The DOI matches its Crossref record; 23 of the 23 references it cites were found in a registry or an index.
Rapamycin causes regression of astrocytomas in tuberous sclerosis complex
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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Cites
Phosphorylation and Functional Inactivation of TSC2 by Erk
Ma, L., Chen, Z., Erdjument-Bromage, H., Tempst, P., & Pandolfi, P. P. (2005) · Cell · doi:10.1016/j.cell.2005.02.031 · Cited by 1,268
Galanis, E., Buckner, J. C., Maurer, M. J., Kreisberg, J. I., Ballman, K., Boni, J., Peralba, J. M., Jenkins, R. B., Dakhil, S. R., Morton, R. F., et al. (2005) · Journal of Clinical Oncology · doi:10.1200/jco.2005.23.622 · Cited by 694
mTOR-targeted therapy of cancer with rapamycin derivatives
Vignot, S., Faivre, S., Aguirre, D., & Raymond, E. (2005) · Annals of Oncology · doi:10.1093/annonc/mdi113 · Cited by 617
An expanding role for mTOR in cancer
Guertin, D. A., & Sabatini, D. M. (2005) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2005.06.007 · Cited by 523
Rhebbing up mTOR: New Insights on TSC1 and TSC2, and the Pathogenesis of Tuberous Sclerosis
Kwiatkowski, D. J. (2003) · Cancer Biology & Therapy · doi:10.4161/cbt.2.5.446 · Cited by 387 · cited 1×
“When mTOR is constitutively activated through mutations in either hamartin or tuberin, this results in the hamartomatous lesions of tuberous sclerosis in the brain, kidney, heart, lung, and other organs of the body (Fig 1).(8,9) Recent studies have also shown that to function under homeostatic conditions, the mTOR pat…”
BackgroundBoulay, A., Rudloff, J., Ye, J., Zumstein-Mecker, S., O'Reilly, T., Evans, D. B., Chen, S., & Lane, H. A. (2005) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-04-2402 · Cited by 335
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Hepatocellular Carcinoma: Molecular Pathways and New Therapeutic Targets
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Rapamycin: an anti-cancer immunosuppressant?
Law, B. K. (2005) · Critical Reviews in Oncology/Hematology · doi:10.1016/j.critrevonc.2004.09.009 · Cited by 252
Goudar, R. K., Shi, Q., Hjelmeland, M. D., Keir, S. T., McLendon, R. E., Wikstrand, C. J., Reese, E. D., Conrad, C. A., Traxler, P., Lane, H. A., et al. (2005) · Molecular Cancer Therapeutics · doi:10.1158/1535-7163.101.4.1 · Cited by 243
Cited by
Defining the role of mTOR in cancer
Guertin, D. A., & Sabatini, D. M. (2007) · Cancer Cell · doi:10.1016/j.ccr.2007.05.008 · Cited by 2,833
The Tuberous Sclerosis Complex
Crino, P. B., Nathanson, K. L., & Henske, E. P. (2006) · New England Journal of Medicine · doi:10.1056/nejmra055323 · Cited by 1,793
Northrup, H., Krueger, D. A., Northrup, H., Krueger, D. A., Roberds, S., Smith, K., Sampson, J., Korf, B., Kwiatkowski, D. J., Mowat, D., et al. (2013) · Pediatric Neurology · doi:10.1016/j.pediatrneurol.2013.08.001 · Cited by 1,502
Self-citationCarracedo, A., Ma, L., Teruya-Feldstein, J., Rojo, F., Salmena, L., Alimonti, A., Egia, A., Sasaki, A. T., Thomas, G., Kozma, S. C., et al. (2008) · Journal of Clinical Investigation · doi:10.1172/jci34739 · Cited by 1,381
Self-citationmTOR and cancer: insights into a complex relationship
Sabatini, D. M. (2006) · Nature Reviews Cancer · doi:10.1038/nrc1974 · Cited by 1,322
Metadata
- Authors
- Franz, D. N., Leonard, J., Tudor, C., Chuck, G., Care, M., Sethuraman, G., Dinopoulos, A., Thomas, G., & Crone, K. R.
- Venue
- Annals of Neurology
- Publisher
- Wiley
- Published
- 2006-02-17
- Volume
- 59
- Issue
- 3
- Pages
- 490-498
- ISSN
- 0364-5134 (ISSN-L)
- 1531-8249
- OpenAlex
- W2032555040
- Cited by
- 614
- References
- 23
- FWCI
- 26.07
- Type
- journal article
- Language
- en
- Open access
- closed
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