Everolimus (RAD001): First Systemic Treatment for Subependymal Giant Cell Astrocytoma Associated with Tuberous Sclerosis Complex

Abstract

Everolimus (RAD001), a mTOR inhibitor, was initially used as an immunosuppressant in organ transplant patients; however, it also has significant antineoplastic properties. In patients with subependymal giant cell astrocytomas (SEGAs) associated with tuberous sclerosis complex who are not candidates for surgery, single-agent everolimus has demonstrated the ability to significantly reduce SEGA volume with good tolerability. In the Phase III, randomized, placebo-controlled trial, everolimus was associated with a SEGA response rate of 35% compared with 0% in the placebo group. The most common adverse events in clinical trials were stomatitis/mouth ulceration and upper respiratory tract infections, and most adverse events were grade 1 or 2; grade 4 events were rare.

Keywords:

• everolimus
• mTOR inhibition
• subependymal giant cell astrocytomas
• tuberous sclerosis complex
• tumor volume

Financial & competing interests disclosure

Funding for development of this manuscript was provided by Novartis Pharmaceutical Corporation. S Józwiak has received speaking and advisory board honoraria from Novartis Pharmaceuticals. K Stein is an employee of Novartis Pharmaceuticals. K Kotulska has received speaking honoraria from Novartis Pharmaceuticals. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Editorial assistance and technical support was provided by ApotheCom (supported by funding from Novartis Pharmaceutical Corporation) for the development of this manuscript.

Notes

TSC: Tuberous sclerosis complex.

Roach ES, Gomez MR, Northrup H. Tuberous Sclerosis Complex Consensus Conference: Revised Clinical Diagnostic Criteria. Copyright © 1998; J. Child. Neurol. 13, 624–628. Reprinted by permission of SAGE Publications.

Additional information

Funding

Funding for development of this manuscript was provided by Novartis Pharmaceutical Corporation. S Józwiak has received speaking and advisory board honoraria from Novartis Pharmaceuticals. K Stein is an employee of Novartis Pharmaceuticals. K Kotulska has received speaking honoraria from Novartis Pharmaceuticals. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.