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.