GIST specialists give support at Miami GIST Day of Learning By Gale M. Kenny LRG Program Associate In memory of April 2013 Bob Bridgewater, Buddy Coats, Larry Ryan, Vol. 14, No.2 Larry Weaver, & David Wicker GIST Day of Learning (GDOL), a free event, was held at the University of Miami on Saturday, March 23. Spon- sored through the generous support LRG research team meets in New Jersey of Bayer, Novartis and Genentech, this event is the second in a series of By Jerry Call with at least one in-person meeting seminars focusing on the education LRG Science Director per year. This year, the team held its and support of GIST patients and in-person meeting at the LRG office their families. in New Jersey March 22-24, 2013. n 2006, the Life Raft Group initi- Attending research The event marked a continuing ated a strategic team members were collaboration between the LRG and research plan Drs. Maria-Debiec Jonathan C. Trent, M.D., PhD, a GIST – Pathway to a Rychter, Anette specialist and Professor of Medicine ICure –that empha- Duensing, Jonathan at the University of Miami Sylves- sized cooperation, Fletcher, Matt van ter Cancer Center. Over 40 patients, coordination and de Rijn, Christopher caregivers and volunteers gathered to accountability and Corless, Brian Rubin, learn, forge new friendships, and con- brought together and Sebastian Bauer. sult with some of the foremost GIST some of the finest The LRG was repre- authorities in the region. GIST researchers sented by Norman in the world. Uti- Scherzer, Jerry Call Although those in attendance came lizing four years of and Roger Camp- primarily from Florida, we were ex- garnered expertise, Brian Rubin, Sebastian Bauer, Jerry Call, bell. Objectives for cited to greet attendees from Alaska, the Research Team Chris Corless, & Jonathan Fletcher the meeting were to Australia, Tennessee and Georgia! morphed into Project report progress on Because of feedback from last year’s D-Day a four-pronged approach to Research Team projects as well as to event, old and new friends were given finding the cure involving Sequencing, discuss future directions and unmet extra time to mingle. Many were Gene Knockdown, Drug Screening meeting other GIST patients for the and Validation. See Research, Page 7 See GDOL, Page 8 The Team meets four times a year, Who’s laughing now? Actress Mo Collins uses humor in her battle with GIST By Erin Kristoff never realized that the passion she and was lucky to find a doctor who LRG Communications Director felt for making people laugh – which knew enough to know that she need- many have witnessed on Mad TV, ed to see a specialist. Parks & Recreation, or movies like o Collins “The 40-Year Old Virgin” - would the After being given neoadjuvant is not tool to battle cancer. Gleevec to shrink the 6 cm tumor, Mo your went in for surgery that December. typical Mo was diagnosed in spring 2011 by a “The hardest thing for me then was Mactress/comedi- stroke of luck when she happened to that we didn’t know going in what enne, because notice an odd lump in her abdomen. kind of surgery I would have. I was Mo Collins is a prepared for a possible Whipple. It’s a GIST survivor. “I would have done nothing about whole lot to swallow, but that’s when it. I was 44, feeling healthy, every- you really find out what you’re made Born Maureen thing was going great for me, but my of.” Collins and fiancé, Alex said, ‘You need to get it nicknamed Mo checked out’.” After surgery Mo was given great by her high news, a Whipple was not needed and school theater Alex’s insistence paid off. She had a Mo Collins teacher, she primary GIST tumor in her duodenum See Mo Collins, Page 9 Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — April 2013 — Page 2

Stivarga becomes first FDA-approved The Life Raft Group third-line drug treatment for GIST Who are we, what do we do?

