Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Breast Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Breast Cancer. ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Breast Cancer: 1 is currently recruiting; 4 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT04591431). It plans or enrolled 400 participants. Registration activity spans 2009 to 2023. The literature layer holds 3 publications for this pair: 1 systematic review and 2 clinical trial publications. Publication years run from 2010 to 2015. Because at least one synthesis-level source exists (systematic review, meta-analysis or Cochrane review), this pair has been assessed beyond single studies, although the synthesis may concern a different indication.
The RepurpOS disease-intelligence file for Breast Cancer ranks 1462 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: Spontaneous regression documented but rare (<0.5%); 5-year survival stage I: ~99%; stage IV: ~28% overall, improving with modern therapy.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 47.0 (trials 20.0, literature 7.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 1 recruiting, 0 active / not yet recruiting, 4 completed, 3 other |
| Linked publications | 3 (2 clinical trial publications, 1 systematic review) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | AKT1 |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT03659136 | The XENERA™ 1 Study Tests Xentuzumab in Combination With Everolimus and Exemestane in Women With Hormone Receptor Positive and HER2-negative Breast Cancer That Has Spread 2018 | completed | Phase 2 | 103 |
| NCT05983107 | Chidamide/Everolimus for PIK3CA Wild-type/Mutant HR+/HER2- Advanced Breast Cancer 2023 | recruiting | Phase 2 | 102 |
| NCT04591431 | The Rome Trial From Histology to Target: the Road to Personalize Target Therapy and Immunotherapy 2020 | status unknown | Phase 2 | 400 |
| NCT01088893 | Everolimus in Breast Cancer Patients After Pre-operative Chemotherapy 2009 | status unknown | Phase 2 | 50 |
| NCT02520063 | Combination of Letrozole, Everolimus and TRC105 in Postmenopausal Women With Hormone-Receptor Positive and Her2 Negative Breast Cancer 2015 | completed | PHASE1, PHASE2 | 15 |
| NCT01111825 | Temsirolimus Plus Neratinib for Patients With Metastatic HER2-Amplified or Triple Negative Breast Cancer 2010 | completed | PHASE1, PHASE2 | 99 |
| NCT02809404 | Dried Blood SPOT Analysis of Everolimus in Cancer Patients (SPOT-study) 2015 | completed | Not applicable | 75 |
| NCT03805399 | FUSCC Refractory TNBC Umbrella (FUTURE) 2018 | status unknown | PHASE1, PHASE2 | 140 |
Published literature
- Clinical trial publication Sirolimus and trastuzumab combination therapy for HER2-positive metastatic breast cancer after progression on prior trastuzumab therapyAcevedo-Gadea C, Hatzis C, Chung G et al. · Breast cancer research and treatment · 2015 · PMID 25687356
- Systematic review mTOR inhibitors in breast cancer: a systematic reviewZagouri F, Sergentanis TN, Chrysikos D et al. · Gynecologic oncology · 2012 · PMID 22967800
- Clinical trial publication Phase I, pharmacokinetic study of temsirolimus administered orally to patients with advanced cancerBuckner JC, Forouzesh B, Erlichman C et al. · Investigational new drugs · 2010 · PMID 19415181
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL, in this case via the biomarker or target AKT1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Breast Cancer?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Breast Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Breast Cancer. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Breast Cancer?
ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Breast Cancer: 1 is currently recruiting; 4 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT04591431). It plans or enrolled 400 participants. Registration activity spans 2009 to 2023.
What does the evidence show for Sirolimus in Breast Cancer?
Sirolimus has both completed registered trials and synthesis-level publications linked to Breast Cancer. That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL, in this case via the biomarker or target AKT1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-05. Page built 2026-10-07.