Caring for Carcinoid Foundation

Transcription

1 Caring for Carcinoid Foundation Annual Report 2012 Dedicated to discovering cures for carcinoid, pancreatic neuroendocrine, and related neuroendocrine cancers

2 Caring for Carcinoid Foundation Annual Report 2012 Caring for Carcinoid Foundation Leadership Nancy Lindholm founded the Caring for Carcinoid Foundation the only carcinoid, pancreatic neuroendocrine, and related neuroendocrine cancer-focused foundation with the mission of discovering cures. A formal Board of Directors, a Board of Scientific Advisors, and a structured research road map support its mission. The Board of Directors governs the Caring for Carcinoid Foundation by managing its strategic, scientific, and financial direction. The Board of Directors is comprised of highly distinguished community leaders. All members have diverse educational and professional backgrounds and share a commitment to CFCF s mission. The Board of Scientific Advisors provides scientific direction to the Caring for Carcinoid Foundation. The Board of Scientific Advisors comprises highly distinguished medical leaders who share a commitment to discovering cures for carcinoid, pancreatic neuroendocrine, and related neuroendocrine cancers. CFCF s Board of Scientific Advisors developed CFCF s Research Road Map to discover cures as quickly as possible. Board of Directors Anne Doyle, Chair Stephen Kaufer, Vice Chair Jonathan Soroff, Secretary Nicholas Vantzelfde, Treasurer Stephen Blackwood Carol Branaman Board of Scientific Advisors George Fisher, M.D., Ph.D., Co-Chairman Ramesh Shivdasani, M.D., Ph.D., Co- Chairman Daniel Chung, M.D. Todd Golub, M.D. David H. Lee, Ph.D. Andrew Leiter, M.D., Ph.D. Arnold Levine, Ph.D. Thomas J. Lynch, M.D. Anil Rustgi, M.D. William Sellers, M.D. Evan Vosburgh, M.D. 1 P a g e

3 The Caring for Carcinoid Foundation Caring for Carcinoid Foundation Annual Report 2012 The mission of the Caring for Carcinoid Foundation (CFCF) is to discover cures for carcinoid, pancreatic neuroendocrine, and related neuroendocrine cancers. To achieve rapid discovery of a cure, CFCF directs 100% of all individual donations to fund breakthrough scientific research of neuroendocrine cancers. This is made possible by the generous support of its Board of Directors and Corporate Sponsors. Along with its focus on research, CFCF is committed to addressing the lack of treatment options for patients and supporting patients, families and caregivers by providing them with complete and up-to-date information. Since its inception, CFCF has awarded millions of dollars in large-scale, multi-year research grants to leading scientists at renowned research institutions worldwide including Cancer Research UK, Cold Spring Harbor Laboratory, Dana-Farber Cancer Institute, Johns Hopkins University, MD Anderson Cancer Center, Massachusetts General Hospital, Memorial Sloan- Kettering Cancer Center, the Rockefeller Institute, Stanford University Cancer Center, University of California San Francisco, University of Massachusetts Medical School, and University of Pennsylvania. CFCF has established itself as the leading and most efficient not-for-profit funder of carcinoid, pancreatic neuroendocrine, and related neuroendocrine cancer research. CFCF has achieved this status through guidance from its Board of Directors, whose support allows CFCF to operate efficiently, and its Board of Scientific Advisors, whose members provide a clear Research Road Map and Research Action Plan. CFCF also organizes an annual state-of-the-science symposium to promote discussion and collaboration between neuroendocrine cancer researchers. In addition to research, CFCF provides support to patients and their loved-ones through its award-winning website ( E-Updates, patient education materials, and outreach programs. Through its website, CFCF also maintains its Clinical Trials Resources, a dynamic service to update patients about clinical trials and new treatment options. This resource consists of a dedicated phone line, electronic updates, and a website with a searchable database of trial listings and supportive information. In 2012, CFCF continued its National Patient Education Series in Los Angeles, CA and Philadelphia, PA. Over 400 patients and supporters attended in total. CFCF supports all carcinoid, pancreatic neuroendocrine, and related neuroendocrine cancer patients, families, and friends! 2 P a g e

