Novel and Evolving Therapies in the Treatment of Malignant Phaeochromocytoma: Experience with the mTOR Inhibitor Everolimus (RAD001)

Druce, MR; Kaltsas, GA; Fraenkel, M; Gross, DJ; Grossman, AB

dc.contributor.author	Druce, MR	en
dc.contributor.author	Kaltsas, GA	en
dc.contributor.author	Fraenkel, M	en
dc.contributor.author	Gross, DJ	en
dc.contributor.author	Grossman, AB	en
dc.date.accessioned	2014-03-01T11:46:23Z
dc.date.available	2014-03-01T11:46:23Z
dc.date.issued	2009	en
dc.identifier.issn	0018-5043	en
dc.identifier.uri	https://dspace.lib.ntua.gr/xmlui/handle/123456789/37869
dc.subject	phaeochromocytoma	en
dc.subject	temozolomide	en
dc.subject	radiopharmaceuticals	en
dc.subject	sunitinib	en
dc.subject	everolimus	en
dc.subject.classification	Endocrinology & Metabolism	en
dc.subject.other	NEUROENDOCRINE TUMORS	en
dc.subject.other	METAIODOBENZYLGUANIDINE I-131-MIBG	en
dc.subject.other	PHASE-II	en
dc.subject.other	PARAGANGLIOMA	en
dc.subject.other	RAPAMYCIN	en
dc.subject.other	PROLIFERATION	en
dc.subject.other	COMBINATION	en
dc.subject.other	OCTREOTIDE	en
dc.subject.other	MANAGEMENT	en
dc.subject.other	SUNITINIB	en
dc.title	Novel and Evolving Therapies in the Treatment of Malignant Phaeochromocytoma: Experience with the mTOR Inhibitor Everolimus (RAD001)	en
heal.type	other	en
heal.language	English	en
heal.publicationDate	2009	en
heal.abstract	Phaeochromocytoma and paraganglioma are rare neuroendocrine tumours (NETS). They may be benign or malignant but the pathological distinction is mainly made when metastases are present. Available treatments in the form of surgery, chemotherapy, and radionuclide therapy may improve symptoms and biochemical markers, but the results for the control of tumour bulk are less favourable. Furthermore, responses to treatment are frequently short-lived. This short review outlines the main molecular and histological features of malignant phaeochromocytoma and the difficulties in differentiating between benign and malignant disease. We list current therapies used for malignant pheochromocytoma, however, these generally achieve relatively low success rates. Hence, there is a need for new and more effective therapies. In vitro studies have implicated the PI3/Akt/mTOR pathway in the pathogenesis of malignant NETS, including phaeochiomocytoma. Everolimus (RAD001, Novartis UK) is a compound that inhibits mTOR (mammalian Target Of Rapamycin) signalling. We have used RAD001 in four patients with progressive malignant paraganglioma/phaeochromocytoma in addition to other therapies (with institutional approval for compassionate use), and evaluated the effects of this treatment. We Outline these four cases and review the theoretical background for this therapy, although the Outcomes were relatively disappointing	en
heal.publisher	GEORG THIEME VERLAG KG	en
heal.journalName	HORMONE AND METABOLIC RESEARCH	en
dc.identifier.isi	ISI:000270456900008	en
dc.identifier.volume	41	en
dc.identifier.issue	9	en
dc.identifier.spage	697	en
dc.identifier.epage	702	en