Approval of Stivarga was based on By Phil Avila the results of a large multinational The Life Raft Group (LRG) directs re- LRG Newsletter Editor trial that recruited GIST patients search to find a cure for a rare cancer very quickly, requiring only eight and help those affected through sup- months to enroll nearly 200 GIST port and advocacy until we do. The Food and Drug Administration, patients. Importantly, benefit was The LRG provides support, information in a much-anticipated announce- seen in multiple subtypes of GIST. and assistance to patients and families ment for Gastrointes- The primary endpoint with a rare cancer called Gastroin- tinal Stromal Tumor of the trial was pro- testinal Stromal Tumor (GIST). The LRG patients, approved gression-free survival achieves this by providing an online Bayer Pharmaceuti- (PFS). The median community for patients and caregivers, cal’s drug, Stivarga, PFS of patients on for use as a third- Stivarga was 4.8 supporting local in-person meetings, line treatment for months compared patient education through monthly advanced GIST in to 0.9 months for newsletters and webcasts, one-on-one February. patients on place- patient consultations, and most im- bo. This PFS time portantly, managing a major research Stivarga (regorafenib) compares favorable project to find the cure for GIST. is a multi-kinase with the PFS time inhibitor that blocks of Sutent given as How to help several enzymes second-line therapy. that promote cancer Because patients that Donations to The Life Raft Group, a growth. It is now the progressed on place- only FDA-approved bo were allowed to 501(c)(3) nonprofit organization, are tax third-line treatment cross over to receive deductible in the United States. for patients who find Stivarga, there was You can donate by credit card at www. that the first-line no significant differ- liferaftgroup.org/make-a-donation/ or treatment, Novartis Pharmaceuti- ence in survival between those that by sending a check to: cal’s drug, Gleevec, and the sec- started on Stivarga and those that The Life Raft Group ond-line treatment, Pfizer’s drug, started on placebo. 155 US Highway 46, Suite 202 Sutent, are no longer effective in Wayne, NJ 07470 battling GIST. Among the possible side effects noted in the trial were liver prob- Disclaimer "This announcement by the FDA lems, hypertension, hand-foot syn- is great news for advanced GIST drome, fatigue and diarrhea. patients," said Norman Scherzer, We are patients and caregivers, not executive director of The Life Raft Dr. Michael Heinrich of Oregon doctors. Information shared is not a sub- Group. "It means they now have a Health & Science University, a GIST stitute for discussion with your new option for treatment until our specialist and member of The Life doctor. Research Team finds a cure." Raft Group’s Research Team, said: “I am excited that Stivarga (rego- Please advise Erin Kristoff, Stivarga was reviewed under the rafenib) has now been approved LRG Communications Director, at FDA’s priority review program, by the FDA. I have personally seen [email protected] of any errors. which provides an expedited six- very good results using the drug month review for drugs that may to treat patients whose tumors are provide safe and effective therapy resistant to Gleevec (imatinib) and when no satisfactory alternative Sutent (sunitinib). Besides pro- therapy exists, or offer significant viding hope and an effective new treatment, the approval of Stivarga orectal cancer will also help encourage other drug that has pro- companies to continue to develop gressed after new drugs to treat GIST.” treatment and spread to other Richard Pazdur, M.D., director of the parts of the Office of Hematology and Oncolo- body. gy Products in the FDA’s Center for Drug Evaluation and Research, said Gleevec re- Stivarga “provides an important ceived acceler- improvement compared to mar- new treatment option for patients ated approval as keted products. The drug was also with GIST in which other approved a first-line treat- granted orphan product designation drugs are no longer effective.” ment in 2002, while Sutent won FDA because it is intended to treat a approval as a second-line treatment in rare disease. Last September, the FDA approved 2006. About 5,000 new cases of GIST Stivarga to treat patients with col- are diagnosed each year in the U.S. Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — April 2013 — Page 3 Family planning with GIST can be a hard decision By Phil Avila One of the more comprehensive sur- had another surgery. She is now on LRG Newsletter Editor veys can be found at 600 mg of Gleevec. bit.ly/imatinibpregnancy. “Women have to weigh the pros and Family planning can be one of the Novartis Pharmaceuticals, maker of cons,” she said. hardest decisions patients with GIST Gleevec, the FDA’s approved first line make, but for Caroli- treatment for GIST, and Barbi Appelquist has thought a lot na Ponce Williams the Tasigna, a drug that about family planning and how it decision was easy. while not approved for affects GIST patients like her. “There GIST has been taken are a lot of issues having a baby even At high risk for pro- by some GIST patient, in perfect health,” she said. These gression, Carolina is currently conducting are magnified with GIST. Women, she longed to start a fam- a clinical trial on preg- acknowledged, have different views ily. So she decided to nancy and exposure to on contraception and abortion, for adopt. those drugs but results example. won’t be ready until at “It was pretty easy least 2014. See 1.usa. “There are still so many unknowns, for me to decide,” she gov/11SmxhO. still so much we don’t know about Carolina Williams, her son said. “I know adoption Antonio, and her husband Terrance GIST,” she said. “Part of you wants to is not for everyone. But Until then, most doc- know the answers.” I had adopted cousins so I knew what tors, like Carolina’s, advise patients to expect.” not to have a baby or go off Gleevec if Barbi said she has put off discussing they are at risk for progression. How- family planning with her husband until Her son, Antonio, now 3 years old, “is ever, some doctors, with patients with she is one-year post-surgery. such an amazing child,” she said. She low risk for progression who want to said she thinks about adopting anoth- have a baby, work with Since these issues are er child everyday, but that will depend patients to go off the so personal in nature, on Antonio. medication for a year to she suggested an give birth. email community be Having her own biological child would created for women of have meant going off of her drug That was the case for child-bearing age with Gleevec for a year, a risk she didn’t April Stephens. She had GIST. “I didn’t feel very want to take. Her advice to other clean scans for five years supported by the gen- women: “make sure you investigate it before working with her eral GIST email com- thoroughly.” doctor to go off Gleevec munity,” she said. April Stephens, her daughter to give birth to her Mary Catherine and her husband While there isn’t a lot of data on daughter, Mary Cath- Heath “I can’t imagine what pregnancy and GIST, the prescription erine, who is now five it would be like to be information on first-line drug Gleevec years old and healthy. “I followed all 18 and dealing with these issue,” she clearly warns of risks if a women of my doctor’s guidelines,” she said. said. “That’s a tough question.” stays on Gleevec during pregnancy and advises patients to use a form of “I wouldn’t trade it for anything,” April This is the first in a series of articles birth control. Several studies indicate said. It was the first time a pediatric on family planning and GIST. We will mixed results for women who stayed member of the Life Raft Group had a also explore being a mother while on on Gleevec during pregnancy, with child. Gleevec and considerations for men some babies being born with similar with GIST who want to have chil- deformities and others without com- After giving birth in 2007, April went dren. If you’d like to comment for plications. back on 400 mg of Gleevec. In 2011, these articles, contact Phil Avila at scans showed progression and she [email protected]. Pediatric GIST patient welcomes baby boy!

After a long and arduous labor that made the Marine Corps Marathon seem like a 5K, Jill and I are proud to introduce our firstborn son and first child of the DeLorenzo Family, Simon Joseph.

