2013 Annual Chairman's Report


Update on the PheoPara Alliance


Over the past year we have continued to grow the Board not only in numbers but in diverse areas of expertise by adding one new member. We have eleven dedicated Board members with interesting backgrounds all committed to the success of the Alliance.


We continue to work with and fund international researchers and organizations to collaborate with one another and to share information in Asia, Europe and the US. We also continue to lay the groundwork in an effort to establish Centers of Excellence for Neuroendocrine Diseases nationally and abroad where interest exists and to share best demonstrated practices among centers that treat Pheochromocytoma and Paraganglioma. We will once again provide financial support for the Patient conference to be held in Bethesda in June of 2014 as we did for the conference held in 2013. Furthermore, we will provide an educational grant to support the ISP 2014 to be held in Kyoto Japan from September 17th to 20th 2014 which has as its main theme and final goal to establish "a Global Partnership to Detect and Cure Pheochromocytoma and Paraganglioma", a worthy title indeed.


We held two very successful Spreading the Word Gala’s in New Jersey and Washington, DC in 2013. Both of these events raised the visibility of the Alliance and our mission and had the added benefit of raising thousands of dollars to support the Alliance's activities. It should be noted that approximately 90% of what we raise from our fundraising is used to support research grants and educational activities. We want to express our sincere gratitude to our donors for their continued financial support. Their generous donations continue to help us realize our mission and keep our aspirations and dreams alive.


The following represent some of the research activities that the Alliance has supported in 2013.


Montefiore Einstein Center for Cancer Care's Endocrine and Neuroendocrine Tumor Program


The Alliance has funded an Endocrine Surgery Research Fellowship that will allow a fellow in Endocrine Surgery to pursue research centered on a better understanding of the pathogenesis of pheochromocytoma and paraganglioma with the goal of identifying new targets for the development of novel therapies. The initial phase of the project is focused on genome wide methylation analysis of tissues isolated from familial and sporadic pheochromocytoma with a comparison to tissue from normal adrenal medulla. Results to date demonstrate that there are distinct differences in epigenetic patterns between normal adrenal medulla and familial pheochromocytomas.  


National Institute of Child Health and Care Development


The Alliance funded the National Institute of Child Health and Care Development for the following; genetic testing for pheochromocytoma and paraganglioma patients and their families, the study of various anti-cancer agents in the therapy of metastatic pheochromocytoma and paraganglioma, and studying possible SDHB animal models.



University of Pennsylvania – Abramson Cancer Center


The Alliance funded two grants at the University of Pennsylvania Medical Center for advancing pheochromocytoma and paraganglioma research, specifically to develop a novel radiation therapy for metastatic pheochromocytoma and paraganglioma. Work on the novel radiation therapy has been delayed, but activities are now back on track. In addition, an effort to characterize the genetic and genomic aberrations associated with pheochromocytoma and paraganglioma, and to determine if distinct patterns of somatic mutations and genomic aberrations are associated with inherited mutations in different proteins. The Alliance funding for this project has provided invaluable support for generation of preliminary data for other grant applications focused on the somatic genetics of pheochromocytoma and parganglioma. The monies from the PheoPara Alliance have also supported tumor collection and annotation which can be used for this important project.



Kolling Institute, University of Sydney, Australia


The Alliance is supporting three interesting studies at the University of Sydney in Australia. The first is to determine the penetrance of PC/PGL in carriers of SDH mutations. The second is to determine genotypic and clinical risk factors predicting occurrence of PC/PGLs in SDH mutation carriers; and the third is to identify and address the information needs, communication preferences and barriers to testing in affected and unaffected individuals requiring genetic testing for SDH as well as provider needs. The University of Sydney continues to be the principal diagnostic facility for genetic testing of patients with pheochromocytoma and paraganglioma in Australia and New Zealand. They have steadily recruited new families (contacted via index cases) into the study. At present, they are on target to enroll and interview 15 individuals per quarter, and intend to increase their recruitment by holding further Patient Information Days in Melbourne Victoria and Brisbane Queensland. The first Patient Information Day held in Sydney in February 2012 generated significant interest and momentum for recruitment amongst families based in New South Wales. Subsequent Patient Information days in 2013 were also held and were well attended. During 2013 the University of Sydney has also focused on optimization of the coimmunoprecipitation methodology and determination of any interaction differences arising due to SDHB mutations as well as added effects on subunit interaction arising as a result of hypoxia.



Erasmus MC University Medical Center, Rotterdam, The Netherlands



Finally, we are supporting a grant at Erasmus University in The Netherlands to select and validate candidates for Pheochromocytoma and Paraganglioma malignancy in vitro and in vivo, which may represent novel biomarkers and therapeutic targets to be further developed and used in the clinic. In addition, they are studying the therapeutic potential of various M-Tor inhibitors in clinical trials to treat malignant tumors.



Timothy G. Rothwell, Chairman





Pheo Para Alliance

1411 Pathfinder Lane

McLean, VA 22101

eMail: info@pheo-para-alliance.org

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