4 Caring for Carcinoid Foundation Annual Report 2012 Research Progress in 2012 The Caring for Carcinoid Foundation holds an annual research symposium in order to discuss the latest findings from our research grants and to update and review our Research Action Plan and Research Road Map. In 2012, the symposium took place on April 11 at the Whitehead Institute of Biomedical Research. Evan Vosburgh, M.D., served as scientific organizers of the event, which was sponsored by CFCF and the Raymond and Beverly Sackler Foundation. Over 70 distinguished researchers, physicians, and patient advocates attended the symposium. The Caring for Carcinoid Foundation continued its partnership with the American Association for Cancer Research (AACR). The AACR is an international organization whose mission is to prevent and cure cancer through research, education, communication and collaboration. The AACR is the oldest and largest scientific organization in the world focused on every aspect of high-quality innovative cancer research. In 2012, CFCF pledged $275,000 to support research projects in conjunction with the AACR. The grants will support innovative ideas and approaches with direct application and relevance to carcinoid or pancreatic neuroendocrine cancer. In 2012, the Caring for Carcinoid Foundation awarded over 1.6 million dollars to launch high-impact research initiatives across all three steps of our Research Road Map. The Mechanistic Underpinnings of Pancreatic Neuroendocrine Tumors Guillermina Lozano, Ph.D., has extensive experience in generating mouse models to study the effects of specific mutations on tumor development. Her laboratory at MD Anderson Cancer Center has focused on the p53 pathway, in particular, and understanding how it regulates tumor development. The p53 pathway is mutated in over half of all human malignancies. faculty.mdanderson.org Previous CFCF-funded researchers discovered mutations in the genes Daxx and ATRX among tumors from patients with non-functioning pancreatic neuroendocrine tumors. Despite these exciting and promising findings, the precise role of ATRX and Daxx in neuroendocrine tumor development is yet to be understood and treatments exploiting these findings have yet to be developed. 3 P a g e

5 Caring for Carcinoid Foundation Annual Report 2012 Furthermore, researchers do not have the research tools they need to develop potential new therapies for patients exploiting these mutations. In this project, Dr. Lozano will create the mouse models necessary to identify the cellular changes that occur with loss of Daxx and ATRX to determine the impact of Daxx and ATRX mutations on tumor growth. The mouse models that the team creates will both define the importance of the p53 pathway in the maintenance of pancreatic neuroendocrine tumors and be useful to test potential new therapies. The Impact of MEN1, DAXX, ATRX, and PTEN in Pancreatic Neuroendocrine Cancer Pathogenesis Kwok-KinWong, M.D., Ph.D. and his laboratory at the Dana-Farber Cancer Institute specialize in integrating genomic information with relevant mouse models to study novel treatments with the ultimate goal of moving new treatments into clinical trials for patients. Previous CFCF-funded researchers discovered mutations in the genes ATRX and DAXX among tumors from patients with non-functioning pancreatic neuroendocrine tumors. Despite these exciting and promising findings, the precise role of ATRX and DAXX in neuroendocrine tumor development is yet to be understood and treatments exploiting these findings have yet to be developed. Furthermore, researchers do not have the research tools, including mouse models, they need to develop new therapies for patients. In this project Dr. Wong will create the mouse models necessary to determine the impact of recently identified mutations in neuroendocrine doctors.dana-farber.org tumor development. Next, Dr. Wong will conduct experiments using his mouse models to identify the cellular pathways that are deregulated as a result of these mutations. Any pathways identified represent potentially new and novel therapeutic targets for treatment of patients with neuroendocrine tumors. Understanding the Tumor Suppressor Activities of ATRX-DAXX Through Epigenomic Profiling and Animal Models The chromosomes in our cells are composed of equal amounts of DNA and protein. The cellular machine of ATRX-Daxx helps to build and maintain chromosome structure at specific sites in our genome, including telomeres, the special structures that cap and protect the ends of our chromosomes. Previous CFCF-funded researchers discovered mutations in the genes ATRX and Daxx among tumors from patients with non-functioning pancreatic neuroendocrine tumors. Despite these exciting and promising findings, the precise role of ATRX and Daxx in neuroendocrine tumor development is yet to be understood and treatments exploiting these findings have yet to be developed. 4 P a g e

6 Caring for Carcinoid Foundation Annual Report 2012 C. David Allis, Ph.D., has assembled an impressive team of collaborators from both Rockefeller University and Memorial Sloan Kettering Cancer Center to answer these important questions. This team brings together expertise in epigenomics, neuroendocrine tumor biology and pathology. Dr. Allis' team will conduct experiments and create models to understand the role of ATRX and Daxx in neuroendocrine tumor development with the ultimate goal of developing new therapies for patients by targeting these processes. Furthermore they will establish the precise changes in chromosome structure resulting from mutations in ATRX and Daxx. Knowledge of these changes could shed light on not only neuroendocrine cancers but many other cancer types as well. Mouse Model Project Using Forward Genetics David Tuveson, M.D., Ph.D, and his team at Cold Spring Harbor Laboratory will use their expertise in forward genetics and mouse cancer modeling to mutagenize enterochromaffin cells, enteroendocrine cells found in the digestive and respiratory tracts, to both generate models of carcinoid cancer and simultaneously identify genes and pathways that promote carcinoid cancer formation. This research is critical because, according to Dr. Tuveson, the development of more effective treatment regimens for patients with carcinoid metastasis and carcinoid syndrome has been hampered by the lack of effective in vivo models, which recapitulate the disease process in humans." The lack of model systems that accurately recapitulate the behavior of neuroendocrine cancers has long been a significant hurdle to developing targeted treatments for patients. Building upon Dr. Tuveson s previous grant from CFCF and the AACR, this project has the promise to create faithful animal models; therefore, eliminating one of the barriers to treatment development. 5 P a g e

