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Life Raft Group Nov. 2007 Newsletter

Life Raft Group Nov. 2007 Newsletter



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Published by: The Life Raft Group on Jan 22, 2008
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November 2007 Vol. 8, No. 11
Battling gastrointestinal stromal tumor
In memory of Bob Caraway, Russ Jones,Michele Scheiperpeter & Paty Roig
Viva Carolina’ team exceeds$10,000 goal for Life Rafter
Compassionateuse process is notso compassionate
Thanksgiving for LRG research
By Norman Scherzer
LRG, Executive Director
woman sits at her Thanks-giving Day table with tearsof joy in her eyes. This year she was thankful for a pieceof paper and three letters. The paper was the results of her last CT scan and the three little letters were NED. After  fighting so hard for so long, she wasclassified as “no evidence of disease”. But these tears were bittersweet. Shecould not forget all of her friends whomight not be so thankful, those friendswho still had a long way to go and per-haps might never make it there. She alsoremembered that her three little letterswere not a guarantee of more Thanks-givings, at least not yet.
More research is needed if we want tobring patients like this their peace. It isnot enough to be stable or NED for anundetermined length of time.
One pa-tient
is not enough, every life is impor-tant. To do that, we need your help.Prior to 2000 there was no viable treat-ment for GIST. In that year, clinical tri-als discovered that GIST patients re-sponded to a new targeted oral drugcalled Gleevec (imatinib). Gleevec pro-duces an excellent initial response ineighty-five percent of GIST patientswith metastatic or unresectable disease.While this response is relatively long-lasting, half of all patients fail Gleevecwithin two years and almost all patientscan be expected to eventually failGleevec. Half of the patients respondingto Sutent, a second-line therapy, willprogress within six months.
What this means is that unless wecan discover new therapies to moreeffectively prevent treatment resis-
See RESEARCH, Page 6
By Judi Lifton, GIST caregiverInterviewed by Erin Kristoff 
udi Lifton had no idea what shewas getting into when she tried toget compassionate use of AMN107 for her partner, LelandEmerson. He could not qualify for thetrial as he had alreadytaken Gleevec, Sutent andAMG706. Being ever thevigilant caregiver, Judibegan the process whichwould end up consuming all of her timeand possibly her sanity.There are four required parts to ob-taining AMN107 via “compassionateuse”: the doctor application, the clinicaltrial agreement (CTA), the InstitutionalReview Board (IRB) approval and thepatient and doctor signature.The actual story is a long and compli-cated one that would make your headspin. However, here are the highlights of what was almost a tragedy.The first problem Judi encounteredwas going to a satellite location of adowntown cancer center. They wereunable to handle such a task and put the
By Erin Kristoff 
Newsletter Editor
n Sunday, October 14, theself-named “Viva Carolina”marathon team finally ac-complished what they hadbeen preparing for: the Long BeachMarathon and reaching their fundraisinggoal.Paul Montuori thought he had an am-bitious goal when he set it at $10,000but he wanted to “shoot high and think big and actually make a difference.”Well, Paul exceeded even his own ex-pectations; over 98 percent of thoseasked for a contribution helped out. Thegroup managed to raise an astounding$14,053 for the Life Raft Group in honorof member, Carolina Ponce-Williams!Chad, Kira, Paul and Hillary Montuoriwould like to thank everyone who par-ticipated that day including: Phillip andEriko Archunde; Randy and WendyHorn; Carlos and Beatriz Fernandez;Jean Paul and Bob Mercado; Gerald
See MARATHON, Page 5
See AMN107, Page 11
Runners pause before the big event.
Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — November 2007 — 
The Life Raft Group
Who are we, what do we do?
The Life Raft Group is an interna-tional, Internet-based, non-profit organi-zation offering support through educa-tion and research to patients with a rarecancer called GIST (gastrointestinalstromal tumor). The Association of Can-cer Online Resources provides thegroup with several listservs that permitmembers to communicate via secure e-mail. Many members are being suc-cessfully treated with an oral cancerdrug Gleevec (Glivec outside theU.S.A.). This molecularly targeted ther-apy represents a new category of drugsknown as signal transduction inhibitorsand has been described by the scientificcommunity as the medical model for thetreatment of cancer. Several new drugsare now in clinical trials.
How to join
GIST patients and their caregiversmay apply for membership free ofcharge at the Life Raft Group’s Website, www.liferaftgroup.org or bycontacting our office directly.
Privacy is of paramount concern, andwe try to err on the side of privacy. Wedo not send information that might beconsidered private to anyone outsidethe group, including medical profession-als. However, this newsletter serves asan outreach and is widely distributed.Hence, all articles are edited to maintainthe anonymity of members unless theyhave granted publication of more infor-mation.
