Battling gastrointestinal stromal tumor Personalized medicine: is it LLIFEIFE RRAFTAFT myth or reality? GGROUPROUP Executive Director’s report focuses on better treatment In memory of Doug Horst, Michael Lerner, Eric Pontes, William Clyde January/ Turner, Dan Butt, Richard Bodden, Ann Kremer, Ron McLelland, John Vol 14, No. 1 February 2013 Aloise, Nely Jaques, George Logan, Bob Book & Maura Cesarini options for GISTers

LRG Research Team members publish By Norman Scherzer LRG Executive Director results of imatinib combination study or my after treatment withdrawal.” The study Execu- By Pete Knox tive Di- LRG Special Projects Coordinator looked to compare each drug separately, as well as both drugs in combination to rector’s Freport, I usually rs. Jonathan Fletcher, of see which approach was most effective. The study design was as follows: 136 highlight the many Brigham & Women’s Hos- accomplishments pital, Harvard University nude mice (mice with a compromised immune system) had human GIST tu- of the Life Raft and Maria Debiec-Rychter, Group. Some of Dof the Catholic University in Leuven, mors introduced into their systems (a SCHERZER which include: Belgium, members of the LRG Research total of six GIST xenograft models were used). They were then divided into four  Launching a Team were part of a recently published GIST Day of Learning program in study that looked at the effectiveness of different groups and had their tumors assessed after four weeks: Miami, which is being expanded in GDC-0941, a PI3K inhibitor when used 2013 in combination with imatinib (Gleevec). 1. Control (no treatment) 2. GDC-0941 only  Using our GIST Collaborative Tis- The study, published in Clinical Cancer sue Bank to help physicians in Latin Research (see http://clincancerres. 3. Imatinib only 4. GDC-0941 and imatinib in combi- America identify misdiagnosed aacrjournals.org for the abstract), was GIST cases entitled “A potent combination of the nation Groups three and four (imatinib and  Major presentations to the FDA, novel PI3K inhibitor, GDC-0941, with FasterCures & CTOS imatinib in gastrointestinal stromal tu- the combined treatments) also had an mor xenografts: long-lasting responses See PI3K, Page 13 See 2013, Page 10 Lon Garber’s Excellent Adventure: facing Gollum, P.T. & Paul Simon with R2D2

By Lon Garber new episode grew in bulge pressing against my rectum from Namibia LRG Local Group Leader duration and intensi- the abdomen. A follow-up MRI uncov- ty. My doctor, proba- ered a 9.5cm tumor of unknown origin. t all began in Africa with a pain bly suspecting colon My Namibian surgeon urged me to re- in my backside. My wife, Val, jok- cancer, recommend- turn to the States for diagnosis and treat- ingly called it payback time since ed a colonoscopy. ment. I’d been a pain in her backside for The scope vindicated Two weeks later (May 2009), a biopsy I37 years. The pain was just an annoy- my colon, but re- in Denver returned a GIST diagnosis. ance at first, but it persisted and each vealed a troublesome GARBER See GARBER, Page 11 Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — February 2013— PAGE 2 Patients’ face numerous obstacles The Life Raft Group in quest for life-saving treatment Who are we, what do we do?

The Life Raft Group (LRG) directs re- search to find a cure for a rare cancer By Phil Avila than Trent, a GIST specialist at Sylvester and help those affected through sup- LRG Writer Cancer Center in Miami. Dr. Trent says port and advocacy until we do. he frequently prescribes Votrient to his The LRG provides support, information and assistance to patients and families essica Marchionda GIST patients when the with a rare cancer called Gastrointestinal was desperate for a first- and second-line Stromal Tumor (GIST). The LRG achieves drug that would treatments of Gleevec and this by providing an online community for patients and caregivers, supporting local help her fight GIST Sutent no longer work. J And his patients frequent- in-person meetings, patient education in November 2011. through monthly newsletters and Her mother, Linda Mar- ly face denials from insur- webcasts, one-on-one patient consulta- tin, recalls how Jessica ance companies. tions, and most importantly, managing a This is partially due to major research project to find the cure for had tried and failed on a GIST. series of drugs since being the fact that the drug is diagnosed in 2007. being used off-label, not having been How to help “It was such a frustrating time. It was approved for treatment of GIST by the Food and Drug Administration (FDA). Donations to The Life Raft Group, a awful,” Linda says, recalling how doc- 501(c)(3) nonprofit organization, are tax tors had recommended she give Glaxo- Even though deductible in the United States. SmithKline’s drug Votrient a shot but clinical trials have You can donate by credit card at was unable to get Jessica’s insurance not shown the effi- www.liferaftgroup.org/make-a-donation/ or company to cover it despite appeals doc- cacy of Votrient in by sending a check to: umented by GIST specialists. GIST, Dr. Trent The Life Raft Group 155 US Highway 46, Suite 202 “We even sent a scan showing how says as many as 20 percent of the pa- Wayne, NJ 07470 Votrient had shrunk the tumor,” she tients he puts on says. Disclaimer The family’s decision had been to start the drug respond the drug, which costs around $60,000 a well. Dr. Trent We are patients and caregivers, not doc- tors. Information shared is not a substitute year, and somehow pay for it on their uses information from his practice to for discussion with your own through fundraising efforts, until doctor. coverage could be obtained. help patients ap- TRENT Please advise Erin Kristoff, Jessica died in February 2012 at age 36 peal denials by insurance companies. LRG Communications Director, at while an application for patient assis- [email protected] of any errors. tance with the drug was awaiting ap- And sometimes the insurance compa- proval by Glaxo-SmithKline. Insurance nies acquiesce. “If you can show the approval never came through. patient is benefitting, the insurance com- track is the slow Susan Farmer, another GIST patient, pany doesn’t have a leg to stand on,” Dr. line for people also was denied coverage by her insur- Trent says, saying as many as half of his fighting for their ance company for Votrient. When ap- patients who are denied Votrient cover- lives.” peals failed she opted to participate in a age eventually win appeals. “You have Burroughs says clinical trial of Votrient to gain access to to get past the first couple of levels and his organization has been pushing the talk to the medical FDA to follow a White House panel that the drug. Still another pa- director,” he says. in 2007 recommended a process for tient, who asked not Frank Burroughs, speeding approval of off-label drugs. to be identified, President of the Abi- “This would be a big help in getting in- says Votrient is gail Alliance for Better surance approval of these drugs,” he what's keeping her Access to Develop- says. alive. After switching insurance compa- mental Drugs, puts part of the blame on A spokesperson for the FDA said the nies she lost coverage only to win it the FDA for what he considers an out- panel’s recommendation is under consid- back after lengthy appeals. dated approval process. eration, but didn’t comment further. The obstacles Jessica and others faced “There’s a need for earlier access, for Burroughs suggested that the FDA take in getting insurance coverage for Votri- provisional or early approval of drugs,” into consideration evidence from spe- ent are common, according to Dr. Jona- he says. “What [the FDA] calls the fast See TREATMENT, Page 13 Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — February 2013— PAGE 3

