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Original Article

Rapamycin causes regression of astrocytomas in tuberous sclerosis complex

  1. David Neal Franz MD1,2,*,
  2. Jennifer Leonard MSN, FNP1,2,
  3. Cynthia Tudor MSN, PNP1,2,
  4. Gail Chuck BS1,2,
  5. Marguerite Care MD1,3,
  6. Gopalan Sethuraman PhD4,
  7. Argirios Dinopoulos MD1,2,
  8. George Thomas PhD5,
  9. Kerry R. Crone MD1,6

Article first published online: 1 FEB 2006

DOI: 10.1002/ana.20784

Issue

Annals of Neurology

Annals of Neurology

Volume 59, Issue 3, pages 490–498, March 2006

Additional Information

How to Cite

Franz, D. N., Leonard, J., Tudor, C., Chuck, G., Care, M., Sethuraman, G., Dinopoulos, A., Thomas, G. and Crone, K. R. (2006), Rapamycin causes regression of astrocytomas in tuberous sclerosis complex. Ann Neurol., 59: 490–498. doi: 10.1002/ana.20784

Author Information

  1. 1

    Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH

  2. 2

    Department of Neurology, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH

  3. 3

    Department of Radiology, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH

  4. 4

    Department of Neuroscience Platform, Eli Lilly & Company, Indianapolis, IN

  5. 5

    Department of Genome Science, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH

  6. 6

    Department of Neurosurgery, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH

*Department of Neurology, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, ML 2015, Cincinnati, OH 45229-3039

Publication History

  1. Issue published online: 17 FEB 2006
  2. Article first published online: 1 FEB 2006
  3. Manuscript Accepted: 21 NOV 2005
  4. Manuscript Revised: 14 NOV 2005
  5. Manuscript Received: 9 OCT 2005

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Abstract

Objective

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.

Methods

Five subjects with clinically definite TSC and either subependymal giant cell astrocytomas (n = 4) or a pilocytic astrocytoma (n = 1) were treated with oral rapamycin at standard immunosuppressive doses (serum levels 5–15ng/ml) from 2.5 to 20 months. All lesions demonstrated growth on serial neuroimaging studies. Magnetic resonance imaging scans were performed before and at regular intervals following initiation of therapy.

Results

All lesions exhibited regression and, in one case, necrosis. Interruption of therapy resulted in regrowth of subependymal giant cell astrocytomas in one patient. Resumption of therapy resulted in further regression. Treatment was well tolerated.

Interpretation

Oral rapamycin therapy can induce regression of astrocytomas associated with TSC and may offer an alternative to operative therapy of these lesions. Ann Neurol 2006

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