Est. 2000 finding the cure in our time SFA Newsletter 2012 Board of Directors President’s Corner Mark Thornton, MD, PhD President Dear Friends, John Brooks, MD Vice President of Scientific and On behalf of the Sarcoma Foundation of America (SFA), I would Government Affairs like to thank you for taking the time to read this issue of the SFA Thomas Tobin, MD Newsletter. We sincerely thank you for supporting our mission to Vice President of Financial advocate for increased research to find new and better therapies for Affairs & Treasurer treating patients with sarcoma, and we hope you find the articles in Gary Tomei, Esq. Vice President of Legal Affairs this issue illuminating and inspiring. Stacey Breidinger Secretary The past year has been a transformational one in our community! Matthew Alsante On the drug development front, the first new therapy for sarcoma Executive Director in over 30 years, Votrient, was approved by the FDA in April! Chris Connery Votrient will prolong the time to progression in nearly all sarcoma Anthony Fedorov patients to one degree or another, but more importantly it begins Dr. Mark Thornton Alexandra Gillespie a new era where other therapies will be combined with Votrient to Michael Mandell hopefully synergize their actions against the disease. Your SFA was right there when the FDA reviewed Patrick Mortimer Votrient for approval, advocating publically for its quick approval, and in the process of a cordial and Rita Saverino Romeo respectful advocacy effort with the FDA has opened new doors for future collaborations to streamline Candace Sue drug development pathways for sarcoma and its subtypes. Patricia Thornton Honorary Board Members On the research front, the SFA funded our first three-year Conquer Cancer Foundation Career Development Award, is currently funding our sixth Conquer Cancer Foundation Young Investigator Laurence H. Baker, DO Award, and 10 additional SFA sarcoma research grants worth $500,000! These grants are designed to Murray Brennan, MD foster the world’s best scientists in the field of sarcoma and ignite ideas for new research avenues to Ed Fry pursue in the quest to find new treatments to eradicate sarcoma. Peri Gilpin Sean Lennon Yoko Ono The SFA has continued to advance our mission on many additional fronts including growing our Sarcoma Marisa Petroro Patient Registry to over 1,400 patients! The Registry is allowing us to match patients in need to clinical U.S. Senator Mark Pryor trials and is providing outcomes data for epidemiologic research studies to understand better sarcoma’s Marisa Tomei natural history. Future plans are to add patient genomic data, which combined with clinical data, will Martha Wainwright help move us toward the vision one day soon of individualized treatment programs. Rufus Wainwright Heather Urich With your help, our programs have touched the lives of so many. Though we have made progress, there Medical Advisory Board is still an urgent need for further sarcoma research, education and awareness. I encourage everyone to give generously in support of this cause. Thank you, once again, for your continued support. Together, Chairman we have the very real opportunity of finding the cure in our time. George Demetri, MD Robert Benjamin, MD Charles Forscher, MD Richard Gorlick, MD Lee Helman, MD Inside this issue: Marc Ladanyi, MD President’s Corner ........................................... 1 Annual Gala .................................................... 5 Jonathan Lewis, MD, PhD SFA Partners with NDRI ................................. 2 2012 Sarcoma Research Grants ...................... 6 Robert Maki, MD, PhD Rare Disease Lobby Day ................................ 2 Conquer Cancer Foundation Awards .............. 6 Crystal L. Mackall, MD Volunteerism ................................................... 3 Sarcoma Patient Registry ................................ 6 Alberto Pappo, MD SFA Combined Federal Campaign Number.... 3 Fundraising Events .......................................... 7 Shreyaskumar Patel, MD Californians for a Cure .................................... 4 Mission ............................................................ 8 Peter Pisters, MD SFA Educational Conference .......................... 4 Donate ............................................................. 