LRG & colleagues make their mark on 17th annual CTOS meeting in Prague

By Roberto Pazmino testing survey. The poster from the fourth day. These patients were able to LRG Administrative Director study, entitled “The Investigation of experience low levels of side-effects and Routine Mutational Testing and Routine have not had further progression. While epresentatives from the Life Plasma Level Testing: A Survey of Pa- this study did deal with a small number Raft Group recently traveled tients, Caregivers, and Physicians” was of cases, it is interesting to note that to Prague, Czech Republic to displayed alongside numerous other these patients did respond to doses that attend the annual Connective GIST-related posters throughout much would be considered lower than normal. RTissue Oncology Society (CTOS) meet- of the conference. If this were to be validated through fur- ing. CTOS is an international group A number of these posters were of in- ther study, the potential exists to pre- comprised of physicians and scientists terest to the GIST community. Among scribe patients experiencing significant with a primary interest in the tumors of them was one from Dr. Mikael Eriksson side-effects even lower doses of imatinib connective tissues. The goal of the soci- from Skane University Hospital in Swe- before switching them to other drugs. ety is to advance the care of patients den, who presented a poster along with In addition to the standard poster ses- with connective tissue tumors and to one of his colleagues, entitled “Low sion, there was also a juried poster ses- increase knowledge of all aspects of the Dose Imatinib May Be Active in Many sion and a number of presentations. A biology of these tumors, including basic GIST Patients with Intolerance to Stand- number involved members of the LRG and clinical research (see www.ctos.org ard Dose.” The study looked at a retro- Research Team. One of these, from Dr. for more information). spective analysis of five cases of GIST Maria Debiec-Rychter and her col- The LRG was honored to present a patients, all Exon 11 and with metastatic leagues at the Catholic University in poster that summarized our recently disease, who have responded satisfacto- Leuven, Belgium, entitled “MIR-17, completed mutational and plasma level rily to very low doses of imatinib – from MIR-20A and MIR-222 Target KIT and 200 mg daily to as low as 100 mg every See CTOS, Page 15 LRG Research Team holds quarterly meeting

By Pete Knox example of this collaboration. The LRG Special Projects Coordinator paper will be examining the role of a molecule that is active in a particular he LRG Research Team re- metabolic pathway relevant to GIST cently held a teleconference – in order to study this, a number of that focused primarily on a things need to be accomplished – study the team will collaborate including providing relevant clinical The LRG Research Team in Leuven, Belgium. Ton and intends to publish. The LRG Re- data through patient histories, doing search Team consists of nine members, tests for the presence of the molecule, only after multiple layers of approval with seven at institutions throughout the performing analysis on tissue samples, from the various institutions involved. United States and two in Europe and writing a draft of the results. The The LRG providing information to the (Germany and Belgium) and meets four LRG, through its patient registry, was researchers directly via the patient regis- times a year, either via teleconference or able to provide patient histories to the try is also not something typically found in-person. The team works in a truly team, and the other tasks were divided in other research environments – in addi- collaborative fashion, sharing resources up among the various team members, all tion to sponsoring the research of its and ideas when working on their various of whom generously offered their time team members, the LRG is also actively projects, all of which focus on under- and equipment. participating in it by directly providing standing the nature of GIST and devel- This all occurred in real time through data. oping compounds that can be trans- conferencing software – this is part of The paper is currently in the process of formed into drugs used to treat the dis- what makes the LRG Research Team development and we all anxiously await ease. unique, as normally this process does the publication of its results, and will be The creation of this paper is a perfect not occur in such a timely and efficient sure to inform you as soon as that oc- fashion, but instead slowly and often curs. Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — February 2013— PAGE 4 LRG launches new & improved website, convenient mobile app & online store!

By Erin Kristoff, LRG effects management and our clinical Communications Director & Matt trials database. In Understanding GIST Mattioli, LRG Office Manager you will find information on the science of this disease, such as GIST types and fter two years of planning, survival statistics. Additionally, most toiling, and more planning, pages include a small summary of the the LRG unveiled its rede- subject with a link to read more. This signed website on January 1 was done so that anyone, regardless of A their GIST knowledge can understand at our same web address: www.liferaftgroup.org. Finding the bal- the subject, but can very easily go more ance between providing life-saving in- in-depth into each subject- in keeping formation and explaining GIST in a con- with the quality information we have been giving you all these years. cise, understandable way, has always are always welcome. Email us at life Newly Diagnosed been a struggle for the Life Raft Group. [email protected] with your input. This new site was created to offer both When first diagnosed with GIST, peo- forms of information with a couple more ple are often overwhelmed and desperate Online Store great new features thrown in. for information. We have added a fea- We are also really excited to share our ture to our homepage that allows these The Homepage new online store. Run through Cafe Besides the overall look of the LRG survivors to find what they need and find Press, we have set up a store where you website, the homepage is where you’ll it quickly. This section offers a detailed can get almost anything you can think of find the most change. We’ve organized slideshow, walking you through issues LRG-branded. Some of the items availa- the menu differently, in the hopes that that you might find important, like an ble for purchase are t-shirts, pajamas, you will be able to find things like where explanation of GIST types, survival rates sweatshirts, pillows, phone cases, mugs, to join and how to volunteer more easily. and the importance of support groups. and doggy clothes. But these are just a We’ve also added a slideshow to the top Throughout the year we will be making few of the hundreds of items to choose which will constantly be scrolling even more changes to improve this vital from and there is sure to be something through upcoming events, a link to join, area. for everyone. All of these items are and patient support-, advocacy- and re- GIST Survivor & Medical great, fun, and creative ways to show off search-related content. This way the Professional Areas your LRG pride and to raise GIST main services of the LRG are right there Also on the homepage, are two buttons awareness. You can find the store at at your fingertips. Below you’ll always asking whether you are a medical profes- http://www.cafepress.com/liferaftgroup find the most up-to-date news info as sional or GIST survivor. We wanted to well as links to our social media sites. include these areas so that anyone can Mobile App find the information they are looking for, Managing GIST/Understanding GIST The Life Raft Group is also proud to One of our biggest goals was to make even if they don’t know exactly where to announce our brand new mobile app. our GIST Education section more struc- look. On the Survivor page, you can find From this app you can read the latest tured and easy to follow. We understand links to Coping content and information newsletter articles, check out what’s how hard it is to be faced with a cancer on managing your care, all in the same happening on our Facebook and Twitter, diagnosis or recurrence and how quickly place. A medical professional at any and get all of our contact info. Two of you want the information you need. stage of their career can find useful in- the biggest features are the mobile Medi- We’ve separated the content on this site formation on our Medical Professional cal Update form and the ability to re- into two categories: Managing GIST & page some items found there are infor- quest your GISTory. This app will not Understanding GIST. By separating mation on our Research Team, Clinical only help everyone in the GIST commu- these areas, you can now more easily Trials Database and how to order a Phy- nity stay informed & connected but easi- pick and choose the content you want to sician Outreach package. ly and quickly update or request their read at the pace you decide. Managing There are many more new features to medical records from their own phone. It GIST is where you will find items relat- the new LRG site than we can list here. is available on the Apple app store, the ed to the management of your GIST We encourage everyone to explore and Android play market, and the Windows care, subjects like treatment types, side- let us know what you think. Comments, Phone app store, just search “Life Raft suggestions and words of encouragement Group”. Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — February 2013— PAGE 5 LRG welcomes Deputy Director and two new members to Board

