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 Even though tors had recommended she give Glaxo- 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.