How to help
Donations to The Life Raft Group,incorporated in New Jersey, U.S.A., asa 501(c)(3) nonprofit organization, aretax deductible in the United States.Donations, payable to The Life RaftGroup, should be mailed to:The Life Raft Group40 Galesi Dr., Suite 19Wayne, NJ 07470
We are patients and caregivers, notdoctors. Information shared is not asubstitute for discussion with your doc-tor. As for the newsletter, every effort toachieve accuracy is made but we arehuman and errors occur. Please advisethe newsletter editor of any errors.
Looking for new ways to fight GIST 
By Jerry Call
LRG Science Coordinator
n 1991, Joseph Schlessinger,PhD, and Axel Ullrich, PhD,co-founded a small bio-techcompany. Taking the first letterof each of their names, they namedthe company Sugen. Sugen was con-sidered to be one of the early pio-neers in the signal transduction field.Many Life Raft Group memberswill recognize the name Sugen. It iswhat GIST patients called the experi-mental drug SU11248 before it had areal name. SU11248 was a mouthful,so patients made up their own name,Sugen, named after the drug manu-facturer.Sugen was acquired by Pharmacia in2003 and Pharmacia was later acquiredby Pfizer. Pfizer completed developmentof SU11248 bringing the drug to trialsfor both Gleevec-resistant GIST andadvanced kidney cancer. In early 2006the drug became the first drug to be ap-proved for two cancers at the same time.The drug is now known as Sutent; ap-parently in tribute to those early pioneersat Sugen.On page six of this newsletter, we havepublished a Yale press release describ-ing recent advances in understanding themechanisms of KIT signaling. JosephSchlessinger and his colleagues continueto try to find ways to improve the treat-ment of GIST.The current generation of KIT inhibi-tors (drugs like Gleevec and Sutent)work on the inside of GIST cells byblocking the binding of ATP (needed forsignaling to occur) in the kinase regionof the receptor. This works very well aslong as the receptor is not mutated somuch that the drug is no longer able toreach the binding pocket in the kinase.Primary (initial) mutations seldom pre-vent Gleevec (the drug used as front-linetherapy) from reaching the drug/ATPbinding pocket. Secondary mutationscan develop over time (or they were ini-tially present at low levels and becomedominant over time under selective pres-sure) and change the shape of the recep-tor enough that Gleevec can no longerbind to the receptor. Sutent and someother drugs have activity against some of the secondary muta-tions that cause re-sistance to Gleevecbut so far, no drug isknown to have activ-ity against all of themany secondarymutations that canoccur. Newer drugslike HSP90 inhibi-tors are trying toovercome this limi-tation.In their paper pub-lished in the journal,
, Dr. Schlessinger and his colleaguesdescribe how two KIT receptors jointogether to become activated. This proc-ess, called dimerization, occurs outsideof the cell. Since dimerization and bind-ing of ATP are both required for KITsignaling to occur, both represent drugtargets. Blocking ATP binding withGleevec is a proven approach, but sub- ject to derailment by secondary muta-tions. Blocking dimerization with a dif-ferent type of drug that “can form wedgein the KIT molecule”, as noted bySchlessinger, provides a potential ap-proach that could conceivable bypass thesecondary mutations that occur in thekinase domain. Since the dimerizationarea is at or near the exon 9 region of KIT, it will be interesting to see if Schlessinger’s work translates into in-creased understanding of exon 9 muta-tions.According to Schlessinger, “Thiswork provides a roadmap for how todevelop new drugs that will overcomethe resistance to Gleevec and Sutent thatdevelops in GIST patients and in otherdiseases driven by activated form of KIT. We are now starting to pursue thisidea and raise the necessary funds forthis to be accomplished. Although it willtake time and quite a lot of funds I amvery optimistic that this goal could beaccomplished.”
Ensuring That No One Has To Face GIST Alone — Newsletter of the Life Raft Group — November 2007 — 
November 2007 clinical trial update
By Jim Hughes
LRG Science Team
Phase III continues to enrollpatients at a rapid rate, according to No-vartis. Sites listed in the clinicaltri-als.gov database are open with the fol-lowing exceptions as of October 12: LeeMoffitt in Tampa, FL, MD Anderson inHouston, TX, and New York University,New York, NY. These sites were not yetopen. Over 30 other sites are open out-side the United States, in Canada,Europe, Australia and Taiwan.
Phase I is suspended.
Oblimersen (Genasense) + Imatinib:
 Phase II is no longer accruing patients.
Phase I for Advanced SolidTumors is open at the Nevada CancerInstitute in Las Vegas, NV. BEZ235 is aNovartis drug that targets the PI3K tyro-sine kinase and indirectly inhibits thedownstream targets AKT and mTOR.
