Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Lung Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Lung Cancer. ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Lung Cancer: 1 is currently recruiting; 1 is active or not yet recruiting; 1 has completed; 5 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT04591431). The largest enrolment is 600 participants (NCT02432560). Registration activity spans 2006 to 2020. The literature layer holds 5 publications for this pair: 5 clinical trial publications. Publication years run from 2007 to 2017. These are primary trial reports rather than syntheses, so results have not yet been pooled or graded independently.
The RepurpOS disease-intelligence file for Lung Cancer ranks 465 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 is rare (<0.5% of cases); 5-year survival without treatment: <5% for advanced NSCLC; ~20% for stage I-II with surgery.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 44.0 (trials 14.0, literature 10.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 1 recruiting, 1 active / not yet recruiting, 1 completed, 5 other |
| Linked publications | 5 (5 clinical trial publications) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | ALK |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT04591431 | The Rome Trial From Histology to Target: the Road to Personalize Target Therapy and Immunotherapy 2020 | status unknown | Phase 2 | 400 |
| NCT00375245 | Rapamycin With Grapefruit Juice for Advanced Malignancies 2006 | completed | Phase 1 | 41 |
| NCT00807755 | Everolimus, Carboplatin, and Etoposide in Treating Patients With Small Cell Lung Cancer or Other Advanced Solid Tumors 2009 | terminated | Phase 1 | 5 |
| NCT02061800 | CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant 2013 | active, not recruiting | PHASE1, PHASE2 | 14 |
| NCT02432560 | Safety and Durability of Sirolimus for Treatment of LAM 2015 | recruiting | Not applicable | 600 |
| NCT02920450 | Study of Paclitaxel, Carboplatin, and PF-05212384 in Advanced or Metastatic NSCLC (UF-STO-LUNG-002) 2017 | terminated | PHASE1, PHASE2 | 3 |
| NCT00921310 | Temsirolimus and Pemetrexed for Recurrent or Refractory Non-Small Cell Lung Cancer 2009 | terminated | PHASE1, PHASE2 | 12 |
| NCT01175096 | Safety and Tolerability Profile of RAD001 Daily in Chinese Patients With Advanced Pulmonary Neuroendocrine Tumor 2010 | status unknown | PHASE1, PHASE2 | 20 |
Published literature
- Clinical trial publication A phase Ib trial of continuous once-daily oral afatinib plus sirolimus in patients with epidermal growth factor receptor mutation-positive non-small cell lung cancer and/or disease progression following prior erlotinib or gefitinibMoran T, Palmero R, Provencio M et al. · Lung cancer (Amsterdam, Netherlands) · 2017 · PMID 28625629
- Clinical trial publication Sirolimus plus prednisone for Erdheim-Chester disease: an open-label trialGianfreda D, Nicastro M, Galetti M et al. · Blood · 2015 · PMID 26041743
- Clinical trial publication Revisiting clinical trials using EGFR inhibitor-based regimens in patients with advanced non-small cell lung cancer: a retrospective analysis of an MD Anderson Cancer Center phase I populationWheler J, Falchook G, Tsimberidou AM et al. · Oncotarget · 2013 · PMID 23800712
- Clinical trial publication A Phase 1 clinical study of temsirolimus (CCI-779) in Japanese patients with advanced solid tumorsFujisaka Y, Yamada Y, Yamamoto N et al. · Japanese journal of clinical oncology · 2010 · PMID 20430774
- Clinical trial publication Phase I trial of sirolimus combined with radiation and cisplatin in non-small cell lung cancerSarkaria JN, Schwingler P, Schild SE et al. · Journal of thoracic oncology : official publication of the International Association for the Study of Lung Cancer · 2007 · PMID 17762343
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 ALK. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Lung Cancer?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Lung Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Lung Cancer. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Lung Cancer?
ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Lung Cancer: 1 is currently recruiting; 1 is active or not yet recruiting; 1 has completed; 5 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT04591431). The largest enrolment is 600 participants (NCT02432560). Registration activity spans 2006 to 2020.
What does the evidence show for Sirolimus in Lung Cancer?
Sirolimus has 1 completed trial and 5 linked publications for Lung Cancer. Completed trials may or may not have posted results; follow the NCT links to check. 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 ALK. 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.