He was born at 6:58am on 3/24/13. 6 lbs. 11oz. 20 inches long. Simon is a wide-eyed, very attentive and curious baby. He unfortunately has a lot of his daddy's features, like bushy eyebrows, pointy ears, and long fingers; but has his mom's chin and nose. So far, what we know is he likes computer screens, and didn't seem to like flash photography. He also likes taking his time after a 29 hour labor. But with a positive and motivated attitude that destroyed all negative stereotypes daddy had about women in childbirth, mother and Simon are doing great. Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — April 2013 — Page 4 How to make the most of LRG website By Jim Napier on the website, you may have noticed small box will open up allowing other LRG Website Manager two important elements. First, at the sharing options including a way to bottom of each post, we have sharing email and print the article. options and a comment section. Both s you read in our last news- are great ways for you the reader to The comment system that we use letter, the Life Raft Group has get involved in the conversation and is called Disqus, and it is very user launched a friendly. Many sites new web- use this form of Asite. If you have comments because not already, be it allows you to sure to visit the organize all com- new site at liferaft- menting you may group.org for up- do on the web in to-date news and one place. Simply information about type your com- GIST Cancer. ment in the open box, and then The website uses choose which op- WordPress, which tion to log in with, is widely con- Facebook, Twitter, sidered the gold Google or Disqus standard of con- so you can publicly tent management post a comment. If systems. It allows you would rather the LRG to create post anonymous- content on both ly, we offer that pages and posts option as well. (aka articles). Whether it is the The next time you announcement of are reading an Stivarga, an update on Clinical Trials, share these articles with your friends. article on the site, consider sharing it or Janeen's Top Ten Valentine's Day and leaving us a comment! For more Tips, the LRG is able to share engag- For the sharing icons, we feature information on how to use these fea- ing and informative content quickly a Facebook Share, a Twitter Tweet tures email Jim Napier at and easily. button, a Google + button, and then [email protected]. a button for other sharing options. If If you have read any of our articles you scroll your mouse over the icon, a Pioneer Dan Vasella gave the gift of time By Erin Kristoff great achievements made by some by thanking Dan for the gift of time LRG Communications Director of his life-long business colleagues he had given to Norman and his wife, and friends. But when Norman took Anita. ecently, LRG Executive Director the stage, he kept his speech simple Norman Scherzer had the He presented Dan a framed clock opportunity to speak at the which also had a picture of Nor- retirement celebration for man, Anita and their seven grand- Rformer Novartis CEO and Chairman children in Disney World. It was of the Board, Dr. Dan Vasella in a trip Norman had promised his Basel, Switzerland. family if Anita became well again, which at that pre-Gleevec time As CEO, Dr. Vasella was instru- seemed unlikely. Next to the clock mental in getting Gleevec (then and photo were notes written by STI-571) to market for a fairly Norman’s children and grandchil- small patient population, but after dren also thanking Dr. Vasella for 25 years at Novartis and Sandoz, the gift of many more days with he has chosen to step down as their beloved mother and grand- Chairman. mother.

There were many illustrious speak- It was emotional moment for ers at the event, including a very everyone in attendance that day cheeky speech made by Novartis’ and for Norman and Dan Vasella architect, Marco Serra, and pow- Daniel Vasella, former chairman and in particular. erful speeches about Dr. Vasella’s CEO of Novartis Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — April 2013 — Page 5 Distribution by State of U.S. GIST Patients in LRG Registry

