Oliver's mTOR Atlas Evidence Platform
Reading level
Mode

Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicentre, randomised, placebo-controlled phase 3 trial

Franz DN, Belousova E, Sparagana S, Bebin EM, Frost M, Jozwiak S · 2013 · Lancet · Atlas ID FRA2013

What this study shows

Phase 3 RCT (n=117) in tuberous sclerosis, the disease where mTOR is stuck ON by a genetic fault. Everolimus shrank brain tumors (SEGA) by >=50% in 35% of patients versus 0% on placebo. Because the underlying cause here is direct mTOR overactivation, this is arguably the cleanest randomised human evidence that blocking mTOR works in a genetically defined mTORopathy -- which does not extend to mTOR inhibition in people without such a mutation.

Abstract

ShowHide

Tuberous sclerosis complex is a genetic disorder leading to constitutive activation of mammalian target of rapamycin (mTOR) and growth of benign tumours in several organs. In the brain, growth of subependymal giant cell astrocytomas can cause life-threatening symptoms--eg, hydrocephalus, requiring surgery. In an open-label, phase 1/2 study, the mTOR inhibitor everolimus substantially and significantly reduced the volume of subependymal giant cell astrocytomas.

Read the full abstract on PubMed →

At a glance

Evidence type H Human study Marked H because it is direct evidence from a human clinical trial or human cohort; the code names the kind of study, not its quality -- a small, well-run trial is still H.
Study type2 - Human Clinical Trial
Model systemHumans, phase 3 RCT (n=117, TSC)
JournalLancet
Year2013
Peer reviewedYes
Record last updated2026-07-29
SourceDOI 10.1016/S0140-6736(12)61134-9 · PMID 23158522

Extracted findings

InterventionEverolimus
TargetmTOR
ModelHuman – phase 3 RCT (EXIST-1, n=117, TSC)
EffectEverolimus reduced subependymal giant cell astrocytoma (SEGA) volume vs placebo in tuberous sclerosis

In the Atlas

Related topics

mTOREverolimusmTORC1Tuberous sclerosis complex

Answers that reference this study

More studies on this topic

Learn the biology

Want to understand the biology behind this study? → The TSC Complex — Where the Inputs Meet

Cite this paper

ShowHide 
Franz, D. N., Belousova, E., Sparagana, S., Bebin, E. M., Frost, M., & Jozwiak, S. (2013). Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicentre, randomised, placebo-controlled phase 3 trial. Lancet. https://doi.org/10.1016/S0140-6736(12)61134-9

@article{FRA2013,
  author       = {Franz, D. N. and Belousova, E. and Sparagana, S. and Bebin, E. M. and Frost, M. and Jozwiak, S.},
  title        = {{Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicentre, randomised, placebo-controlled phase 3 trial}},
  journal      = {Lancet},
  year         = {2013},
  doi          = {10.1016/S0140-6736(12)61134-9},
  note         = {PMID: 23158522},
}

Cite this Atlas record

The record is the Atlas's own work — the evidence label, the extracted findings and the links. It is cited as part of the dataset, not as the paper. 

Barton, O. (2026). Oliver's mTOR Atlas (record FRA2013) [Data set]. https://mtor-atlas.org/study/FRA2013/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_FRA2013,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}, record FRA2013},
  howpublished = {Data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/study/FRA2013/},
  doi          = {10.5281/zenodo.22059963}
}