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. I hope to take part in I attended my first meeting with Danielle, another Lobby Day participant more advocacy work in the future.

Page 2 www.curesarcoma.org Volunteerism by Sue Embree-Davis, SFA Chapter Leader,

“I expect to pass through this life but once. If therefore there be any kindness I can show, or any good thing I can do to any fellow being, let me do it now, and not defer or neglect it, as I shall not pass this way again.” (accredited to both William Penn and Stephen Grellet)

This is a favorite motto of mine. I was surprised to find that its origin can be traced back to the 1600’s. I certainly agree that it was good advice back then, and continues to be great advice today. Why volunteer? Here are only a few of the benefits of volunteering: 1. It takes us outside our four walls, often relieving loneliness or boredom. 2. We meet new people, perhaps making a new friend or two. 3. Volunteering is extremely beneficial to the groups, organizations, or companies for which we offer our knowledge, enthusiasm, and hours of labor free of charge. 4. We often receive appreciation from those we help, allowing us to feel needed and useful. 5. If we volunteer for a cause which has touched our lives personally, we have the knowledge that we are helping someone else to weather a difficult time in their life, or to live a better and fuller life than might have been possible before. As I sit here writing this article I am volunteering my time to a cause that is very near to my heart, finding a cure for sarcoma cancers through the efforts of the Sarcoma Foundation of America (SFA). I am the volunteer SFA Chapter Leader for the state of California, and I have had the privilege of serving in this position since 2011. Yes, volunteering with the SFA is helping me to recover and give purpose to some of the pain my family has felt as the result of our battle with a sarcoma cancer. There are an endless number of ways for you to volunteer with SFA in support of the mission to find the cure for these devastating cancers! Here are just a few: • If there is no one currently volunteering in your state as the volunteer chapter leader of the SFA (click on your state under SFA chapters to find out) consider taking on this important role • Volunteer to assist the SFA Chapter Leader in your state • Hold a fundraiser to support SFA (I just raised almost $2000 for SFA by inviting friends and family to recognize my birthday with donations to SFA in lieu of gifts) • Distribute SFA materials to your local hospital, schools and universities, cancer centers, community and infusion centers • Share your SFA connection with nurses and ask them to share SFA information with their patients • Ask your friends and family to JOIN SFA – it is free – www.curesarcoma.org • Ask your friends and family to follow SFA on: • Facebook (www.facebook.com/curesarcoma) • Twitter (www.twitter.com/curesarcoma) • Pinterest (www.pinterest.com/curesarcoma) • Help SFA develop online auction opportunities • And we welcome any volunteer ideas you have to support SFA’s work and mission with your time or contributions! We give of ourselves and our time to others when we volunteer, but over and over I hear people say they receive back so much more than they give! Today, consider volunteering for SFA to make your positive impact on the cure for sarcoma!

SFA Now a Member of the Combined Federal Campaign (CFC #57785)

The Combined Federal Campaign (CFC) is the world’s largest and most successful annual workplace charity campaign, raising millions of dollars each year. SFA asks all federal employees – civilian, military and postal – to designate SFA CFC #57785 in support of our mission to eradicate sarcoma. Each fall, national and local charities are able to make payroll contributions directly to the SFA. Our CFC number is 57785. All of us at the SFA sincerely appreciate your generosity.