By Roberto Pazmino Texas with 71, Florida with 66, Illinois researchers find a cure. We currently LRG Administrative Director with 63, and Michigan with 51. Fifteen have about 1,600 patients in the Reg- other states have 20 or more patients istry from all over the world. in the Registry. Those states with high e have put together a map numbers of patients range from the We are working on an interactive map showing the distribution of East to the West Coast, with many showing patients, GIST specialists patients in the U.S. who are states in between. and treatment facilities that can be living with GIST in the LRG searched using multiple criteria. WPatient Registry by state. The map The geographical diversity of the shows how geographically diverse Patient Registry makes it a valuable If you are not already participating in the Patient Registry is, with patients tool for planning events and local the Patient Registry, you may do so spread throughout the country. meetings and for use in clinical trial by joining here: liferaftgroup.org/ recruitment. The Registry is used patient-registry/. California has the most patients with in conjunction with the LRG’s GIST 140, followed by New York with 74, Collaborative Tissue Bank to help LRG Advocates for Cancer Funding in New Jersey The LRG is continually involved in testified before the Senate Budget financial necessity will move to loca- advocacy efforts to increase govern- Committee last week and the Assem- tions such as Texas, North Carolina ment spending on cancer research. bly Budget Committee yesterday to and Florida — states that have made We have joined forces with other highlight the importance of funding a significant commitment to financing cancer agencies to once again urge cancer research in New Jersey, and cancer research.” New Jersey Governor Chris Christie urging the legislature to restore the and the New Jersey Legislature to $1 million budget appropriation. Last year, joint efforts by the Life restore the $1 million budget appro- In a story in the Star Ledger, Dr. Raft Group and several other cancer priation for cancer research in the Gammies revealed that NJ residents organizations helped to restore the state. are moving out-of-state for treat- funding. We hope for the same out- ment, and states: come this year. Dr. Alison Gammie, a cancer re- “New Jersey risks losing some of its searcher from Princeton University most brilliant citizens, who out of Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — April 2013 — Page 6 Larry Ryan lived to love, loved to live insurmountable odds several times drawled, “Well yer gonna be here fer By Janeen Ryan against this incurable cancer. The di- quite some time, that thar is a drain- LRG Outreach Coordinator agnosis was terrible—the side effects, age ditch.” Larry pulled out his line, the weakness, surgeries…. All of it which was covered with crawdads. wore us down. But it also worked as He turned to the man and said, “Look arry Ryan was born in Brooklyn, the catalyst that brought the family there, I caught some hors d’oeuvers New York, on September 25, back together. Larry’s children and for tonight,” then proceeded to walk 1945; he passed from this world my own family were able to overcome back up the bank, dropping his “hors into the loving past issues and d’oeuvers” along the way. Oh, how his Larms of Christ on band together to quick mind amazed me, always one February 25, 2013. form a strong bond step ahead and quick with a wink. He was holding that would help to tight to my hand as sustain us all over Dashing our hopes, tumors returned he left, to his last the next six years. in December 2010. Surgery followed breath he tried not at Dana Farber, but traveling in the to leave me. He put I asked him once RV was no longer possible. Heartbro- up a valiant battle if he could change ken, he and I returned home to Arizo- with GIST for over anything, what na in 2011 to be near the support of six years, but he would it be? He said our family. In June of 2012, he could could fight no more. he’d have taken no longer tolerate oral chemo and more golf lessons entered Hospice. I met Larry when I and completed was interviewing for his pilot’s license In 2006, when we first heard the a job at a VW deal- training. I asked, words, “We’ve found a mass,” we ership. During our interview we talked wouldn’t you choose not to have got- were overcome with grief. When the for an hour and forty-five minutes but ten cancer? He said he didn’t choose news had sunk in and the tears had only spoke of the job for five or 10. to get it in the first place, how could dried, Larry looked at me and said, We hit it off like old friends—a spark he choose not to? Besides, he said, it “I feel so sorry for you, the caregiver was ignited that day, and unknown to brought his family back together. . . has the toughest job.” Even then he me for months, Larry said he fell in That’s all that mattered. was thinking of me. He had cared for love that day. For me a special con- his first wife through her cancer and nection was made and Larry became In 2009, we were losing our house, so in the years he fought GIST he some- my best friend very quickly. Then he we decided to sell everything in it and times expressed regret that he had began to ask me out, repeatedly! buy an RV so we could travel! Larry not known how to be an advocate for But I had two children and could not had recovered from his liver surgery her as I had been for him. afford to lose my job over an office and became well enough to travel the romance. Nowadays that would be country in an RV with me for 18 glo- In the last months of his life, Larry considered a no-no but rules never rious months. We enjoyed adventures told me several times that if I could meant much to Larry. Months later he from the Oregon coast (his favorite) continue, if I could use the experi- entered one more time into my office to Cape Cod, Massachusetts (my ences and lessons we’d learned as a and said, “I just quit my job, you got favorite) and everywhere in-between. team fighting for his life against GIST, a promotion to my position, no more We were chased by a tornado in Okla- then his death would not be in vain. excuses, now will you go out with homa, nearly flooded in Wako, Texas. The cancer would not have won, but me?” I was so surprised I said, “Of Drove inland by a hurricane on Cape only served to continue to be the course I will.” Heck, I’d marry him! Cod, and treated driving force behind And I did, just seven months later to the beauty and my resolve to help in a tiny chapel in the New Mexico majesty of watch- others who are walking desert. ing every change the path of surviving of season known this diagnosis. I prom- Larry delighted in spoiling me; lit- to the U.S. ised him I would. He tle gifts would show up around the wanted his legacy to house. Flowers, sweet cards, and oth- Larry loved to be the continued sup- er thoughtful gestures from him were fish—he tossed port and assistance of something I got to enjoy for 15 years. his line in the Pa- GISTers everywhere. I loved him fully and completely, as cific, the Atlantic, It’s just like him to did he me. All this showed me I had lakes, rivers, and be thinking of others found my true love, my forever mate, streams, any- at the end of his life, and we both basked in the joy of where he could even strangers, only finding each other. This was a second find to enjoy the connected by this one chance for each of us, and we reveled water and the tug of a fish on his common thread. It shouldn’t surprise in the good fortune we felt was a spe- line. I remember once he was sit- anyone, as Larry never once asked, cial gift from above. ting by a river in East Texas with his “Why me?” He never complained, line in the water and I was reading but often tried to console others who In December of 2006, Larry began a book nearby. A man approached were upset for him. Now he watches his journey with GIST. He won many and asked what Larry expected to over me, my guardian angel, forever battles with the help of The Life Raft catch. Larry said he didn’t know, just perfect— forever my love. Group. Larry overcame seemingly whatever is in the water. The man Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — April 2013 — Page 7 RESEARCH From Page 1 needs. It is important to note that the develop from giving the two drugs to- needed on more cases. In order to do two-day meeting provides a chance gether in sequence. This is an innova- this, wildtype samples from the LRG to share the progress of each lab in a tive way to try to improve outcomes tissue bank will be studied; but more more detailed manner than is possible for patients using all the tools proven cases are needed. Wildtype patients any other way. There are some major effective against this disease. that are not currently participating in differences in the way that the LRG the tissue bank are welcome to par- Research Team operates compared Dr. Chris Corless, reporting for the ticipate in this research. See the Life to traditional research. First, they Heinrich/Corless labs, reported new Raft Group website for details about are not focused on 10 or 20 different data about methylation status of the how to participate in the LRG tissue cancers; the SDH proteins in bank and LRG GIST Patient Registry. focus of each pediatric-like GIST lab is on GIST. (SDH deficient In other SDH-related news, LRG In addition, GIST). Mutations Research Team member Dr. Brian the research in one of the SDH Rubin has managed to locate a SDH labs share data subunits (either deficient paraganglioma cell line. He on an ongoing SDHA, SDHB, is currently working on access to that basis. This is SDHC or SDHD) cell line. A recent study in a yeast much different have been report- model of cells with SDH deficient than the tradi- ed in about half suggested several drugs that might tional model in of pediatric-like be effective in cells with these defi- which research- GIST cases. ciencies. The paraganglioma cell line, ers operate in However, all pe- if available, should allow for further more isolation diatric-like GIST drug testing in a cell line that more and data is not patients seem closely resembles the GISTs and para- distributed until Jonathan Fletcher, Maria Debiec-Rychter, Anette to have a defec- gangliomas that occur in patients. the publication Duensing, & Matt van de Rijn tive SDH protein. As reported at the meeting, tech- cycle is com- Pediatric-like GIST nology to detect very small bits of plete. This makes the data a year or patients stain negative (indicating the DNA that are circulating in plasma is more old before it is distributed. protein is missing or defective) for getting better. In theory, this tech- the SDHB protein and all other GIST nology could be used to identify GIST Highlights from this year’s patients stain positive for this protein. patients at risk of relapse due to re- meeting A change in methylation has been sistance or after surgery or adjuvant suggested as a possible aberration in therapy from a simple blood draw. Dr. Jonathan Fletcher’s lab, working pediatric-like GIST by Dr. Katherine Plans were made to start looking at with Drs. Suzanne George, George Janeway. the logistical requirements