# Clinical activity of mTOR inhibition with sirolimus in malignant perivascular epithelioid cell tumours

Source: https://onco.cc/key-papers/paper-wagner-sirolimus-pecoma-jco-2010/  
OnCo record `paper-wagner-sirolimus-pecoma-jco-2010` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

This report of three patients with malignant PEComa responding to oral sirolimus was the first evidence that these tumours, which share TSC1/TSC2 loss with tuberous sclerosis, depend on the mTOR pathway.

## Summary

Case series of three patients with metastatic malignant PEComa treated with sirolimus, all showing radiological responses, with molecular analysis of tumour tissue demonstrating loss of TSC2 and activation of mTORC1 signalling.

## Fields

- Kind: Key paper
- Last checked: 2026-09-17
- Journal: Journal of Clinical Oncology
- Year: 2010
- DOI: 10.1200/JCO.2009.25.2981
- Authors: Wagner AJ, Malinowska-Kolodziej I, Morgan JA, et al.
- Findings: Radiological responses in all three patients treated with sirolimus.; TSC2 loss and mTORC1 activation in tumour tissue.
- What it means: mTOR inhibition became the therapeutic strategy for PEComa, culminating in the AMPECT trial and approval of nab-sirolimus.
- Caveats: Three patients; hypothesis-generating.

## Sources

- J Clin Oncol 2010: https://doi.org/10.1200/JCO.2009.25.2981
- PubMed: https://pubmed.ncbi.nlm.nih.gov/20048174/

## Connected records

- cancers: [Perivascular epithelioid cell tumour (PEComa)](https://onco.cc/cancers/pecoma/)
- journals: [Journal of Clinical Oncology](https://onco.cc/journals/jco/)

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