Campbell brings Wall Street their lives in the World Trade Center Dan comes to the LRG with the shared attacks. Over $13 million has been dedication to find a cure. After learning smarts and warrior spirit to raised over the past six years for more that his wife Shawna was diagnosed deputy director position than 75 charities. Roger also has been with GIST in 2011, he became involved actively involved in supporting the in the GIST research currently underway

Wounded Warrior Project (WWP) since at Oregon Health & Sci- By Karen Kelley, LRG Writer & 2007 and currently serves on its Board ence University (OHSU) Gale Kenny, Program Associate of Directors and is Chairman of its In- in Portland, dedicating vestment Committee. both resources and com- he Life Raft Group is pleased In addition to his legal expertise, Rog- mitment. Dan has to welcome Roger C. er brings to the LRG a commit- worked in the telecom Campbell as our new ment to serving others and a industry for over 35 Deputy Director. Rog- wide-ranging skill set of pro- years. He and Shawna MILLER Ter, an attorney, brings to the LRG gram management, fundrais- have six children and over 25 years of experience in ing, and marketing experience. nine grandchildren, and currently live in the global financial services sec- As he stated in his welcome Brush Prairie, Wash. They delight in tor. He served in various capaci- message to staff, “The LRG family time outdoors, whether playing in ties, including General Counsel, provides me with a wonderful the winter snow or boating on the water. Senior Vice President, and Chief CAMPBELL opportunity to contribute in the They also love traveling. Administrative Officer in several non-profit sector in a mission Gary heads research at Lucidus Capi- Wall Street firms. When he joined BCG driven organization that works toward tal Partners, which focuses on the glob- Partners, a subsidiary of Cantor Fitzger- improving the lives of others.” al macro-economy, financial ald, he managed the merger, integration He is married to Kathy institutions, and real estate. and re-branding of a major financial McNamee, PhD., a Psychologist at He was previously Head of company and its subsidiary divisions the VA Vet Center in White Plains, Research at Caxton Associ- which more than doubled the size of New York. They are the parents of ates following 15 years of BCG’s business in New York. four children and live in Ridge- investment banking primari- While Roger has had a distinguished wood, New Jersey. ly with Goldman Sachs and career on Wall Street, he is most recog- JPMorgan. Gary joined the nized for his contributions to raising GLASSER Miller & Glasser use LRG Life Raft Group shortly after significant sums of money for charities. his father, Lawrence, was diagnosed. On behalf of BCG Partners in 2007, he devotion in new board roles Sadly, his dad lost his battle with GIST initiated, orchestrated and implemented Effective January 1, 2013, The Life on October 10, 2011. Gary's mother, the production of BCG Charity Day Raft Group Board of Directors an- Eileen, is a welcomed and hardworking which has grown to be a major event of nounced with pleasure the addition of volunteer who donates time weekly for remembrance and giving on September two new members, Gary Glasser and the LRG. Gary, a graduate of Yale, re- 11 of each year, honoring the 658 em- Dan Miller. We wish to extend a warm sides in Fort Lee, N.J. with his wife and ployees of Cantor Fitzgerald who lost welcome to these valued team members. four children. LRG sets GDOL Miami date for March

he second annual GIST Day of Learning (GDOL) will be held on March 23, 2013. This year we are privileged once again T that Dr. Jonathan Trent of Sylvester Cancer Center in Miami has agreed to host our gathering and we are honored to hear what he has to say. This is an important opportunity for continuing our ongoing education so we can all learn more and be empowered by the knowledge we receive. It is also an opportunity to meet others facing the same issues as well as connect with local experts in GIST treatment. Go to: liferaftgroup.org/events/ for more info. Dr. Trent (far right) with other GDOL 2012 presenters.

Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — February 2013— PAGE 6 LRG’s annual holiday fundraiser going strong!

By Gale Kenny patient support, education, and advocacy send out, it is not too late. Consider LRG Program Associate knows no boundaries, as we continually passing one on to a neighbour, co- strive to spread awareness and survivor- worker, friend or family member. This ur Holiday Fundraiser is still ship into all corners of the world. Of gesture will not only raise funds to help going strong, and has thus equal importance is the support those living with GIST, but will also far has brought in $45,000. these funds provide to our research spread much needed awareness about The Life Raft Group remains team who share in our passion- this rare disease. O ate commitment to find a There are so many relying upon the beholden to all who have donated both large and small, as it is the collective cure for GIST. thoughtful giving of others. We generosity of those who support us that If you have been mailed wish to express our warm grati- make The Life Raft Group the dynamic a fundraising packet and tude that we are the organization it is. As you are aware, our still have donor cards to recipient of yours.

Life Fest 2012 hits big in Las Vegas!

By Janeen Ryan tel. Thank you to Neill Miller of PM LRG Outreach Coordinator Digital Group for videotaping the event. Big thanks to our Presenting Sponsor, ife Fest 2012 was not only the Bayer Pharmaceuticals; Gold Sponsor, premier gathering of the Life Pfizer Pharmaceuticals; Silver Sponsor, Raft Group community but a Novartis Pharmaceuticals; Sapphire celebration of our ten year Sponsor, LRG Board President Jerry Lanniversary as well! And, what better Cudzil; Ruby Sponsors, Sanofi, Genen- venue to celebrate than Las Vegas! The tech and Michael & Maggie Mar- glitz and glamour of this beautiful, leg- cantonio; and Emerald Sponsor, Sylvest- endary city could only be eclipsed by er Cancer Center. We would also like to our immense joy of reconnecting with extend special thanks to Nadine Sta. old friends, giving and receiving hugs Brigida, Shun Otsubo and Matt Consoli and of course, meeting so many new for volunteering to photograph and take faces. People from around the country videos of the event. and across the globe came together for Thank you to all those who volun- three spectacular days of learning and teered to be mentors during the event as fellowship. well as all of the LRG members who generously gave of their time to help us coordinate this event. Special Thanks ______The LRG would like to thank everyone who helped put this event together: Read the whole article at: Melissa Tribus from Red Rock Resort http://tinyurl.com/Lifest2012 and Cheri Martinez from Suncoast Ho- Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — February 2013— PAGE 7 Swiss GIST Group awards 2012 GIST Prize