for incor- Demetri and colleagues at Dana-Far- porating plasma collection into the ber Cancer Institute, as well as with Methylation is an important regu- LRG tissue bank. Dr. Michael Heinrich at Oregon Health lator of which genes are used in a and Science University, has identi- cell. Alterations in DNA methylation In summary, LRG research team fied resistance mutations (secondary are common in a variety of tumors. members are continuing to examine mutations) associated with resistance Alterations include all possible avenues to Stivarga (regorafenib). Stivarga hypermethylation in their search for a was approved February 25, 2013 in (too much) and cure for GIST. The the United States as a third-line ther- hypomethylation research mentioned apy in GIST, and is currently under (too little). One here, which rep- review for approval in Europe and consequence of hy- resents a small part across the world (Approved in Japan). permethylation can of the data report- Some patients benefit from Stivarga include silencing ed in the meeting, for significant periods of time, but of tumor suppres- directly affects par- the typical duration of benefit (me- sor genes. Several ticular subsets of dian time before progression) was drugs are avail- patients with GIST, about five months in the Phase III able that target such as those who clinical trial. Guided by recent lab hypermethylation would benefit from insights, the Boston group is studying in cancer. At this combination thera- approaches that combine Stivarga time, it is unknown Some of the research team members at pies and those with with other drugs to maximize clinical if these drugs would dinner with LRG Executive Director, specific mutation benefit. One possibility that is being be appropriate for Norman Scherzer types. It also bene- intensively pursued is to sequentially SDH-deficient tumors (or a subset of fits the GIST community as a whole, administer Stivarga with a second ef- them). however, as lessons learned may be fective drug in GIST. This plan would transferrable to other subtypes, and require a clinical trial to test the dose Dr. Corless noted a change in meth- every approach eliminated helps us and schedule, and to monitor for new ylation for a small group of pediat- narrow our focus and ultimately find or unexpected toxicities that might ric-like GIST. Additional research is the correct one. Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — April 2013 — Page 8 GDOL From Page 1 first time, and expressed feeling both completed a number of resections He further underscored the crucial inspiration and optimism from this for GIST patients previously deemed role mutational testing has in direct- experience. inoperable by outside facilities. ing a course of treatment, and recom- mended this be determined for every Contributing to the tremendous Lunch was combined with “A Roundta- GIST that is diagnosed. success of the event were the GIST ble Discussion on Side Effects”, an im- professionals who all displayed an portant topic led by Elizabeth Fontao, “Clinical Trials Explained and Ex- exemplary spirit of MS, and PA-C. plored” was discussed by Dr. Trent. giving and dedica- Ms. Fontao, a After a review of the trials across the tion for the benefit licensed and cer- United States, Dr. Trent discussed of this unique com- tified Physician the importance of these efforts in munity of patients. Assistant, has identifying new medications for GIST. We are thankful to extensive expe- Fourth and fifth-line drugs are cur- the University of rience in oncolo- rently being sought and there is a Miami for provid- gy, and currently great need for GIST patient participa- ing the venue, and dedicates much tion. wish to acknowl- of her time to edge their team of sarcoma and This event was a result of many specialists, who are GIST. She works people from various locations working leaders in provid- in concert with together, and we wish to extend our ing excellence in Dr. Trent at gratitude to all who offered their time GIST diagnosis, the Sylvester and talent. We would like to send a management and Comprehensive special thank you to Alice Sulkows- awareness. Dr. Jonathan Trent presenting at GIST Day of Cancer Center, ki, R.D., C.D.E. A nutritionist, a GIST Learning in Miami, Fla. where she is survivor, and a longtime LRG friend, Dr. Trent, whose responsible for Alice provided handouts on “Nutrition cooperation has made GDOL possi- patient chemo and immune therapy and GIST” for all our attendees. She ble, is the Co-Director of the Mus- support, side effect management and works at Mountain States Health Alli- culoskeletal Center, Director of the follow-up care. The format of this ance in Johnson City, Tennessee. Sarcoma Medical Research Program, segment bolstered the participation and Professor at the University of of the attendees and individual side We also wish to acknowledge with Miami (UM). His practice is located effect woes were brought up and gratitude our sponsors, Bayer, Genen- at the Sylvester Comprehensive Care addressed. tech and Novartis for their generous Center. Outstanding in his field, his support of the meeting. In addition research findings on GIST have been A lecture on “Pathology and Role we extend a befitting thank you to presented in several esteemed med- of Mutations in GIST Treatments” Dr. Derek Naten, Kristen Alleque and ical publications. In addition to facili- was presented Richard Proctor tating the meeting, he shared his vast by Andrew E. from Bayer for their knowledge in “GIST Overview” and Rosenberg, M.D., attendance and spoke on “Clinical Trials Explained and Chief of Anatomic unwavering support Explored.” Pathology, Direc- for our educational tor of Bone and events. Alan S. Livingstone M.D., FACS, is Soft Tissue Ser- Professor and Chairman of the Lucille vice and Professor Pictures from the and DeWitt Daughtry Department of of Pathology at event were courte- Surgery at the University Of Miami the University of sy of our volunteer Miller School Of Medicine, where he Miami Sylvester photographer Don maintains a busy surgical practice in Comprehensive Edgar of DL Pho- addition to an active involvement in Care Center. Dr. tography based in translational research for a variety of Rosenberg has Miami. Don is dou- cancers. He is the author of numerous published exten- ble thanked, as this books, book chapters, peer reviewed sively and actively is the second GDOL surgical periodicals, has lectured consults for the Dr. Rosenburg giving his presentation at event he worked GIST Day of Learning in Miami, Fla. world-wide and has a prime interest College of Amer- for us and we are in educating the medical students ican Pathologists truly appreciative. and residents at UM. His presentation and The American Joint Commission It was our good fortune to locate on “Surgery and Other Options” was on Cancer, serves on several Edito- Phillip Lwiza, a Miami-based Videog- enormously informative, and offered rial Boards and has been an invited rapher to offer his discerning eye and a variety of slides from actual surgical lecturer at local and international equipment in the recording of this resections on advanced GIST tumors. medical conferences. His slides and event. Thank you both for providing It is without question that Dr. Living- accompanying information offered a your photographic perspective to this stone sets the standard in advanced comprehensive explanation of what important meeting. surgical expertise; having recently GIST looks like under a microscope. Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — April 2013 — Page 9 Mo Collins From Page 1 they had extracted the tumor with positive and then I feel better.” by staying fit. clear margins. “That had to be the greatest moment of my life when I However, life for Mo isn’t always a “What I started to do is bench press- first woke from surgery and they told walk in the park. Mo, like most GIS- ing. It takes 10-15 minutes, and my me I had plan A, which was the sweet Alex trains me. When I’m good plan. There was nothing in done I’m not physically exhausted, my life before that could compare I’m not run down, I’m strong. I’ve to that feeling.” never been able to do a pushup and now I do three sets of them Now, Mo is over a year NED and post-GIST! I like that it makes me keeping a positive outlook on life feel strong.” despite GIST, partially because she has seen first-hand the neg- But it’s her passion for laughter ative effects fear & worry can that is her biggest weapon against have on a body. Within the month GIST, “My gift here on this plan- before starting treatment, Mo’s Mo Collins, as Joan Callamezzo planking on “Parks et is that I’m a story-teller and fear and anxiety over her cancer and Recreation” a funny person. I need to do it. diagnosis dominated her life. I need to get up there and laugh Ters, struggles with side-effects like and I need other people to laugh with “Within the month before I started fatigue and edema of the eyes, which me. It’s kind of selfish actually.” treatment, my tumor started growing can make her look worn down and so fast that it increased from five cm drunk-looking. For an actress it can But Mo is using this gift in a very to six cm and I realized the power of be a particular challenge, “I call them selfless way by organizing a comedy fear. I realized I had to find a way to my fetal pig eyes. For me to audition fundraiser for the Life Raft Group and not be afraid. Fear is something that and go on camera, GIST research. Bravely we create.” it takes a whole dif- “Nutrition and exercise coming out as a can- ferent kind of mental cer survivor in a tough Now Mo is using the power of positive strength.” keep me above my fear. business, Mo is using thinking each and every day, “I talk They build my armor. her diagnosis experi- to myself a lot. I tell myself that it’s In fact, her recurring ence to try to increase just a cell—I diminutize it. Nutrition role as Joan Callamez- I feel like I’m winning. GIST awareness so and exercise keep me above my fear. zo on NBC’s Parks & I say, ‘This is good for that no patient goes to They build my armor. I feel like I’m Recreation has been me; there’s no way a doctor that doesn’t winning. I say, ‘This is good for me; a blessing in disguise. know what GIST is. there’s no way cancer can get through “Joan has been a drunk cancer can get through this.” lately so it’s kind of a this.” “I want to go have fun. relief.” I need this for me. Mo believes that the battle against But we need money GIST is fought not just with treat- Besides Parks & Recreation, Mo has for research. This is part of what I’m ments and surgeries but within your- been doing a lot of voice work, which supposed to do—talk about it, laugh self, “You’ve got to stay happy. You can be a safe haven of sorts, “I can about it, and shed some light on it need to stay mentally on top of this. sit and do it and not be tired!” so that others who don’t understand That’s where the battle is truly won. GIST may come to understand it be- I’ll do everything I can to stay on top While fatigue is a big problem for her, cause of the humor.” of it, I’ll do the things I need to get in typical Mo-fashion she’s battling it Help the LRG in the search for a cure!

