Rapamycin causes regression of astrocytomas in tuberous sclerosis complex

Franz DN, Leonard J, Tudor C, Chuck G, Care M, Sethuraman G, Dinopoulos A, Thomas G, Crone KR · 2006 · Annals of Neurology · Atlas ID FRA2006

What this study shows

Five people with tuberous sclerosis complex whose brain astrocytomas were growing on serial scans (four subependymal giant cell astrocytomas, one pilocytic astrocytoma) were treated with oral rapamycin at standard immunosuppressive levels (blood 5-15 ng/ml) for 2.5 to 20 months. On MRI, every lesion shrank, and one showed necrosis. In one patient the tumour grew back when treatment was interrupted and shrank again when it was restarted. Treatment was well tolerated. This small uncontrolled series was one of the early reports that mTOR inhibition can shrink TSC brain tumours in patients, and it preceded the EXIST-1 everolimus trial. Boundary: 5 patients, open-label case series without a control group; TSC-associated astrocytomas only; regression depended on continued treatment.

Abstract

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Tuberous sclerosis complex (TSC) is a genetic disorder characterized by the formation of hamartomas in multiple organs. Five to 15% of affected individuals display subependymal giant cell astrocytomas, which can lead to substantial neurological and postoperative morbidity due to the production of hydrocephalus, mass effect, and their typical location adjacent to the foramen of Monro. We sought to see whether therapy with oral rapamycin could affect growth or induce regression in astrocytomas associated with TSC.

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	3 - Human Observational
Model system	Humans: case series of 5 patients with TSC (4 subependymal giant cell astrocytomas, 1 pilocytic astrocytoma) treated with oral rapamycin, followed by serial MRI
Journal	Annals of Neurology
Year	2006
Peer reviewed	Yes
Record last updated	2026-10-01
Source	DOI 10.1002/ana.20784 · PMID 16453317

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	Oral rapamycin (sirolimus), trough 5-15 ng/ml, 2.5-20 months
Target	mTORC1 (via FKBP12)
Model	Human
Effect	Regression of all 5 TSC-associated astrocytomas; regrowth on interruption, re-regression on resumption

In the Atlas

Cite this paper

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Franz, D. N., Leonard, J., Tudor, C., Chuck, G., Care, M., Sethuraman, G., Dinopoulos, A., Thomas, G., & Crone, K. R. (2006). Rapamycin causes regression of astrocytomas in tuberous sclerosis complex. Annals of Neurology. https://doi.org/10.1002/ana.20784

@article{FRA2006,
  author       = {Franz, D. N. and Leonard, J. and Tudor, C. and Chuck, G. and Care, M. and Sethuraman, G. and Dinopoulos, A. and Thomas, G. and Crone, K. R.},
  title        = {{Rapamycin causes regression of astrocytomas in tuberous sclerosis complex}},
  journal      = {Annals of Neurology},
  year         = {2006},
  doi          = {10.1002/ana.20784},
  note         = {PMID: 16453317},
}

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 FRA2006) [Data set]. https://mtor-atlas.org/study/FRA2006/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_FRA2006,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}, record FRA2006},
  howpublished = {Data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/study/FRA2006/},
  doi          = {10.5281/zenodo.22059963}
}

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