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Neuro-oncology

Everolimus for astrocytoma in tuberous sclerosis complex

Nature Reviews Neurology volume 9, page 6 (2013)Cite this article

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Tuberous sclerosis complex (TSC) is a genetic disorder characterized by benign tumours in various organs. Now, a phase III study in patients with TSC has shown that the drug everolimus significantly reduces the volume of subependymal giant cell astrocytomas in these patients, providing hope that the first approved treatment for this aspect of TSC could be on the horizon.

Mutations in tuberous sclerosis genes TSC1 or TSC2 underlie the disease, and mediate their pathogenic effects through aberrant control of mammalian target of rapamycin (mTOR)—an important regulator of cell growth and proliferation. Neurological symptoms such as epilepsy and intellectual disability are common in TSC, and about one-fifth of patients develop subependymal giant cell astrocytomas. Although such tumours are benign, as they continue to grow they can cause life-threatening symptoms such as hydrocephalus, leading to requirement for surgery. Moreover, such intervention carries a substantial risk of postoperative morbidity.

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ORIGINAL RESEARCH PAPER

  1. Franz, D. N. et al. 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 doi:10.1016/S0140-6736(12)61134-9

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  1. Katie Kingwell
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Kingwell, K. Everolimus for astrocytoma in tuberous sclerosis complex. Nat Rev Neurol 9, 6 (2013). https://doi.org/10.1038/nrneurol.2012.257

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  • DOI: https://doi.org/10.1038/nrneurol.2012.257

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