Phase II study of everolimus in children and adults with neurofibromatosis type 2 and progressive vestibular schwannomas

摘要

BackgroundActivation of the mammalian target of rapamycin (mTOR) signaling pathway is thought to be a key driver of tumor growth in Merlin (NF2)-deficient tumors. Everolimus is an oral inhibitor of mTOR complex 1 (mTORC1) with antitumor activity in a variety of cancers.