7 Caring for Carcinoid Foundation Annual Report 2012 Advocating for Change The Caring for Carcinoid Foundation continued to advocate for the carcinoid and neuroendocrine cancer community in CFCF accepted an invitation to join the World NET Cancer Awareness Day Membership Council. World NET Cancer Day is an annual event to raise global awareness of neuroendocrine cancers. The council consists of charitable organizations from eight countries that collaborate on several major campaigns surrounding November 10, World NET Cancer Awareness Day. As neuroendocrine cancers are also rare diseases, CFCF partnered with the Whitehead Institute at the Massachusetts Institute of Technology for an International Rare Disease Day event on February 29. The event aimed to inform decision-makers as well as the general public about how rare diseases impact patients lives. CFCF also participated in the 2012 BIO International Convention in order to educate a broad biomedical sciences audience on neuroendocrine cancers. Hosted by the Biotechnology Industry Organization (BIO), this is the largest biotechnology event in the world. Over 15,500 people from 48 states and 65 countries attended the event. Locally, CFCF partnered with the American Cancer Society s Cancer Action Network (ACS-CAN) to plan an event in New England. The event featured a keynote address from Pam Ter Gast, a neuroendocrine cancer patient from the Boston area. Pam and CFCF s Director of Research Lauren Erb spoke on CBS Boston radio s The Jordan Rich Show. They discussed the trials facing patients with neuroendocrine cancers and CFCF s mission to discover a cure. 6 P a g e

8 Supporting the Community Caring for Carcinoid Foundation Annual Report 2012 The Caring for Carcinoid Foundation maintains a user-friendly, award-winning website featuring a plethora of patient resources, including Patient Education Conference Materials, a Video Gallery, and a Newly Diagnosed Patients section. The Caring for Carcinoid Foundation also supports neuroendocrine cancer patients and their families through its Patient Education Conference series. Since 2009, thousands of individuals have attended these day-long seminars at prestigious medical institutions in California, Texas, Pennsylvania, and Massachusetts. These seminars bring together our extensive community of leading clinicians and scientists in the fields of oncology, liver transplant, primary care, surgery, endocrinology, genomics, and research. Patients and supporters attending these events are able to learn about the latest breakthroughs in neuroendocrine cancer research, as well as learn about innovative ways to manage their disease. In 2012, over 400 participants attended CFCF s events in Los Angeles, CA and Philadelphia, PA. Speakers at the Los Angeles Patient Education Seminar held in conjunction with Cedars-Sinai Medical Center included Edward Wolin, M.D., Carlos Anaya, M.D., Arash Asher, M.D., Marc Friedman, M.D., Nicholas Nissen, M.D., Jodi Newson, M.S., R.D., C.S.O, Joseph Pisgena, M.D, and a patient panel. Speakers at the Focus on Neuroendocrine Tumors: Patient and Caregiver Conference held in conjunction with the University of Pennsylvania Abramson Cancer Center included David Metz, M.D., Joseph Carver, M.D., Debbie Cohen, M.D., Emma Furth, M.D., Katherine Nathanson, M.D., Vinay Chandrasekhara, M.D., Giorgos Karakousis, M.D., John Kucharczuk, M.D., Michael Soulen, M.D., David Mankoff, M.D., Ph.D., Diane Reidy-Lagunes, M.D., Shana Merrill, C.G.C., Kathleen Montone, M.D., Keith Cengel, M.D., Ph.D., Stephen Keefe, M.D., Lauren Fishbein, Steven J. Marx, M.D., Lauren Erb, Ruth Steinman, M.D., and Carolyn Vachani, M.S., C.R.N.P. 7 P a g e

9 Caring for Carcinoid Foundation Annual Report 2012 Raising Funds for Research and Creating Awareness The Caring for Carcinoid Foundation brings the neuroendocrine cancer community together through a series of highly visible athletic events across the nation. Team members, including patients, caregivers, physicians and friends, run, bike, walk, and swim to a cure. These events benefit patients by empowering them to go out into their communities and advocate and fight for increased awareness of their disease. Athletic events also facilitate growth and development of communities, as they are so often dedicated in honor and/or in memory of loved ones. In 2012, Team CFCF participated in athletic events across the country. Bolder Boulder in Boulder, CO; Boston Marathon Jimmy Fund Walk in Boston, MA; Country Music Marathon in Nashville, TN; Cure Carcinoid Golf Tournament in Alpine, NJ; Cycle for Survival in New York, NY; Kentucky Derby Festival Marathon in Louisville, KY; Los Angeles Marathon and 5K in Los Angeles, CA; MD Anderson SCOPE 5K in Houston, TX; Pan-Mass Challenge in Massachusetts; Register s Annual Great Bicycle Race in Iowa; Run for the Stripes 5K in Philadelphia, PA; Walk With Chuck in Carlsdtadt, NJ. 8 P a g e

10 Caring for Carcinoid Foundation Annual Report 2012 Financial Information 2012 Audited Financial statements are currently in process. To receive information regarding the Caring for Carcinoid Foundation s financial performance, please call In the interim, please see the Audited Financial statements for the 2011 year at under the Financials section. CFCF is committed to transparency and accountability, and is happy to provide accurate information as it becomes available. 9 P a g e

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