By Helga Meier-Schnorf Basel. GIST Gruppe-Schweiz The jury reached this unanimous decision under the chairmanship of The GIST Group of Switzerland award- Dr. Urs Metzger. ed its science prize for the third time on The study by Dr. Perez was de- November 22, 2012. It has been award- signed to establish whether the ratio ed ex aequo to Dr. Daniel Perez, Memo- of neutrophil granulocytes and lym- rial Sloan-Kettering Cancer Center phocytes in the blood circulation (MSKCC), New York, USA, and Dr. Mi- (NLR) is prognostic for GIST. It is a readily available method for chael Montemurro, CHUV Lausanne/ From left to right: Dr. Michael Montemurro, University Hospital Zurich. measuring the degree of immuno- logical activity in the blood. Helga Meier Schnorf, Dr. Daniel Perez, Dr. The presentation was given by Dr. Urs Urs Metzger. Metzger, Chairman of the Awards Com- To this end, a prospective analysis mittee of the GIST Group. was conducted on the blood values of 339 patients with primary, localized (38%), tiredness and diarrhea. A response to sorafenib was seen in 10 he association for the support GIST, who were treated at MSKCC from 1995 to 2010. Patients following percent of patients and a stabilization of of people affected by gastroin- the disease in 57 percent. The median testinal stromal tumors adjuvant imatinib therapy (n=64) were excluded from the study. The NLR was duration of treatment was 4.5 months, (GIST), the GIST Group of median progression-free survival (PFS) TSwitzerland has awarded its science correlated with the clinical course. 6.4 months and overall survival 13.5 prize for the third time. The 2012 GIST Progression-free survival was signifi- cantly longer in GIST patients with a months. The survival data for sorafenib Prize, worth 10,000 francs, is shared were identical for the third and fourth equally by Dr. Daniel Perez for his study low NLR. A correlation was likewise observed between NLR and the number line of therapy. An interesting observa- “Blood Neutrophil-to-Lymphocyte Ratio tion was the trend towards a longer PFS is Prognostic in Gastrointestinal Stromal of mitoses and between NLR and tumor size. Only a small number of neutrophil in patients in whom the sorafenib dose Tumor”, published in the Annals of Sur- was reduced or in whom a skin reaction gical Oncology and Dr. Michael Mon- granulocytes were detected in the tumor tissue. occurred. temurro for his study “Sorafenib as third Sorafenib appears very effective in the or fourth-line treatment of advanced In summary, it can be said that high- risk GIST patients have a high NLR. treatment of previously treated GIST gastrointestinal stromal tumor and pre- patients. treatment including both imatinib and The elevated NLR values are a parame- ter for systemic tumor-induced inflam- Besides the indisputable quality of the sunitinib, and nilotinib. A retrospective work submitted, the jury was also im- analysis”, published in the European mation. Dr. Montemurro and colleagues un- pressed by the engagement of Dr. Mon- Journal of Cancer. The award ceremony temurro in this rare disease. In the last took place on November 22 at the semi- dertook a retrospective analysis of the efficacy of sorafenib (Nexavar) in the five years he has given over 40 lectures annual meeting of the Swiss Group for on the subject of GIST and published Clinical Cancer Research (SAKK) in treatment of patients with a previously treated gastrointestinal stromal tumor nine studies listed in Medline on the based on data from 13 cancer centers. subject, either as author or co-author, Help GIST researchers All 124 patients in this largest series five of which are original studies. ! worldwide had previously been treated SAKK learn more abouT((NF1! with imatinib (Gleevec) and sunitinib The Swiss Group for Clinical Cancer If you have been diagnosed with GIST (Sutent), and 68 patients had also been and your tumor was classified as treated with nilotinib (Tasigna). The Research (SAKK) is a non-profit organi- Neurofibromatosis 1 (NF1), please play a prior treatments were ended in most cas- zation that has carried out clinical can- valuable part in GIST research by helping es because of tumor progression and cer studies as a cooperative group since researchers learn more about NF1. there was therefore an urgent need for 1965. The aim of SAKK is to develop treatment in these patients. existing cancer treatments further and to Contact Magdalena Sarnas at the LRG Sorafenib was started at twice daily investigate the efficacy and tolerability GIST Collaborative Tissue Bank by doses of 400 mg. This dose regimen led of new treatments. phone: 973-837-9092 X 114 or email: Further information: www.sakk.ch, [email protected] to mild side-effects in most cases. Fre- quent side-effects were skin reactions [email protected] Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — February 2013— PAGE 8 LRG closes out 2012 in the spotlight: advocacy, support & research highlighted

he Life Raft Group is dedicat- We are happy to announce that the pitalization of a loved one and stated ed to the welfare and survival article has received over 2000 views that her plan would include calling upon of GIST patients worldwide including 317 within December. If you Norman Scherzer whose survival advice and we carry out our mission would like to view the article, go to would be “Get angry. Get smart. Get Tthrough research, patient support & edu- www.biomedcentral.com/1471- organized.” cation and advocacy. The LRG was 2407/12/90 If you want to read the article, go to e- highlighted in all three areas at the end patients.net and search “As She Lay Dy- of 2012 and we wanted to share the im- E-patients.net post ing” portant work we’re doing with all of channels Norman you. Talking new clinical Susannah Fox, Associate Director of LRG Research Article the Pew Internet & American Life Pro- trials models at Sanofi ject, wrote an interesting post on e- Norman participated in a workshop at Grows in Popularity patients.net called As She Lay Dying - a Sanofi’s site in Bridgewater, New Jer- In 2012, the LRG published an article son calls on the health system to involve sey, along with a number of other patient in the Journal BMC Cancer called patients and families in improving safe- advocacy groups. “Survival of gastrointestinal stromal ty. They brainstormed ways of putting the tumor patients in the imatinib era: life She highlighted what her strategy patient first and how patient advocacy raft group observational registry.” The would be when faced with a sudden hos- groups are important to the clinical trial authors were LRG Science Director Jer- process. They discussed how the phar- ry Call, Christopher D. Walentas, Jens maceutical industry can better deliver C. Eickhoff, and LRG Executive Direc- patient value as well as defining oppor- tor Norman Scherzer. tunities to collaborate effectively. Dave Safford shares his list of happiest moments since GIST and those still to come

ecently, LRG Board Member Kiana, and Alani excel in everything Dave Safford decided to sit they do; tell Cherry and the kids I love down and think about all of them every day. the good fortune that has “Cool Fun things I have planned or Rcome into his life since being diagnosed hope to do: Superbowl party weekend with cancer. We liked his list so much in Vegas with friends and helicopter we decided to put it in the newsletter so tour of Grand Canyon (coming up that everyone in the GIST community soon and would love if Seahawks are can see a story of positivity and hope. in it!); play golf at midnight in Alaska “Cool, Fun things I've done since I've again; have a "“Darjeeling Limited- had cancer: ish” roadtrip with my brothers Ron Topping the list, two-week family vaca- and Scott; Play in the World Series of tion in Maui; Went to game seven of the Poker ( not the main event - too rich Dave and his wife, Cherry. NLCS to send Giants to World Series; for me); gaze at and ponder the Milky played in the Peter Jacobsen Pro-Am Way on a clear night (who knows the Fame; spend as much time as possible golf tournament; helped “invent and best place in the world to do this?); be a with my family. launch” several new products at work; contestant on Jeopardy, Wheel of For- Do you have any fun ideas you’d like saw multiple Broadway shows in N.Y., tune or Who Wants to be a Millionaire; to share with Dave or fellow members? Cirque de Soleil in Vegas; Sunriver Zeb- visit Cooperstown Baseball Hall of Email us at [email protected] ra club weekends; watched Natalie, and let us know! Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — February 2013— PAGE 9

Pediatric GISTer ties the knot! LRG researchers talk microGISTs: how common?