www.curesarcoma.org Page 3 Californians for a Cure In a first-ever partnership to raise funds for better cancer treatments and care, of important causes and is a true philanthropic legend. “The Legend of Hope the Sarcoma Foundation of America (SFA) and the Foundation Award recognizes a significant luminary in the American arts community who for Research and Patient Care (RUF) joined in a fundraising event, Californians has made notable contributions of time and energy working to better the lives for a Cure, that was held on November 6, 2011, at the Music Box in Hollywood, of those stricken with cancer and by doing so has served as an inspiration to California. SFA Co-Founders and Board President and Treasurer, respectively, countless patients and their families,” said SFA President, Mark Thornton, MD, Dr. Mark and Patricia Thornton co-chaired the festivities with RUF Founder PhD. “Barbara Eden embodies all that and more.” and CEO Heather Menzies Urich, and Peter Hankwitz, CFO, RUF. Musical performances from singers/songwriters Martha and Rufus Wainwright, The evening of hope and recognition was hosted by actor Lewis MacLeod. The American Idol finalist, singer/songwriter and SFA Board member Anthony Virtue in Medicine Award was presented to Dr. Charles Forscher of Cedars- Fedorov, and sarcoma survivor and musician Charlie Lustman had special Sinai Medical Center for his unwavering dedication to his patients’ welfare and meaning at this event as each of the performers have felt the impact of sarcoma for his contributions to sarcoma research. SFA and RUF were also honored in their lives. to present Triumph Awards to Dr. Behrooz Hakimian of Cedars-Sinai Medical Center and Shoshana Silver for their strength and courage in overcoming cancer. SFA and RUF especially acknowledge the Californians for a Cure committee for their hard work and commitment to expand awareness of and raise funds for Hollywood icon Barbara Eden received the Legend of Hope Award. Ms. research through this important joint event. Eden, of I Dream of Jeannie fame, has used her celebrity to support a myriad

Rufus Wainwright, Barbara Eden & Marisa Petroro Beverlye Hyman-Fead & Dr. Mark & Tricia Thornton, Barbara Eden, Beverlye Hyman-Fead, Dr. Charles Forscher Heather Urich &

Sarcoma Foundation of America’s Annual Patient Educational Conference Ask the Experts: Advances in Sarcoma Treatment and Research

On April 29 at the Grand Hyatt Hotel in New York, the Sarcoma Foundation of George D. Demetri, MD America hosted its 7th Annual Educational Conference for sarcoma patients, Dana-Farber Cancer Institute their families, caregivers and other medical professionals. SFA President, Dr. Topic: Future Directions of Sarcoma Therapy Mark Thornton, opened the conference with remarks that highlighted the vital Sarcoma patients and their families traveled from across the country to learn work of the foundation. The day was filled with presentations by experts in all about the latest breakthroughs in sarcoma research and treatments including areas of sarcoma, who shared information about the latest advances in sarcoma genetic findings, SFA funded research, clinical trials, and the origins of research and treatment. sarcoma. The conference in now available to view on our website at SFA was honored to present: www.curesarcoma.org.

John S.J. Brooks, MD, FRCPATH Pennsylvania Hospital Topic: Advances in Research in Sarcoma Pathology and SFA Funded Research

Kurt R. Weiss, MD University of Pittsburgh Medical Center Topic: Advances in Sarcoma Surgery

Crystal L. Mackall, MD National Cancer Institute Topic: Advances in Sarcoma Immunotherapy

Marc Landanyi, MD John S. J. Brooks, MD, FRCPATH, Marc Ladanyi, MD, Kurt R. Weiss, MD, Memorial Sloan-Kettering Cancer Center Crystal L. Mackall, MD & George D. Demetri, MD Topic: How Funding Basic Science Can Lead to New Targets & Treatments

Page 4 www.curesarcoma.org SFA Annual Gala Celebrated 10th Anniversary in Style The Sarcoma Foundation of America celebrated the 10th anniversary of the annual gala and was honored to acknowledge the co-chairpersons, Kathryn Quadracci Flores, MD, Raja M. Flores, MD, Laura Hagan, Paul A. Meyers, MD, and Addie & Gary Tomei, and the Gala committee for their great success. SFA is also honored to thank all our generous sponsors, including our major sponsors Merck, ZIOPHARM Oncology, Threshold Pharmaceuticals, the Altman/Kazickas Foundation, Infinity Pharmaceuticals, Millennium Oncology, Pfizer, Robert Norton, Michael Porter and Katherin Gehl.

The festivities were hosted by Julie Chang, entertainment reporter for WNYW/Fox 5 in New York, who joined the gala for her second year as the Mistress of Ceremony. Dr. Marc Ladanyi, Memorial Sloan-Kettering Cancer Center, received the 2012 Nobility in Science Award in recognition of his continued dedication to the science and research in finding the cure for sarcoma. Lisa and Mac Tichenor, founders of the What Would Willy Want Foundation (Quad W), were presented with the 2012 Vision of Hope Award in recognition of their philanthropic commitment to finding new and better treatment options for sarcoma patients.