8 Raphael Pollock, MD, PhD, FACS www.curesarcoma.org Page 1 SFA Partners with NDRI to Advance Sarcoma Research The Sarcoma Foundation of America has partnered with The National that only NDRI employees can link to your personal information. The Disease Research Interchange (NDRI), a non-profit organization, information that will be shared with researchers receiving your donated to provide you with the opportunity to donate tissues and have those tissue sample will be de-identified of any personal information and will tissues distributed to scientists who are studying different aspects of only include information, such as but not limited to, your age, race, sex, sarcoma and related cancers. Sarcoma patients can donate samples of all medical and social history, and sometimes clinical information directly forms of sarcoma obtained from biopsy procedures or other medically associated with the tissue sample you donated. necessary surgical procedures. Other tissues can be donated too. NDRI coordinators are able to discuss with you the two donation opportunities, If you are interested in organ and tissue donation, completing your either from a surgical procedure or post mortem (after death, like in registration as early as possible is vital to success. To learn more about donation for transplant), and will help you make the right donation registering with NDRI as an organ and tissue donor for sarcoma decision for you and your family. research, or to learn more about other donation for research options, please contact an NDRI coordinator today by calling 1-800- Tissue donations are coordinated by NDRI from start to finish, ensuring 222-NDRI (6374). An NDRI coordinator is available to speak with your wishes to donate are made successful. The tissue you donate will you 24 hours a day. be de-identified and will be assigned a unique identification number Rare Disease Lobby Day: My Experience on Capitol Hill By Laura Welch, SFA Member I am a 33-year-old mother of a 2-year-old boy and wife to a wonderful and an experienced US Soldier. In July 2011 we moved from Seattle to DC. Hello sunshine! advocate. We went to the office of Rep. Jim Moran This past August 18 I was diagnosed with Epitheliod Sarcoma (ES), a (D-VA) and spoke with rare and aggressive form of cancer. I did what anyone in this day and age his staff person, Marcia. would do; I consulted the bible that is Google! The prognosis was bleak, She was very welcoming. to put it mildly. However, I am of the mindset that if this disease is so Congressman Moran is rare that it has little data available, I will make my own data and try not already a member of the to dwell on the figures in front of me. Dr. Jack Brooks & Rep. Patrick Meehan Rare Disease Caucus and with other Lobby Day participants. When given the option of a below-the-knee amputation, I was also given Marcia believed he would the best odds of survival I had heard since my diagnosis. It was a no- be supportive of the Acts we brainer. My husband cried in shock and I cried with relief! discussed. As an introduction to advocacy and Capitol Hill, I couldn’t have asked for a better meeting or staff. Following surgery I found a small ES support group online. It was this group that alerted me to the fact that February 29 was World Rare Next was a meeting in Sen. Jim Webb’s (D-VA) office. His staff seemed Disease Day. A Capitol Hill Lobby Day was coordinated by the Rare pleasantly surprised that there was no cost to them involved in the Disease Legislative Advocates (RDLA). RDLA partnered with the Acts. He asked us what resistance has been faced so far. He could Sarcoma Foundation of America, the EveryLife Foundation for Rare appreciate the simplicity and commonsense approach to both bills and Diseases, and several other organizations to ensure that rare disease couldn’t see why there would be much resistance. We left feeling that he advocates were present on Capitol Hill. was interested in what we had to say and that the senator would do further research. With no previous advocacy experience, I was nervous about what to expect and who I would meet. But, I also had a strong urge to want to Finally, in Sen. Mark Warner’s (D-VA) office we were advised that do something to help. I consider myself lucky as far as ES patients go; I healthcare is not an issue that the senator usually plays a lead role on, haven’t had to take any medications or have chemotherapy or radiation but that he would discuss it further with the members of the Senate who treatments. There are many people out there wondering if the latest would be able to take action on our behalf. He appeared to appreciate combination of drugs they are being given will somehow work for them. what a significant impact it would have if the FDA could fast-track the They needed a voice. drugs needed to treat rare diseases. During our breakfast meeting in Washington, DC, we were given an Overall I was impressed with the time given, questions asked and overview of the Congressional Rare Disease Caucus and two important responsiveness to the ULTRA Act and the Creating Hope Act. The pieces of legislation: the ULTRA Act and Creating Hope Act. I found experience highlighted to me that perhaps it is only when we are everyone to be so passionate about their work. I was instantly eager to personally touched in some way that we really understand the get to Capitol Hill to discuss my newfound knowledge. The legislation importance of speaking out for one another and trying to make a made complete sense; how could Congress not want to back them? difference. I came away from the day excited that I can use my personal misfortune and voice to help others.