Wildtype GIST tissue is needed for a current LRG Research Team project. This project, briefly described on page 7, will look at SDH methylation status in wildtype GISTs. If you or a loved one are a wildtype GIST patient, please help the LRG Re- search Team in its search for the cure by donating your tissue. See the LRG website for instructions on joining the LRG Tissue Bank and GIST Patient Registry or call (973) 837-9092 or email us at [email protected] for assistance. The process is easy—the tissue required is taken from an existing paraffin block held in your hospital, so no new biopsy is required.

Note: We are always looking to add more tissue to our Tissue Bank, so regardless of type, if you are thinking of ways to support research, please consider donating your tissue today. Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — April 2013 — Page 10 ‘Buddy’ Coats Jr. passes away larence Charles “Buddy” Coats degree in education from Indiana Uni- Henryville; grandchildren, Bryce and Jr., 65, of Jeffersonville, In- versity. He was a retired school teach- Brandon Romig and Xavier, Zerach, diana, died Sunday, April 22, er, a member of Southeast Christian and Zidon Coats, of Henryville; and 2012, at his home. He was Church, Jeffersonville Masonic Lodge stepdaughter, Kara Hennis and hus- Cburied in Walnut Ridge Cemetery. 340, Scottish Rite, and served on the band, Jeff, of Jeffersonville. He was West Clark School Board. preceded in death by his daughter, He was born June 3, 1946, to the late Faith Michelle Romig, and parents, Bud and Dorothy Coats. He was a He is survived by his loving wife, Clarence Sr. and Dorothy Coats. Ex- 1964 graduate of Jeffersonville High Denise Coats;, a son, Clarence “Bud- pression of sympathy may be made to School and received his master’s dy” Coats III and wife, Melinda, of Kosair Charities.