Did you check out a recent article we sent by LRG Research Team members, Dr. Sebastian Bauer & Jon- athan Fletcher. Here’s an excerpt to he great article coming: FLETCHER “Most of us have moles somewhere on our bodies. Yet, although many moles are akin to benign neo- plasms, and are exceptionally common in the general population, we all know they very rarely transform into malignant skin cancers. What few peo- ple know is that one could describe GISTs as the “moles” of the stomach. BAUER Jason DeLorenzo, LRG member and pediatric GISTer, got married on Every third person has a tiny November 17, 2012 to Jill Medhus. The ceremony was held at Corpus Christi GIST in the stomach, so the incidence of in Hasbrouck Heights, N.J., the reception followed at Tides in North Haledon, GIST in the readers of this LRG newslet- N.J. ter is probably not substantially different from the incidence of GIST in the read- The LRG is excited to hear of another GISTer wedding and we give our ers of ‘Sports Illustrated’.” deepest congratulations to the happy couple! If you missed it, you can find it on our website at liferaftgroup.org. Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — February 2013— PAGE 10

tional test. Further, without mutational 2013 testing, these patients are unaware of From Page 1 mutation-specific trials like AROG’s crenolanib trial.  Helping to restore $1 million in can- this in the world of medicine. Nearly every type of GIST has poten- cer research funding to New Jer- We’ve seen this in breast cancer. How tial for optimizing treatments based on sey’s budget long after the introduction of the lum- mutation type.  Our Research Team making several pectomy did it take for this option to be Mutational testing has been readily breakthroughs in the search for a considered instead of radical mastecto- available for several years. The process cure my? Too long. of obtaining tissue for mutational testing  Debuting features like a new and We find the same issue in lung cancer. is a relatively simple one. Several labor- improved website and mobile appli- How long did the public wait for the atories perform the test on routinely col- cation admission that smoking damages your lected tissue following surgery. In fact Now I would like to focus on our fu- health? How many decades went by be- this testing is free of charge through the ture, so I am going to direct your atten- fore the statement “Smoking causes lung LRG Tissue Bank or directly shipped to tion on personalized medicine—a myth cancer” was legally required on every Oregon Health & Science University. or reality—and begin a dialogue about pack of cigarettes? Again, too long. Let me recap: We know that several its impact upon GIST patient survival. While the world waited for these dis- GIST subtypes require non-standard ______coveries to become common knowledge, treatment. We know that this testing is patients suffered, some even died. fairly easy to have done and is often free Personalized Medicine The past decade has seen enormous of charge. breakthroughs in the molecular charac- But we also know that, according to Question: Myth or Reality? terization of different cancers with GIST a recent report, the total number of Answer: Unfortunately, myth. leading the way. GIST patients receiving such muta- Here is what we know: GIST patients tional tests is six percent1. Let me explain: can be categorized by a range of muta- The LRG thinks this is unacceptable We have the mutational testing tools to tions like exon 11 or 9. These subtypes and we hope you do too. categorize many different subtypes of can have different response rates to dif- We will bring you more information GIST. ferent dosage levels or treatments. about what is going on, how it impacts We have a growing body of evidence For example, approximately 10 percent the survival of GIST patients and what to optimize treatments based upon of GIST patients have a KIT exon 9 mu- we can do about it. In the meantime, you knowing such subtypes. tation. This type of GIST has been found can take a stand with us and join the Despite this, few GIST patients in the to respond better to higher doses of LRG Patient Registry & Tissue Bank to United States receive mutational testing. Gleevec. get mutational testing free of charge. The LRG is determined to make per- Even more telling, approximately five 1. Pisters PWT, Blanke CD, Von sonalized treatment for GIST a reality. percent of GIST patients have a D842V Mehren M, Picus J, Sirulnik A, Stealey Throughout time there have been enor- PDGFRA mutation which is totally re- E, Trent JC: A USA registry of gastroin- mous time gaps between the discovery sistant to Gleevec. Following surgery, testinal stromal tumor patients: changes of new ideas and the systematic imple- these patients are routinely prescribed in practice over time and differences mentation of these ideas. We have seen three years adjuvant Gleevec (standard between community and academic prac- some fairly extraordinary examples of care) because they have not had a muta- tices. Ann Oncol 2011.

Life Raft Group adds new local group leaders & international partners

Newest local group leaders: Ananth Pai– Minnesota Debbie Farmer - Indiana [email protected] Janeen Ryan, LRG Outreach Coordinator [email protected] is currently filling positions in Arkansas, Jill Heinrich - Alaska New international group Hawaii, Kansas, Kentucky, Mississippi, [email protected] leaders: Montana, New Mexico, North and South Joan Smith - Nevada Roy Neil - Australia Dakota, Vermont, W. Virginia, Wyoming [email protected] [email protected] and Washington DC. If you are interesting Paula Stover - South Carolina Piotr Fonrobert– Poland in being a local group leader, please con- Peru [email protected] [email protected] tact herat 973-837-9092 ext 113 or Eva Maria Ruiz de Maryanne Snowbrick - Ohio Marie Lagaluga- New Zealand [email protected]. Castilla Yabar [email protected] [email protected] [email protected] Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — February 2013— PAGE 11 both a colo-rectal surgeon and the only my GIST was attached to my rectum for GARBER surgeon in their surgical group who per- its entire length of 6.5 cm. With that From Page 1 formed robotic surgery. The colo-rectal amount of surgical disruption, Dr. Kortz surgeon assured me that it was impossi- felt it necessary give me a temporary Up until then, the word ‘gist’ meant ‘the ble to avoid a permanent colostomy and diverting ileostomy in order to let the essential part of a matter;’ but now the openly mocked the idea of robotic sur- rectum heal for three months before fur- essential part of my GIST became a tiny gery and he surprisingly mocked his ther use. He had warned me before sur- orange bullet - gery that a lengthy tumor-to-rectum Gleevec. I had “Up until then, the word ‘gist’ meant ‘the essential part of connection would necessitate this braced myself for a matter;’ but now the essential part of my GIST became a procedure. In keeping with the fami- the horrors of tiny orange bullet - Gleevec.” ly naming tradition, I named my bag chemotherapy P.T. (an abbreviated form of “Poop and the oncologist replaced my imag- own colleague who had embraced this Tank”). ined nightmare with a once-a-day pill new technology. I left his office totally True to his assurance, three months that amazingly came with almost no discouraged, but convinced I would nev- later in June 2010, Dr. Kortz was ready side-effects. Instead of chemotherapy, I er let such a man touch my body with a to reverse the ‘ostomy. Before that pro- prescribed baseball therapy for myself. I scalpel. cedure, he performed a proctoscopy to took my pill in the morning and went to make sure the surgical site was com- Colorado Rockies baseball games in the R2D2 to the Rescue pletely healed. I thought the hospital afternoons and evenings while waiting My next and last surgical consultation receptionist would split a gut laughing for Gleevec to do its job. My nightmare was with Dr. Warren Kortz whose office the morning I walked up to her desk and became a dream come true. Since my suite adjoined Porter Adventist Hospital said, “Hi, I’m here to play a starring role son-in-law works for the Rockies, my in Denver. He was as optimistic as the in your ‘bend-over’ movies.” That was therapy was free. prior surgeons had been discouraging. the last fun I had that morning. When I So on February 26, 2010, they carted me Oh No, Anything But That asked Dr. Kortz if I would be under an- into the sterile operating room where the esthesia for the procedure, he laughed The only problem on my medical hori- scary looking robot waited and said, “No; instead zon was the specter of a permanent co- patiently in the corner. For you’ll get to watch the lostomy. My family physician assured the next 11 hours, without whole thing on live me a colostomy would be the certain ever touching my body TV.” So for an hour aftermath of any surgery to remove Gol- with his own hands, Dr. he took me on a guid- lum (more on that later). The required Kortz painstakingly and ed tour of a part of my surgical margins would severely com- meticulously excised Gol- body I’d never seen promise the surrounding sphincter mus- lum while sitting at a before and hope to cles, thereby requiring that my computer console on never see again; down The DaVinci robot...or R2D2? closest friend be an unmention- the opposite side of the where the sun don’t able bag. room. The DaVinci shine, but the camera My daughter decided my tu- robot has three surgical arms and one lights do. Immediately following my mor needed a name and what camera arm that move around your movie debut, I had the privilege of en- better name than Gollum, the GOLLUM body with a minimum of disruption. during a barium enema. Oh boy! I gray and slimy, mysterious The camera shows the surgeon a passed both tests with flying colors and creature who kept popping up unwanted view of the surgical site that he could believe me, with the enema there were a in the Lord of the Rings trilogy. Gleevec never see with his own eyes alone. lot of flying colors. Next stop – ileosto- quickly shrunk Gollum to 6.5 cm, but no Just a few hours after waking up, I my reversal. more. My surgeon assured me that the spent most of the day out of bed, sitting So I celebrated my 60th birthday on only way to avoid a colostomy would be in the guest chair in my room. I was the operating table, while Dr. Kortz for Gleevec to reduce Gollum to three walking the ward the next day. And only ripped me a new you know what. Later centimeters or less. It wasn’t happening, three days after surgery, I walked out of his surgical assistant told me that while but my surgeon wanted to cut anyway. the hospital under my own power. I love he was stuffing my intestines back into In the meantime, I’d heard about doc- robots and I’m absolutely in awe of Dr. my body, he sang, “Back in the bus, tors using DaVinci robotics to perform Kortz’s care, compassion and capability Guts” to the tune of Paul Simon’s “50 rectal surgery in a way that preserved as a surgeon. Ways to Leave your Lover.” A few days sphincter abilities, thus avoiding the per- My Worst Friend – P.T. later he came into my room and said, manent installation of the nasty bag. I “I’m releasing you, but before you go, I mentioned it to my surgeon and he That’s the good news. Moderating that want you to roll on your side and wink frowned. He grudgingly referred me to good news only slightly is the fact that See GARBER, Page 15 Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — October 2011 — PAGE 12 Cesarini’s ten year GIST battle ends with family by her side