Previous SFA Courage Award recipients, Rita Saverino Romeo and Mark Herzlich, New York Giant XLVI Champion , presented the 2012 Courage Award to Maura Cesarini, McKenna Eileen Coyle, Paul Icovitti, Wendy Landes, Robert Russo, Rhoda Sapers and Dr. Kurt Weiss in recognition of each of the award recipient’s heroic battle with sarcoma.

Guest performances by Rock of Ages Tony Award nominee and former American Idol finalist Constantine Maroulis and 2012 American Idol finalist Elisa Testone were well received. The powerful stories, speeches and resolve toward finding the cure for sarcoma reaffirmed SFA’s determination and mission.

2012 Courage Awardees and Presenters: Rita Saverino Romeo, Dr. Kurt Weiss, Addie & Gary Tomei, Laura Hagan, Lisa & McKenna Eileen Coyle, Rhoda Sapers, Maura Cesarini, Robert Russo, Wendy Landes, Mac Tichenor Mark Herzlich, Barbara Icovitti & grandson Nathan

Dr. Tom Tobin, Dr. Jack Brooks, Tricia & Dr. Mark Thornton, Doctors Raja & Kathryn Flores Dr. Marc Ladanyi, Anne-Sophie Ladanyi Alex Gillespie & Matt Alsante & Dr. Maureen Zakowski

Mistress of Ceremony, Julie Chang India Rogers-Shepp, Anne-Sophie Ladanyi & Constantine Maroulis, Elisa Testone & Mark Herzlich McKenna Eileen Coyle

www.curesarcoma.org Page 5 Sarcoma Foundation of America 2012 Research Grants Alexander Burdo/ZIOPHARM Research Award Sarcoma Foundation of America Research Award “Oncogenic Targets of Hedgehog-GL/1 Activation in Ewing Sarcoma” “Targeting ETV1 in Gastrointestinal Stromal Tumor (GIST)” William A. May, MD Ping Chi, MD, PhD Associate Professor Assistant Member and Assistant Attending Physician Children’s Hospital Memorial Sloan Kettering Cancer Center

Alexander Burdo Research Award “Targeting the Hedgehog pathway to inhibit osteosarcoma growth Conquer Cancer Foundation Awards through dual effects on tumor and microenvironment cells.” Career Development Award Michelle A. Hurchla, PhD A Phase III, double-blind, randomized, placebo-controlled trial of Research Instructor sorafenib in desmoid tumors or aggressive fibromatosis (DT/DF) Washington University Mrinal Gounder, MD Mandell/Kropp Run for a Sarcoma Cure Research Award Memorial Sloan-Kettering Cancer Center “ROS1 kinase as a candidate molecular target in angiosarcoma” Young Investigator Award Jonathan Pollack, MD, PhD “A role for injury in Sarcomagenesis” Associate Professor of Pathology Stanford University David Van Mater, MD, PhD Medical Center Mark Herzlich Research Award “C-MYC Target Gene Network in the Ewing’s Sarcoma Cancer Stem Cell” 2012 Jason T. Yustein, MD, PhD Assistant Professor Conquer Cancer Baylor College of Medicine Foundation Kate McGarrigle Memorial Research Award Top Donors “Differentiation Therapy for Rhabdoid Tumors” Nancy Daly, Sandra Orsulic, PhD Director, Women’s Cancer Biology Dr. Martin Murphy, Cedars-Sinai Medical Center Dr. Mark Thornton, Dr. Michael Link

Sarcoma Foundation of America Research Award ©ASCO Phil McCarten 2012 “Desmoplastic small round cell tumor: validation and further development of novel active compounds emerging from high-throughput Call for Grants chemical screens and establishment of xenografts for preclinical studies” Heather Magnan, PhD We will begin accepting grant requests in October 2012. Grants Instructor of $50,000 are available­ to cover equipment and supplies Memorial Sloan-Kettering Cancer Center in support of research on the etiology, molecular biology, Sarcoma Foundation of America Research Award pathogenesis, diagnosis, and treatment­ of human sarcomas. “Preclinical Testing of Compounds Identified in a High Throughput Preference will be given to proposals that focus on novel agents Screen as Potential Chordoma Therapies” and/or promis­ing molecular targets. David M. Loeb, MD, PhD Associate Professor, Oncology and Pediatrics For more information please contact­ Lori Hoffman at Johns Hopkins University [email protected].