Audit report gives glimpse into priorities

of America, those standards require statements of functional expenses. By Roberto Pazmino that the auditor plan and perform the Administrative Director audit to obtain reasonable assurance Last year expenses distribution per- about whether the financial state- centages were as follows: ments are free of material misstate- Program Services: very year, the Life Raft Group’s ment. An audit includes examining, Research 76% financial statements are au- on a test basis, evidence supporting Family Services 4% dited, providing a glimpse into the amounts and disclosures in the Advocacy 4% the priorities of the organiza- financial statements. An audit also Information and Education 8% Etion. Audited results for 2012 will be includes assessing the accounting Management & General 4% received soon. principles used and significant esti- Fundraising 4% mates made by management, as well Operating expenses are broken down as evaluating the overall financial The 990 also documents the tremen- between program services, which statement presentation. dous amount of time our volunteers include research, family services, give to help the GIST community. advocacy, and information and edu- In every audit one specific section is cation; fundraising, and operational the contributions audit, all contribu- We will be posting our 2012 audited expenses such as management of tions including unconditional prom- results on our website as soon as the nonprofit. These financial state- ise to give, are recorded as made. they are completed. ments are the responsibility of the All contributions are available for organization’s management. unrestricted use unless specifically You can see our budget philosophy restricted by the donor. Cost of pro- and all previously filed 990 at Audits must be conducted in accor- viding the organization’s programs, liferaftgroup.org/budget-and- dance with auditing standards gen- management and fundraising activi- operating-philosophy/ erally accepted in the United States ties are separately summarized in the Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — April 2013 — Page 11 Bulgarian GIST group increases reach some leaflets with useful information, Our organization is a member of By Juliana Popova providing psychological support for SPAEN (www.sarcoma-patients.eu/) GIST Alliance for Patients-Bulgaria people with GIST and assisting them and we attended the SPAEN Annu- in obtaining the appropriate treat- al Conference in November 2012, ment. when we presented GIST Alliance for y name is Juliana Popova and Patients –Bulgaria. Also received an I’m the chairperson of the Bul- In June 2012, the first national meet- invitation to attend the New Horizon Mgarian support group for people ing of GIST patients in Bulgaria was GIST Conference in Miami this year. with GIST – “GIST Alliance for Pa- held in Sofia. In December, we start- tients-Bulgaria”. ed a training seminar about different At present, I’m trying to reach more conditions in our treatment. GISTers (I’m in touch with about 40), Our organization was founded in Jan- The number of GIST patients on to continue with the training semi- uary 2012. All patients can contact us treatment is constantly increasing, nars, and to help establish a center by e-mail at [email protected] or by and now there are more than 80. for GIST treatment. The local prob- phone at: +359 888 585 366; +359 There is no clinical register for GIST lems in Bulgaria are quite different. 889 242 228. We have already done patients in Bulgaria, so my informa- For example, here the Gleevec patent some activities, such as creating a tion may not be exact. has expired and the first generic ima- website, www.gist-bg.com/, making tinib is coming. Around the World...Chile Edition

Piga Fernandez, the LRG’s global relations coordinator, partici- Joel Salazar, a Chilean GIST patient, is running marathons to pated in an activity called “I have cancer, …lets talk” organized raise awareness of the need for GIST to be codified and ap- by Red Chile Cancer, a coalition of cancer patient groups in proved as a pathology by the health system in Chile. Chile. The main objective was to let people know the reality of GIST patients in Chile and create awareness of the need that different types of cancer are covered by the health systems.

for details about the events check out Carol Heller to be LRG CALENDAR liferaftgroup.org/events local leader in GDOL Chicago NIH th th th Eastern Pennsylvania May 5 2013 June 26 -28 2013 Carol Heller will be working with Jeff ASCO Night to Fight Cancer Bernstein to cover the state of Penn- May 31st-June 4th 2013 September 12th 2013 sylvania as local group leaders. She is very excited to help and hold meet- New Horizons The Art of the Cure Gala ings, and will be handling the eastern th th th region of the state. June 5 -7 2013 November 7 2013

