Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis

Krueger DA, Care MM, Holland K, Agricola K, Tudor C, Mangeshkar P, Wilson KA, Byars A, Sahmoud T, Franz DN · 2010 · New England Journal of Medicine · Atlas ID KRU2010

What this study shows

In patients whose TSC1/TSC2 mutations cause brain tumors, everolimus shrank tumor volume by 30%+ in three-quarters of patients. Open-label and uncontrolled (n=28); randomised confirmation came later (FRA2013).

Abstract

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Neurosurgical resection is the standard treatment for subependymal giant-cell astrocytomas in patients with the tuberous sclerosis complex. An alternative may be the use of everolimus, which inhibits the mammalian target of rapamycin, a protein regulated by gene products involved in the tuberous sclerosis complex. Patients 3 years of age or older with serial growth of subependymal giant-cell astrocytomas were eligible for this open-label study. The primary efficacy end point was the change in volume of subependymal giant-cell astrocytomas between baseline and 6 months.

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 type	2 - Human Clinical Trial
Model system	Humans, open-label trial (n=28)
Journal	New England Journal of Medicine
Year	2010
Peer reviewed	Yes
Record last updated	2026-07-29
Source	DOI 10.1056/NEJMoa1001671 · PMID 21047224

Extracted findings

These fields were extracted from the paper with AI assistance and have not been checked against the source. Read them as a pointer into the paper, not as curated claims; where they disagree with the curated summary above, the curated summary stands.

Intervention	Everolimus
Target	mTOR
Model	Human – open-label trial (n=28, TSC)
Effect	Everolimus reduced subependymal giant-cell astrocytoma (SEGA) volume in tuberous sclerosis

In the Atlas

Links in the pathway map

Supports Everolimus inhibits mTORC1
Supports Everolimus inhibits Tuberous sclerosis complex
Supports TSC1/TSC2 inhibits mTORC1

Cite this paper

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Krueger, D. A., Care, M. M., Holland, K., Agricola, K., Tudor, C., Mangeshkar, P., Wilson, K. A., Byars, A., Sahmoud, T., & Franz, D. N. (2010). Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis. New England Journal of Medicine. https://doi.org/10.1056/NEJMoa1001671

@article{KRU2010,
  author       = {Krueger, D. A. and Care, M. M. and Holland, K. and Agricola, K. and Tudor, C. and Mangeshkar, P. and Wilson, K. A. and Byars, A. and Sahmoud, T. and Franz, D. N.},
  title        = {{Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis}},
  journal      = {New England Journal of Medicine},
  year         = {2010},
  doi          = {10.1056/NEJMoa1001671},
  note         = {PMID: 21047224},
}

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 KRU2010) [Data set]. https://mtor-atlas.org/study/KRU2010/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_KRU2010,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}, record KRU2010},
  howpublished = {Data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/study/KRU2010/},
  doi          = {10.5281/zenodo.22059963}
}

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