Sarcoma Foundation of America Research Award * application deadline is January 31, 2013. “Polarity and Ploidy in Peripheral Chondrosarcoma” Kevin B. Jones, MD Assistant Professor of Orthopaedics Join the Sarcoma Patient Registry University of Utah – Huntsman Cancer Institute Sarcoma Foundation of America Research Award Help us defeat sarcoma by joining our Sarcoma Patient “Study of miR-26a-2 gene as a therapeutic target of human liposarcoma” Registry today and with our collective strength we will make H. Phillip Koeffler, MD a difference. If you are diagnosed with sarcoma and would Mark Goodson Chair in Oncology Research like more information about joining the registry, please call Cedars-Sinai Medical Center 1-866-501-6870 or email [email protected].

Page 6 www.curesarcoma.org Fundraising Events

Bud’s Run, IL Bud’s Run, IL Super Hero’s Run, DE

Ronny Gras, IL 5k Run/Walk for a Sarcoma 9th Annual Bassett Tournament, CT Cure, FL

Olivia Lives, FL Kicking it for a Cure, NY Cup of Hope, FL Rachael Morris Runs in Memory of Beth Van Dyke, NJ

Gifts made to memorialize a loved one are greatly appreciated Upcoming SFA Fundraising Events and will be acknowledged to the individual or family. They Nationwide are a heartfelt way to express your love and appreciation. Gift amounts are kept confidential. Honoring a loved one who is Oct. 14, 2012 Skate for a Sarcoma Cure in Memory of battling sarcoma and/or celebrating the life of someone who has Darell Martin, Vandalia, Ohio survived is a gift in itself. A personal webpage allows people to Oct. 21, 2012 5th Annual Florida 5k Run/Walk for a share memories as well as donate to the Sarcoma Foundation of Sarcoma Cure, Plantation, Florida America and build a legacy committed to fighting sarcoma.

If you are interested in setting up a Memorial or Honor page, Watch for more information about the 11th Annual Gala coming please contact SFA Program Specialist, Cindy Saunders at in the spring of 2013. [email protected] or 301-253-8687.

www.curesarcoma.org Page 7 Our Mission Donate To SFA

The mission of the Sarcoma Foundation of America (SFA) is to Help us fund future sarcoma research by contributing to the advocate for increased research to find new and better therapies with Sarcoma Foundation of America. The SFA is a national, nonprofit which to treat patients with sarcoma. The organization raises money advocacy group for the increased research and treatment of sarcoma. to privately fund grants for sarcoma researchers and conducts (Tax ID # 52-2275294) education and advocacy efforts on behalf of sarcoma patients.

Cut out this form and mail it with your donation or donate online at www.curesarcoma.org

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Contact the SFA about the following ways of giving: Stock Gifts – Giving appreciated stocks or bonds are excellent ways to support the foundation. Planned Gifts – Wills, life insurance policies, property, trusts, gift annuities and retirement plans are examples of planned gifts which provide significant tax savings as well. We will be happy to provide you with general information about how to incorporate a planned gift to the foundation into your estate plans. Matching Gifts – Many employers will match your charitable contribution. This is an extremely effective way to maximize your donation to our cause. Check with your human resources office today to see if your charitable donation can be matched by your employer. Please charge my credit card: ❑ MasterCard ❑ Visa ❑ American Express CID # (3 digit number found on the back of MasterCard and Visa) CID # (4 digit number on front of American Express cards)

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Signature: Mail to: Sarcoma Foundation of America, 9899 Main Street, Suite 204, Damascus, Maryland 20872 or fax it to: 301-253-8690 finding the cure in our time www.curesarcoma.org Survivor Photo, Ask the Experts Follow us on Educational Conference Facebook, Twitter, & Pinterest 2012 www.facebook.com/curesarcoma; www.twitter.com/curesarcoma and

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