aura Cesarini passed officer with the away on the fifth of Janu- Town of Braintree ary after a 10 year battle and was later pro- with cancer. She died moted to detective. Mpeacefully surrounded by her husband On December 26, and siblings. Maura was the daughter of 1998, Maura mar- Irish immigrants, Humphrey Moynihan ried Michael. They from Gneeveguila, County Kerry, Ire- had two daughters land and Maura Derwin from Athlone, Michaella and Bel- County Westmeath, Ireland. She leaves la. Shortly after behind her husband Michael, daughters Bella’s birth, Michaella and Bella, sisters Mary Con- Maura was diag- neely (Galway, Ireland) and Sharon nosed with a rare Moynihan (Duxbury), brother Frank and incurable can- Moynihan (Randolph), nephew Rory cer, called GIST. Conneely (Galway, Ireland), niece Jade This diagnosis Maura, with husband Michael, and daughter Michaella and Bella. Moynihan (Randolph) brother-in-law made Maura and Kevin Conneely (Galway, Ireland) sis- her family deter- ter-in-law Judy Woods (Randolph), mined to appreciate every moment of these groups, Maura made friends all mother-in-law Nancy Cesarini life and to live their lives to the fullest. over the world. Maura was dedicated to (Brockton) and father-in-law Joseph Maura loved spending time with her trying to find a cure for this disease and Cesarini (Brockton). family. She was very close to her sisters did extensive fundraising for research Although only 43 at the time of her Sharon Moynihan and Mary Conneely with her husband Michael. death, Maura accomplished a great deal and her brother Frank Moynihan. It was Maura’s determination to beat this dis- in her life. Maura was a graduate of Em- very important to her that her family ease was recognized by the Sarcoma erson College where she received both a remain close. Despite her cancer diagno- Foundation of America whom presented bachelor’s and Master’s degree in Com- sis, Maura and her family travelled to her with a Courage Award at a ceremo- munications. Maura worked for a time in many places including, Ireland, France, ny in New York City in April 2012. the telecommunications field both here Canada, the Caribbean and numerous In lieu of flowers, donations can be in Massachusetts and in London, Eng- National Parks. Maura was very com- made to the Cesarini G.I.S.T. Research land where she lived for two years. In passionate and empathetic. She belonged Fund at The Dana Farber Cancer Insti- 1995, Maura decided to change careers to several on-line support groups for tute, PO Box 849168, Boston, MA and went to the police academy. It was people battling GIST offering guidance 02284-9168 and/or Cesarini Education here that she met her future husband and encouraging words to other patients. Fund at Michael J. Cesarini, FBO The Brockton Police Detective Michael Ce- She also set up a support group in Cesarini Children, Acct XX0739 c/o sarini. Maura graduated from the police Duxbury for other women battling can- Eastern Bank, PO Box 1716, Duxbury, academy in 1996. She became a police cer. Through her active participation in MA 02331. LRG Canada holds first GIST Day of Learning in Toronto

RG Canada held it’s first GIST Day of Learning in November and L Dr. David Josephy had nothing but great things to say the event, “Our Day of Learning was a 100% success— rave reviews all around!

“The two doctors from Sunnybrook Hospi- tal, Dr. Ko (medical oncologist and Dr. Law (surgeon) were just outstanding - very knowledgeable, approachable, and generous with theirSee time.” ASCO, Page 13

From left to right: Presenters Ko, Josephy, Koch & Law Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — October 2012— PAGE 13 (cell death) was not observed. However, and loss of PTEN both on the gene and PI3K when GDC-0941 was combined with protein levels. From Page 1 imatinib, profound tumor regression (as In conclusion, this study showed that opposed to stability) was observed, as the combination of GDC-0941 and additional measurement taken four was a nearly complete stopping of tumor imatinib is more effective than either weeks after treatment was discontinued cell proliferation and an increase in alone in terms of antitumor efficacy, and to see if there was any further tumor apoptosis, making the combined treat- had a sustained effect even after with- growth. Tumor response was assessed ment superior. This was also confirmed drawal of treatment. In addition, assess- via volume measurements, micro-PET when looking for tumor regrowth. In ment of PTEN status may represent a imaging, histopathology and immunob- three out of the six models, the tumor useful biomarker for patient selection. lotting. Evaluations of genomic altera- size remained reduced and stable even Combining drugs to enhance efficacy is tions in the PTEN/PI3K/AKT pathway after the treatment was discontinued for an interesting approach to research, and were also performed. the combined treatment group. This was perhaps we will see more studies like The results of the study showed that in not observed in the imatinib-only group. this going forward. all models GDC-0941 alone did cause In addition, a positive correlation was Read more about the LRG Research stabilization of tumor growth and inhibit demonstrated between response to the Team on our website: liferaftgroup.org/ tumor cell proliferation, but apoptosis combined GDC-0941/imatinib treatment the-lrg-research-team

