{"id":"d3e91597-81f7-43ba-ab6f-0651eb631938","slug":"from-the-bench-to-the-bedside-everolimus-for-subependymal-giant-cell-astrocytomas-in-tuberous-sclerosis-complex-optic-nerve-regeneration-targeted-cytotoxins-for-gliomas","title":"From the bench to the bedside: Everolimus for subependymal giant cell astrocytomas in Tuberous sclerosis complex, optic nerve regeneration, targeted cytotoxins for gliomas","authors":["Jason S. Hauptman"],"abstract":"Background: Tuberous sclerosis complex (TSC) is an autosomal dominant disorder characterized by multiple hamartomas, including subependymal giant-cell astrocytomas (SGAs). The condition is linked to dysregulation of the mammalian target of rapamycin (mTOR), leading to increased cell growth and proliferation. Everolimus, an mTOR inhibitor, has emerged as a potential treatment for SGAs in TSC, offering a non-surgical therapeutic option. Methods: In this study, 28 patients with SGAs and TSC received Everolimus for a mean duration of 21.5 months. The treatment included a mandatory six-month period, with 25 patients continuing beyond this timeframe. The primary outcomes measured were tumor volume reduction, changes in hydrocephalus, intracranial pressure, seizure frequency, and quality of life. Results: Everolimus treatment resulted in a significant reduction in SGA size, with 75% of patients experiencing at least a 30% reduction in tumor volume and 32% achieving at least a 50% reduction. Improvements were also noted in hydrocephalus and seizure frequency, with no patients requiring surgical intervention or experiencing worsening symptoms. Adverse events were primarily self-limiting infections and stomatitis, with only four patients experiencing serious adverse effects. Conclusion: The study demonstrates that Everolimus is an effective treatment for subependymal giant-cell astrocytomas in patients with tuberous sclerosis complex, providing a promising alternative to surgical management. This research highlights the potential of molecular medicine in neurosurgery and opens avenues for further exploration of Everolimus as an anti-epileptic medication. Keywords: Tuberous Sclerosis, Everolimus, Subependymal Giant-Cell Astrocytomas, mTOR Inhibitor, Neurosurgery, Molecular Medicine, Seizures, Tumor Volume Reduction","thumbnailUrl":null,"publishDate":"2011-01-14T00:00:00.000Z","doi":"10.4103/2152-7806.75587","categories":["Original Article"],"fullTextUrl":"https://surgicalneurologyint.com/api/articles/d3e91597-81f7-43ba-ab6f-0651eb631938/pdf"}