Lymphangioleiomyomatosis

Disease · 2 studies in the Atlas

LAM - a rare progressive cystic lung disease in women driven by inappropriate mTOR activation; sirolimus stabilizes lung function for as long as it is taken (MILES trial), with decline resuming after the drug is stopped.

A rare progressive lung disease.

MILES tested sirolimus and stabilised FEV1; EXIST-2 tested everolimus against renal angiomyolipoma, not the lung disease – a distinction routinely blurred.

In the pathway model

1 interaction in the curated mTOR pathway model. Each row is a claim with the evidence behind it; the evidence code is the kind of study, not its quality.

Interaction	Mechanism	Evidence	Studies
Rapamycin inhibits Lymphangioleiomyomatosis clinical-outcome · on the map table	In a clinical trial, rapamycin stabilised lung function in a rare lung disease while patients kept taking it – the decline came back once they stopped. The MILES trial: sirolimus stabilised lung function (FEV1) in lymphangioleiomyomatosis while patients took it, and decline resumed after stopping. This - not the everolimus angiomyolipoma trial - is the evidence that mTOR inhibition treats LAM lung disease. The MILES trial: sirolimus stabilised lung function (FEV1) in lymphangioleiomyomatosis while patients took it, and decline resumed after stopping. This - not the everolimus angiomyolipoma trial - is the evidence that mTOR inhibition treats LAM lung disease.	H	MCC2011

Evidence at a glance

Evidence	What it means	Studies
H	Human study	2

Studies

Year	Evidence	Study
2013	H	Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicentre, randomised, double-blind, placebo-controlled trial BIS2013 The companion phase 3 RCT (n=118) to EXIST-1, targeting kidney tumors (angiomyolipomas) in tuberous sclerosis and LAM. Everolimus shrank them by >=50% in 42% of patients versus 0% on placebo. Together EXIST-1 and -2 sealed everolimus as a disease-modifying therapy across multiple TSC tumor types.
2011	H	Efficacy and safety of sirolimus in lymphangioleiomyomatosis (MILES) MCC2011 A landmark placebo-controlled RCT (n=89) - the first to show that rapamycin (sirolimus) benefits a human lung disease. In LAM, lung function normally declines relentlessly; sirolimus STOPPED that decline while patients took it (and it resumed after stopping). Randomised human evidence that mTOR inhibition can suspend progression of this disease; the benefit did not persist after withdrawal.

Related entities

mTORC1 2 Rapamycin 1 mTOR 1 Everolimus 1 Renal angiomyolipoma 1 Tuberous sclerosis complex 1

Cite this page

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Barton, O. (2026). Lymphangioleiomyomatosis [Entity page]. In Oliver's mTOR Atlas [Data set]. https://mtor-atlas.org/disease/lymphangioleiomyomatosis/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_disease_lymphangioleiomyomatosis,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}: Lymphangioleiomyomatosis},
  howpublished = {Entity page in a data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/disease/lymphangioleiomyomatosis/},
  doi          = {10.5281/zenodo.22059963}
}

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