TREATMENT From Page 2 cialists like Dr. Trent according to some indus- the ACA, such as coverage for pre- rather than relying on try observers. existing conditions, which will help can- just clinical trials for Susan Pisano, a spokes- cer patients. drug approvals. “A big person for America’s The issue of access to off-label drugs is factor is modernizing Health Insurance Plans, only going to become more important as clinical trials,” he says, an industry association, targeted treatments become more availa- particularly doing away says insurance companies ble and personalized medicine grows. with studies where can- take three factors into “As we get more data, we’ll figure out cer patients who are fighting to survive consideration in coverage of off-label which patients respond better to which are given placebos. drugs: FDA approval, support from drug. We’re not quite there yet,” Dr. “I’d much rather have my patients on compendiums such as the Trent says. Votrient than taking a placebo in a clini- National Comprehensive As for Linda Martin, she cal trial,” Dr. Trent says. Cancer Network is continuing the fund- In the meantime, insurance companies (NCCN), and published raising she started to help have been cutting back on their coverage studies on the efficacy of pay for Jessica’s Votrient. of off-label drugs for cancer treatment, the drugs. Pisano says she Jessica loved learning, doesn’t know of any trends limiting cov- and completed her master’s degree in Mark your calendars! erage of off-label cancer drugs. “I’m not May 2011. In her honor, Linda has sure if some kind of early approval of raised money to start a college fund for  The US & Canadian Academy of drugs by the FDA would make a differ- people with life-changing diagnoses. Pathology conference ence,” she says, since the other factors (USCAP) will be held are also taken into consideration. “It’s just one of the things they look at.” The LRG will be bringing you March 2-8 in Baltimore. Pisano says she’s not aware of any more and more of these  The next GIST Day of provisions in the Affordable Care Act Learning will be held in (ACA) that might affect how off-label investigative articles in the Miami on March 23 at the Sylvester drugs are covered in the future, but some future. If you know of an issue Cancer Center. More details coming soon. fear that insurance companies, facing a that should be addressed by squeeze, will cut back more.  The LRG researchers will be meeting “The jury is still out on that,” the Abi- the LRG, please email us at March 24-26 at the LRG office in N.J. gail Alliance’s Burroughs says, noting [email protected] that there are significant benefits from Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — February 2013— PAGE 14 Beloved husband, father, grandfather and trusted LRG Board Member, Bob Book passes away at 82

ob Book, 82, of serving until 1992. Bob Bart J. Book (Sherri). Grandchildren Carmel, Ind. and Dr. Bud Harmon es- include: Benjamin R. Klunder, Lauren passed away the tablished the Purdue Uni- E. Book Linos (Constantine), Hilary R. morning of De- versity Book Harmon Book, Christine Hatfield Waldrip Bcember 9, 2012. He was Leadership Program in (Brad), Michael M. Hatfield, and great- born on December 31, 1929 Animal Sciences in 2005. granddaughter, Isabella Waldrip. Others in Indianapolis, IN to Wm. Board memberships in- include cousins Martha Powell and fam- H. and Margaret V. Book. cluded: The Marine Corps ily, and Kathryn Ann Hammons, and A 1948 graduate of Arsenal League, the Indiana Mili- daughter Kathy Hammons, as well as Technical High School, tary Coalition, Military many good friends and associates. Bob enlisted in the United Officers of America The family wishes to thank the St. Vin- States Marine Corps Re- Assn., the Farm Founda- cent New Hope Foundation staff for ex- serve that year, and spent SEMPER FI tion, Neogen Corporation, cellence in care and kindness toward 31 years in both US Marine Ag Alumni Seed, U.S. Colonel Book and his family and many Corps and US Marine Corps Reserve, Feed Grains Council, National FFA and friends. retiring in 1983 with the rank of Colo- The Life Raft Group. For 29 years he Memorial contributions may be sent nel. was both an elected representative on to: After earning a BA in animal science the Clay Township Board in Hamilton Purdue Founda- from Purdue University in 1952, Bob County, Indiana, and President of Clay tion; Book- earned an MBA from Indiana University Township Regional Waste District. He Harmon Leader- and completed the Senior Executive Pro- was Precinct Committeeman for Clay ship Forum; St. gram at Columbia University. He be- Center 2, for over 30 years and served Vincent Health came a driving force behind Elanco St. Vincent’s Carmel Hospital from Foundation, the Products Company, a division of Eli ground breaking until he was named Life Raft Group, Lilly Company, when he joined as a Chairman of the Board, that board mem- Arsenal Tech- sales representative in 1955. After retir- bership continuing until June 2012. nical High ing in 1983 as Group Vice President of Bob is survived by his wife of 47 School Alumni Agricultural Marketing, Bob became the years, Jeanne Book; their children, Mary Association, or first president of The Indiana Institute of M. Klunder (Robert), Nancy Joanne Marine Corps Agriculture, Food and Nutrition, Inc., Fletcher, William H. Book (Dede), and University at Quantico, VA. Chilean GISTers & CML patients meet!

On Saturday December 15, 2012, a group of CML and GIST patients got together to summarize activities done during 2012, and celebrate the end of the year. Thanks to Piga Fernandez, LRG Global Relations Coordinator and head of Corporacion Nacional Maxi Vida Chile for sending a picture. Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — February 2013— PAGE 15

ble/metastatic/recurrent GIST patients, trointestinal Stromal Tumor (GIST)” CTOS and looked at a number of different was led by Life Raft Group Research From Page 3 prognostic factors. The results deter- Team member Dr. Anette Duensing. The mined that long-term survivors (five study re-evaluated some chemotherapy ETV1 in Gastrointestinal Stromal Tu- years or greater) were characterized by drugs for their sensitivity to GIST. A mors (GIST)”, looked at microRNA having smaller tumors at start of total of 89 FDA-approved compounds (miRNA), a small RNA molecule that imatinib treatment, higher neutrophil or were studied and tested against five helps regulate gene expression, and albumin levels, better World Health Or- GIST (imatinib-sensitive and imatinib- “aimed to discover any GIST specific ganization (WHO) performance status, resistant) and two leiomyosarcoma cell miRNA expression patterns and identify and surgery that removed all residual lines. GISTs were found to be highly miRNAs involved in GIST tumorigene- disease. Perhaps the most striking aspect sensitive to several drug classes, most sis.” The study involved 50 primary was that the median progression-free notably inhibitors of transcription and GIST tumors and 10 leimyosarcomas of survival (PFS) of these patients on first- topoisomerase II – in some cases more the GI tract. The results identified a line imatinib was 35.6 months, a figure so than imatinib or sunitinib. In the number of miRNAs that may be impli- that is greater than the 24 months that is words of the study authors, “these drug cated in GIST. A decreased level of generally reported. classes should be considered as viable three of these, MIR-17, 20A, and 222, Another presentation, led by the afore- treatment options for GIST patients, and may contribute to the overexpression of mentioned Dr. Michael Eriksson but also the initiation of clinical trials is warrant- KIT as well as ETV1, genes that play a involving Drs. Peter Reichardt, Heikki ed.” key role in GIST. When expression of Joensuu, Peter Hohenberger, and LRG Finally, Dr. Peter Reichardt and his these miRNAs was increased, KIT and Research Team member Dr. Sebastian colleagues gave updated results on the ETV1 protein levels were decreased, Bauer, was entitled “Needle Biopsy GRID trial for regorafenib, which in- thus inhibiting cell proliferation and Through the Abdominal Wall for the cluded 199 patients. Their results causing cell death (apoptosis). This Diagnosis of GIST – Does it Pose any showed that the study met its primary points to the possibility of these specific Risk for Tumor Cell Seeding” and endpoint, significantly improving medi- microRNAs as targets for future thera- looked to see if performing this particu- an PFS both per central review and in- peutic treatments. lar type of biopsy was associated with vestigator assessment. The median PFS Another study, led by Dr. Piotr negative outcomes for patients in terms for central review was 4.8 months (vs. Rutkowski and also involving Dr. Maria of PFS or overall survival (OS). The 0.9 months for placebo) and for investi- Debiec-Rychter, was a long-term analy- results suggest that this is not the case, gator assessment 7.4 months (vs. 1.7 sis of GIST survivors. The study, enti- but the authors suggest they should be months for placebo). tled “How Did the Results of Advanced viewed with caution due to the retro- All in all, the CTOS meeting delivered GIST Treatment Improve Over 10 spective nature of the study and the rela- a number of studies that showed particu- Years: Analysis of Inoperable/ Metastat- tively small number of cases studied lar promise for GIST, many involving ic/Recurrent GIST Treated With (393). esteemed members of the LRG Research Imatinib in 2001-2009. Who are Long An additional study, entitled “A Medi- Team, and the Life Raft Group was hon- Term Survivors?” was a retrospective um Throughput Compound Screen Iden- ored to both participate and present their analysis of 370 inopera- tifies Novel Treatment Options for Gas- poster at the event.

