Oliver's mTOR Atlas Evidence Platform
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Pathway link RAPA-LAM

Rapamycin Lymphangioleiomyomatosis

Rapamycin inhibits Lymphangioleiomyomatosis.

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 conditions it holds under

Benefit is suppressive: lung function declined again after withdrawal.

Time it takes
long exposure (days to weeks)
Step
indirect (through steps not drawn here)
Measured in
human
Reaches people?
shown in people
How settled
settled in the literature

Studies behind it

YearEvidenceStudy
2011 H Efficacy and safety of sirolimus in lymphangioleiomyomatosis (MILES)MCC2011 MILES was a 12-month placebo-controlled trial of sirolimus in 89 women with lymphangioleiomyomatosis (LAM), a rare lung disease driven by overactive mTOR. On placebo, lung function (FEV1) fell by about 12 ml a month. On sirolimus it held steady (+1 ml a month), a 153 ml difference over the year. In the next year nobody took the drug, and the sirolimus group's decline resumed in parallel with placebo, so the benefit lasted only while the drug was taken. Walking distance and gas-transfer capacity did not improve. Boundary: patients had fairly advanced disease (average FEV1 about 48% of predicted), many dropped out of the observation year, and mouth ulcers and rashes may have revealed who was on the drug. Sirolimus was held at a blood level of 5 to 15 ng/ml, so this was continuous dosing, not intermittent.

Points the other way

No study in the Atlas contradicts this link. That means none in this curated set, not none in the literature.

Who has checked this

Checked by
No researcher outside the project has checked this link yet.
Curator sign-off
Not signed off yet. This link was drafted with AI help and has not been reviewed link by link; treat it as a proposal until it is.
Curated by
Oliver Barton, with AI help for drafting; see how links are checked.

“Inhibits” describes the effect on the target's activity, not on how much of it there is.

“How settled” describes how far researchers in the field agree. It is not the same as the sign-off above, which only says whether a named curator has checked this page.

If this link rests on your own work: a click on “This looks right” below, or a short note through “Report an error”, helps us more than anything else on this page.

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