Horm Metab Res 2009; 41(9): 680-686
DOI: 10.1055/s-0029-1214381

© Georg Thieme Verlag KG Stuttgart · New York

Malignant Pheochromocytomas and Paragangliomas: Molecular Signaling Pathways and Emerging Therapies

L. Santarpia 1 , M. A. Habra 1 , C. Jiménez 1
  • 1Department of Endocrine Neoplasia and Hormonal Disorders, The University of Texas M. D. Anderson Cancer Center, Houston, Texas, USA
Further Information

Publication History

received 19.12.2008

accepted 03.02.2009

Publication Date:
02 April 2009 (online)


Patients having malignant pheochromocytomas and paragangliomas traditionally have been treated with systemic chemotherapy and 131I-meta-iodobenzylguanidine. However, these therapies have limited efficacy and the potential for significant toxicity. Over the last decade, researchers have discovered new gene mutations associated with malignant pheochromocytomas and paragangliomas, facilitating a better understanding of the molecular pathways involved in the development of these tumors. This new knowledge has brought with it the potential to test new medications that specifically target the signal transduction abnormalities known to be involved in malignant transformation. We are among the groups to have recently reported the use of the tyrosine kinase inhibitor sunitinib in a limited number of patients with malignant pheochromocytomas and paragangliomas. The use of sunitinib was associated with a reduction in the size of the tumors, their biochemical markers, and symptomatic improvement. In this review, we will explore these newly understood molecular pathways and the emerging therapies that may change the management of this disease.



Dr. C. Jiménez

Department of Endocrine Neoplasia and Hormonal Disorders

The University of Texas M. D. Anderson Cancer Center

1515 Holcombe Boulevard

Unit 435


77030 Texas


Phone: +1/713/792 28 41

Fax: +1/713/794 40 65

Email: [email protected]