excellent surgeon and a really “I remain cancer-free and I’m living each day to funny guy. His father was a GARBER its fullest with joy in my heart for another day From Page 11 surgeon; his brother is also a surgeon. When the rest of the to make a difference in someone else’s life.” at me.” So I obediently rolled away from neighborhood kids were play- him and “winked” in a way I'd never ing doctor, he and his brother were oper- with joy in my heart for another day to winked at anyone before, all through the ating on the cat. make a difference in someone else’s life. breezy side of my hospital gown. He Back to Work Our team just completed building a com- was suffi- munity training center in a very poor ciently sat- Two months later I was back in Na- squatter’s camp (see photo). isfied that I mibia continuing my missionary du- Life is beautiful, even in a life raft. could ties. It took a full year for my plumb- Carpe Diem. squeeze the ing to return to pre-operative effec- right mus- tiveness, but I’m now completely For an introduction to the DaVinci cles. Dr. back to normal. I remain cancer-free robotic surgical technique: Kortz is an Garber’s team recently built this and I’m living each day to its fullest www.davincisurgery.com community training center. Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — February 2013— PAGE 16 T HE L IFE R AFT G ROU P

Staff Volunteers Board of Directors Executive Director Norman Scherzer Database Consultant Steven Rigg Executive Committee Deputy Director Roger Campbell Official Greeter Gail Mansfield Jerry Cudzil, President Executive Assistant Diana Nieves Latin America Liaison Vicky Ossio Clinical Trials Coordinator Jim Hughes Stan Bunn, Past President Science Director Jerry Call Photographer Kim Tallau Ray Montague, Secretary-Treasurer Asst. Development Director Lisa Hart Member Birthday Coordinator Mary Kluth Special Projects Coordinator Peter Knox Special Projects Asst. Eileen Glasser Robert Book* Communications Director Erin Kristoff Science Team Tanya DeSanto Mia Byrne Office Manager Matthew Mattioli Jim Hughes David Josephy Chris Carley Administrative Director Roberto Pazmino Michael Josephy Gary Glasser Program Director Sara Rothschild Rick Ware Jim Hughes Patient Registry Supervisor Magda Sarnas Glenn Wishon Jerry Knapp Development Director Christine Schaumburg Paula Vettel Dan Miller Global Relations Coordinator Piga Fernandez John Poss Bookkeeper Debbie Haroldsen Marietta Robinson Program Associate Gale Kenny David Safford Program Associate Loren Morales Rodrigo Salas Websites Manager Jim Napier Larry Selkovits Outreach Coordinator Janeen Ryan Silvia Steinhilber Program Associate William Sumas *in loving memory

155 US Highway 46, Ste 202 Phone: 973-837-9092 Contact the LRG Wayne, NJ 07470 Fax: 973-837-9095 www.liferaftgroup.org E-mail: [email protected]

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

Alabama Pat George Maine Jodi Merry Oregon Gail Mansfield Alaska Jill Heinrich Maryland Bonnie Emerson Pennsylvania Jeffrey Bernstein Arizona Janeen Ryan Michigan Ellen Rosenthal Rhode Island Susan Farmer Colorado Ann Bridgewater Minnesota Ananth Pai South Carolina Paula Stover California Dina Wiley Missouri Katie Bloss Tennessee Alice Sulkowski Martha Zielinski Montana Dirk Niebaum Texas Dee Hawkins Delaware Cindy Bones Nebraska Sally Norton Utah Mike Ginsberg Florida Skip Ryan Nevada Joan Smith Virginia Sally Jackson Georgia Pat Lemeshka New Hampshire Julie Thorne Washington Deanne Snodgrass Idaho Janet Conley New Jersey Anita Getler Wisconsin Rick Ware Illinois Jim Hughes New York Pat Bonda Swenson Indiana Debbie Farmer North Carolina Chuck Korte Iowa Barbara Kepple Ohio Maryann Snowbrick Louisiana Jackie Welsh Oklahoma Jane Rowan

Life Raft country liaisons: Learn more about the Global GIST Network & find contact info for your rep at www.globalgist.org

Argentina Melisa Biman Iran Negar Amirfarhad Samoa John Galuvao Australia Roy Neill Ireland Carol Jones Saudi Arabia Mohamed-Elbagir Ahmed Belgium Kris Heyman Israel Avi Zigdon Scotland Helena Koumbouzis Bolivia Virginia Ossio Italy Anna Costato Singapore Robert Richardson Bulgaria Stefan Mandov Japan Sumito Nishidate South Africa Annette Mentasti Brazil Luciana Holtz Jordan Mohammed Milhem South Korea Hyun Jung Yang Canada David Josephy Kenya Francis Kariuki Spain Luis Herrero Chile Piga Fernández Korea HyunJung Yang Sudan Mohamed-Elbagir Ahmed China Ruijia Mu Macedonia Dejan Krstevski Sweden Susanna Allgurin Neikter Colombia Maria Helena Matamaa Malaysia Yong Choo Sian Switzerland Helga Schnorf Costa Rica Michael Josephy Mexico Rodrigo Salas Thailand Kittikhun Pornpakakul Cyprus George Constantinou Namibia Lon Garber Turkey Haver Tanbay Czech Republic Jan Pelouchová New Zealand Marie Lagaluga U.K. Judith Robinson Dominican Rep. Alejandro Miranda Netherlands Contactgroep GIST Uruguay Fabrizio Martilotta Finland Mirja Voutilainene Nicaragua Maria Teresa Ponce Venezuela María Isabel Gómez France Estelle LeCointe Norway Odd Andreas Tofteng Germany Markus Wartenberg Pakistan Muhammad Shahid afique Greece George Constantinou Peru Maurice Mayrides Honduras Xiomara Barrientos Poland Piotr Fonrobert Guatemala Silvia Castillo de Armas Puerto Rico Gerardo Silva Hungary Tünde Kazda Romania Simona Ene India Paresh Majmudar Russia Tanya Soldak