Carol can be reached at Alianza GIST [email protected]. June 7th-9th 2013 Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — April 2013 — Page 12 THE LIFE RAFT GROUP LRG STAFF & CONSULTANTS LRG VOLUNTEERS BOARD OF DIRECTORS Executive Director Norman Scherzer Pediatric GIST Erin McBean Executive Committee Deputy Executive Director Roger Campbell Database Coordinator Steven Rigg Jerry Cudzil — President Sr. Executive Assistant Diana Nieves Official Greeter Gail Mansfield Stan Bunn — Past President Science Director Jerry Call Latin America Liason Vicky Ossio Ray Montague — Secretary/Treasurer Special Projects Coordinator Pete Knox Clinical Trials Coordinator Jim Hughes Administrative Director Roberto Pazmino Photographer Kim Tallau Mia Byrne Program Director Sara Rothschild Member Birthday Coordinator Mary Kluth Chris Carley Development Director Christine Schaumburg Special Projects Asst. Eileen Glasser Gary Glasser Newsletter Editor Phil Avila Science Team Tanya DeSanto Jim Hughes Program Associate Eola Bakhru Jim Hughes Jerry Knapp Program Associate Michelle Durborow David Josephy Dan Miller Asst. Development Director Lisa Hart Michael Josephy John Poss Communications Director Erin Kristoff Rick Ware David Safford Web Programmer James Lee Glenn Wishon Rodrigo Salas Office Manager Matthew Mattioli Paula Vettel Larry Selkovits Patient Registry Supervisor Magda Sarnas Global Relations Coordinator Piga Fernandez Bookkeeper Debbie Haroldsen Program Associate Gale Kenny Program Associate Jim Napier Outreach Coordinator Janeen Ryan Program Associate William Sumas

Life Raft regional chapters: Find your reps info at liferaftgroup.org/find-a-support-group/

A l a b a m a Pat George Louisiana Jackie Welsh O h i o Maryann Snowbrick Alaska Jill Heinrich M a i n e Jodi Merry O k l a h o m a Jane Rowan A r i z o n a Janeen Ryan M a r y l a n d Bonnie Emerson O r e g o n Gail Mansfield C o l o r a d o Ann Bridgewater M i c h i g a n Ellen Rosenthal Pennsylvania Jeffrey Bernstein C a l i f o r n i a Dina Wiley M i n n e s o t a Ananth Pai Carol Heller Martha Zielinski M i s s o u r i Katie Bloss R h o d e I s l a n d Susan Farmer D e l a w a r e Cindy Bones M o n t a n a Dirk Niebaum South Carolina Paula Stover F l o r i d a Skip Ryan N e b r a s k a Sally Norton Tennessee Alice Sulkowski G e o r g i a Pat Lemeshka N e v a d a Joan Smith T e x a s Dee Hawkins I d a h o Janet Conley New Hampshire Julie Thorne U t a h Mike Ginsberg I l l i n o i s Jim Hughes N e w J e r s e y Anita Getler V i r g i n i a Sally Jackson I n x d i a n a Debbie Farmer N e w Y o r k Pat Bonda Swenson W a s h i n g t o n Deanne Snodgrass I o w a Barbara Kepple North Carolina Chuck Korte W i s c o n s i n Rick Ware

Life Raft country liasons: Learn more about the Global GIST Network c& find contact info for your rep at www.globalgistnetwork.com

A r g e n t i n a María Isabel Gómez H u n g a r y Tünde Kazda Poland Piotr Fonrobert A u s t r a l i a Roy Neill I n d i a Paresh Majmudar P u e r t o R i c o Gerardo Silva B e l g i u m Kris Heyman I r a n Negar Amirfarhad R o m a n i a Simona Ene B o l i v i a Virginia Ossio I r e l a n d Carol Jones Russia Tanya Soldak B u l g a r i a Juliana Popova I s r a e l Avi Zigdon S a m o a John Galuvao B r a z i l Valeria Hartt I t a l y Anna Costato Saudi Arabia Mohamed-Elbagir Ahmed C a n a d a David Josephy Japan Sumito Nishidate Scotland Helena Koumbouzis C h i l e Piga Fernández J o r d a n Mohammed Milhem S i n g a p o r e Robert Richardson C h i n a Ruijia Mu Kenya Francis Kariuki S o u t h A f r i c a Annette Mentasti C o l o m b i a Maria Helena Matamaa K o r e a HyunJung Yang S o u t h K o r e a Hyun Jung Yang C o s t a R i c a Michael Josephy M a c e d o n i a Dejan Krstevski Spain Luis Herrero C y p r u s George Constantinou M a l a y s i a Yong Choo Sian Sudan Mohamed-Elbagir Ahmed Czech Republic Jan Pelouchová M e x i c o Rodrigo Salas Sweden Susanna Allgurin Neikter Dominican Rep. Alejandro Miranda N a m i b i a Lon Garber S w i t z e r l a n d Helga Schnorf Finland Mirja Voutilainene N e w Z e a l a n d Marie Lagaluga T h a i l a n d Kittikhun Pornpakakul F r a n c e Estelle LeCointe Netherlands Contactgroep GIST T u r k e y Haver Tanbay G e r m a n y Markus Wartenberg N i c a r a g u a Maria Teresa Ponce U . K . Judith Robinson G r e e c e George Constantinou N o r w a y Odd Andreas Tofteng U r u g u a y Fabrizio Martilotta H o n d u r a s Xiomara Barrientos Pakistan Muhammad Shahid afique Venezuela Melisa Biman G u a t e m a l a Silvia Castillo de Armas P e r u Maurice Mayrides

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