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

Everolimus Renal angiomyolipoma

Everolimus inhibits Renal angiomyolipoma.

In another trial, everolimus shrank kidney growths in patients with tuberous sclerosis or a related lung disease, working in 42% of patients versus 0% on placebo.

EXIST-2 tested everolimus against renal angiomyolipoma in patients with tuberous sclerosis or sporadic LAM, and shrank those lesions by >=50% in 42% versus 0% on placebo.

The conditions it holds under

Scope: the endpoint was the kidney lesion, not LAM lung disease, which is why this arrow lands on the angiomyolipoma and not on LAM. The evidence that mTOR inhibition treats the lung disease itself is the MILES trial with sirolimus - see the RAPA-LAM edge.

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
2013 H Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicentre, randomised, double-blind, placebo-controlled trialBIS2013 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.
2026 H Tumor fat composition predicts response to everolimus and supports low-dose therapy in TSC-associated renal angiomyolipomas: a multicenter prospective studyGAO2026B In a multicenter prospective cohort of 183 TSC-associated renal angiomyolipoma patients, everolimus reduced tumor volume by >=50% in 44% of patients at 3 months and 83% at 6 months; fat-poor lesions responded significantly better than fat-rich ones. No significant difference in response was detected between the low-dose and standard-dose groups in an exploratory, non-randomised subset, and oral mucositis was less frequent on the low dose; tumor regrowth occurred after treatment discontinuation.

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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