Imatinib + Pegylated Interferon-a 2B:
This Phase II trial for GIST is a wholedifferent approach. It is primarily forpatients who are newly diagnosed andwho have not had Gleevec (or have beenoff Gleevec for six months or longer).Dr. Lei L. Chen is the primary investiga-tor at the Huntsman Cancer Institute inSalt Lake City, Utah. Dr. Chen theorizesthat Gleevec causes GIST cell deathwhich in turn marks GIST cells for im-mune system response. Interferon willhelp stimulate that response and aid theimmune system in identifying and de-stroying the remaining GIST stem cellsthat can cause relapse after Gleevec hasshrunk tumors. This trial is planned tohave several other innovative aspectsthat are explained in detail in the May2007 Newsletter article “Immunotherapytrial strives to improve Gleevec re-sponse” by Jerry Call, also available onthe LRG website.
Dana-Farber— Travis Quig-ley, RN, Research Nurse, has indicatedthat both the Sorafenib (BAY 43-9006)Phase II and the Phase III Sunitinib orImatinib trial are available there.
 (Sutent®, Gleevec®)
Safety and effectiveness of daily dosing withsunitinib or imatinib in GIST patients
Phase:Conditions:Strategy:NCT#:US Contact:Telephone:Sites:IIIGISTMultiple TargetsNCT00372567
Pfizer Clinical Trial Informationpfizercancertrials@emergingmed.com
877-369-9753Contact Pfizer
, Boston, MATravis Quigley, RN, 617-632-5117IIGISTMultiple TargetsNCT00455559
Online Collaborative Oncology Group
Cancer Center at Century City,
 Los Angeles, CASant Chawla, MD
Coeur D’Alene, ID
 Oncology Specialists
, Park Ridge, ILKathy Tolzein, RN, 847-268-8200Grand Rapids, MI
Sayre, PA
 MD Anderson
, Houston, TX800-392-1611
Phase II study of Perifosine plus Gleevec for GIST patients
Phase:Conditions:Strategy:NCT#:US Contact:Telephone:Sites:
(nilotinib, Tasigna
 Efficacy and safety of AMN107 compared tocurrent treatment options in GIST patientswho failed imatinib and sunitinib
IIIGISTInihibit KITNCT00471328Novartis800-340-6843, Trial# CAM-N107A2201
, Los Angeles, CAMyung Lee, 310-825-4494
Wash. Cancer Inst.
, Wash. DCJake Paterson, 202-877-5371
Univ. of Chicago
, Chicago, ILPatient Coordinator:773-834-7424
Dana Farber
, Boston, MATravis Quigley, RN, 617-632-5117
Karmanos Cancer Institute.
, De-troit, MIAnne Marie Ferris, 313-576-9373
Wash. Univ
., St. Louis, MONick Fisher, 314-354-5102
Wake Forest
, Winston-Salem, NCScarlet Hutchins, RN, 336-713-6915
Fox Chase
, Philadelphia, PA1-800-FOX-CHASEPhase:Conditions:Strategy:NCT#:US Contact:Telephone:Sites:
Imatinib +Pegylated Interferon-a 2B
Combines targeted therapy with immunother-apy using Imatinib + Pegylated Interferon-a2B in imatinib-naïve GIST patient 
IIGISTKill GIST cellsHCI 22172
Huntsman Cancer Institute
Candace, 801-581-4477Phase:Conditions:Strategy:Study #:US Contact:
 (BAY 43-9006, Nexavar®)
Sorafenib in treating patients with malignant GIST that progressed during or after treat-ment with imatinib and sunitinib
Phase:Conditions:Strategy:NCT#:US Contact:Telephone:Sites:IIGISTMultiple TargetsNCT00265798
Univ. Of Chicago Cancer Re-search Center
, Chicago, IL773-834-7424
City of Hope,
Duarte, CAWarren Chow, MD, 866-434-4673xt 64215
 Cancer Care Specialists,
Decatur,ILJames Wade III, MD, 217-876-6617
 Oncol./Hematology Assoc. of Cent
, Peoria, ILJohn Kugler, MD, 309-243-3605
Dana Farber
, Boston, MATravis Quigley, RN: 617-632-5117
Memorial Sloan-Kettering,
NewYork, NYDavid D’Adamo, MD, 212-639-7573
In last month’s newsletter we reportedthat 2 patients who were resistant toGleevec and other drugs appeared to beresponding to Nexavar. We have subse-quently learned that the patient withsymptomatic improvement, that wasable to leave hospice, demonstrated pro-gression upon further examination.
Up to the minute...
See TRIALS, Page 9

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