSC1 6%, TSC2 4.4%, any PI3K-pathway alteration 44%; mutation status did not predict everolimus benefit | doi.org |
Approximate, population-level figures; the measure column says what was counted. Ranges show the midpoint as a bar.
| Modality | Approved | Phase 2 |
|---|---|---|
| Small molecule 3 |
| Trial | Setting | Result | Products | ||
|---|---|---|---|---|---|
| 3 | Positive | Kidney transplant recipients taking calcineurin inhibitors who had already had at least one cutaneous squamous cell carcinoma: substituting sirolimus for the calcineurin inhibitor against maintaining the existing treatment, with survival free of squamous cell carcinoma at two years as the primary endpoint | New cutaneous squamous cell carcinoma in 22 per cent of patients switched to sirolimus against 39 per cent continuing calcineurin inhibitors (relative risk 0.56, 95 per cent confidence interval 0.32 to 0.98), with 23 per cent stopping sirolimus for adverse events. | ||
| 3 | Planned | Efficacy and Safety of Inetetamab Combined With Rapamycin and Chemotherapy for HER2-positive Metastatic Breast Cancer Patients With Abnormal Activation of PI3K/Akt/mTOR Pathway After Progression on Trastuzumab. | - | ||
AMPECT NCT02494570 | 2 | Positive | Advanced malignant PEComa: nab-sirolimus (albumin-bound sirolimus) | Objective response 39% (independent review), durable, enriched in TSC2-mutant tumours; FDA approval November 2021. | |
| 2 | Active | A Phase 2 Multi-center Open-label Basket Trial of Nab-sirolimus for Adult and Adolescent Patients With Malignant Solid Tumors Harboring Pathogenic Inactivating Alterations in TSC1 or TSC2 Genes. | - | ||
| 2 | Recruiting | An Open-label, Multi-Center, Phase 2 Clinical Trial Evaluating Sapanisertib and Serabelisib (PIKTOR) With Paclitaxel, and a Substudy Evaluating PIKTOR With Paclitaxel Plus an Insulin-Suppressing Diet, in Patients With Advanced or Recurrent Endometrial Cancer | - | ||
| 2 | Recruiting | A Phase 2 Multi-center Open-label Trial of Nab-Sirolimus in Combination With Letrozole in Advanced or Recurrent Endometrioid Endometrial Cancer | - | ||
| 1/2 | Recruiting | Open-Label Umbrella Study to Evaluate Safety and Efficacy of Sapanisertib and Serabelisib (PIKTOR) in Various Combinations in Patients With HR+/HER2- Advanced or Metastatic Breast Cancer | - |
No recorded escape route names this target.
This KEGG map shows how cancer cells rewire the handling of choline, a nutrient used to build cell membranes, so that growth signals and membrane building feed each other. It matters because the resulting build-up of phosphocholine is visible on MR spectroscopy and PET scans and is one of the metabolic hallmarks of cancer.
Which nodes have drugs →This KEGG map shows how hepatitis viruses, alcohol and aflatoxin leave the liver with mutations in telomerase, TP53, Wnt/beta-catenin, PI3K/AKT/mTOR and the oxidative stress sensor NRF2, which together drive liver cancer. It matters because the map explains why liver cancer is treated mainly with angiogenesis blockers and immunotherapy rather than a single targeted drug.
Which nodes have drugs →KEGG's kidney cancer map shows how losing VHL lets the oxygen sensor HIF pile up and order new blood vessels (VEGF, PDGF), while MET and PI3K drive growth in other subtypes. Anti-VEGF drugs, HIF-2a inhibitors and immunotherapy all act on this circuit.
Which nodes have drugs →No companion diagnostic in the registry measures this target.
No model entry for this target yet; check the cancer entries on the models page.
No open questions recorded for this target yet. Suggest one.
Query for this target: (TITLE:"mTOR" OR ABSTRACT:"mTOR" OR TITLE:"MTOR" OR ABSTRACT:"MTOR") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about mTOR, not a curated reading list.
The dossier as machine-readable JSON, at /api/v1/dossiers/mtor.json: identifiers from HGNC, Ensembl, UniProt and ChEMBL, products with status, trials, pathways, hotspots, open questions and assays. The full entity record is in the open API at /api/v1/entities/mtor.json. Licence CC BY-NC 4.0.