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LIFE RAFT GROUP A New Model for Cancer Research    December 2009
THE LIFE RAFT GROUP Dedicated to finding a cure for Gastrointestinal Stromal Tumors (GIST)
<ul><ul><li>G astro- </li></ul></ul><ul><ul><li>I ntestinal  </li></ul></ul><ul><ul><li>S tromal  </li></ul></ul><ul><ul><...
Survival History of GIST <ul><li>Treatment response went from <5% to 85% </li></ul><ul><li>2000 brought Gleevec  </li></ul>
Why is GIST relevant to other cancers? <ul><li>It serves as a model for other more common cancers </li></ul><ul><ul><li>“ ...
GIST Approved Drugs <ul><li>    FDA   2008 </li></ul><ul><li>  Approved   Sales </li></ul><ul><li>Gleevec     2002 $3.7 B ...
Potential Therapies Genotype Current Initial Therapy Potential Initial Therapies Imatinib-Resistance KIT exon 11 Imatinib ...
KIT Gleevec - Sunitinib - Dasatinib-Sorafenib-Nilotinib PKC412-AMG706-MP470-HSP-90 inhibitors (indirect) PI3K BEZ235 - GDC...
Gleevec Resistance: Median Time to Progression is 24 Months
ABOUT THE LIFE RAFT GROUP
Our Mission LRG Member & GIST Patient Andrea Fuller SURVIVAL
<ul><li>Patient Support </li></ul><ul><li>We intervene in cases where patients have needed to gain access to treatment, do...
<ul><li>Our monthly newsletter reaches thousands of patients, caregivers and medical professionals. </li></ul>
Webcasts <ul><li>We hold “webcasts”, online video seminars that enable us to bring up-to-date information to the public by...
Clinical Trials Database <ul><li>We maintain an extensive on-line Clinical Trials database to help patients and physicians...
Networking Dr. David Epstein, head of Novartis Oncology worldwide Dr. James Watson, Nobel Prize Winner Ted Kennedy and  Da...
<ul><li>Global  </li></ul><ul><li>GIST Network </li></ul>Global Outreach <ul><li>The LRG has expanded and strengthened the...
CHALLENGES FOR CANCER RESEARCH
What’s wrong with this? ASCO 2007: “Major Research Advances in Cancer Treatment” Treatment   Improvement   Avastin Doubled...
Issues with the Clinical Trials Process <ul><li>The current system is: </li></ul><ul><ul><li>Too slow </li></ul></ul><ul><...
The LRG Response
LRG’S RESEARCH STRATEGY
GIST Research Strategy <ul><li>Recruit a world class pre-clinical research team </li></ul><ul><li>Create a strategic plan ...
GIST Research Strategy <ul><li>Strengthen data sharing capacity of team </li></ul><ul><li>Create Patient Registry with com...
About the Patient Registry LRG maintains a sophisticated patient database <ul><li>Over 1,200 patient-provided medical hist...
<ul><li>Comprehensive information is  </li></ul><ul><li>collected on every  </li></ul><ul><li>new patient  </li></ul><ul><...
About the Patient Registry <ul><li>Other features: </li></ul><ul><li>Gistory </li></ul><ul><li>Mass mailing </li></ul><ul>...
Outcomes <ul><li>Unique information not being addressed by clinical trials </li></ul><ul><ul><li>Actual Dosage vs. Startin...
Outcomes <ul><li>Quality control of formal clinical trial data </li></ul><ul><ul><ul><li>Capture information which patient...
What We Have Learned So Far <ul><li>Patients/caregivers can reliably report treatment efficacy </li></ul><ul><ul><li>Shrin...
GIST Collaborative Tissue Bank and the Patient Registry <ul><li>Partnership between Patients, GIST Researchers and the LRG...
Our Collaborators: LRG Research Team Dr. Brian Rubin, TheCleveland Clinic Dr.  Jonathan Fletcher, Brigham & Women’s Hospit...
LRG’s Research Priorities
Research Budget <ul><li>Annual research expenditure of $1,000,000 for the last 3 years. </li></ul><ul><li>We want to expan...
What We Are Looking For <ul><li>People and companies that can think outside of the box and help us build a new model for f...
What We Are Looking For <ul><li>Venture Philanthropists who want to support a better way to find a cure for cancer </li></...
Summary <ul><li>We have a world class research team collaborating to implement a strategic plan. </li></ul><ul><li>We have...
Summary <ul><li>We have the largest GIST patient medical database in the world and a collaborative tissue bank of rare GIS...
For More Information Norman J. Scherzer Executive Director 973-837-9092 [email_address] www.liferaftgroup.org
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Life Raft Group: A New Model for Cancer Research

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The Life Raft Group: A New Model for Cancer Research presentation at the 2009 Partnering for Cures conference in New York

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  • PKC-theta – important target in GIST, but no drug yet (1-2009) RAS may be a potential target for NF-1 type GISTs
  • We will discuss mutational and plasma testing further in a general session Sunday. There are also free plasma level testing kits available for patients.
  • We will discuss mutational and plasma testing further in a general session Sunday. There are also free plasma level testing kits available for patients.
  • We will discuss mutational and plasma testing further in a general session Sunday. There are also free plasma level testing kits available for patients.
  • We will discuss mutational and plasma testing further in a general session Sunday. There are also free plasma level testing kits available for patients.
  • Transcript of "Life Raft Group: A New Model for Cancer Research"

    1. 1. LIFE RAFT GROUP A New Model for Cancer Research December 2009
    2. 2. THE LIFE RAFT GROUP Dedicated to finding a cure for Gastrointestinal Stromal Tumors (GIST)
    3. 3. <ul><ul><li>G astro- </li></ul></ul><ul><ul><li>I ntestinal </li></ul></ul><ul><ul><li>S tromal </li></ul></ul><ul><ul><li>T umors </li></ul></ul>A rare type of cancer-- 5,000 to 10,000 US cases/year One type of Sarcoma Sarcoma’s are a group of rare cancers that arise from cells of connective tissues, muscles, cartilage, bone, blood vessels, etc. What Is GIST?
    4. 4. Survival History of GIST <ul><li>Treatment response went from <5% to 85% </li></ul><ul><li>2000 brought Gleevec </li></ul>
    5. 5. Why is GIST relevant to other cancers? <ul><li>It serves as a model for other more common cancers </li></ul><ul><ul><li>“ By studying GIST, we expect to learn new things about the big four killers - breast, colon, lung, and prostate cancers. Science and medicine often make the biggest leaps forward in unusual, rare diseases.“ George Demetri, Harvard Gazette </li></ul></ul><ul><ul><li>“ GISTs appear to serve as a model for molecule-based diagnosis and treatment of solid tumors.” Y. Kitamura; Cancer Sci 2003 </li></ul></ul><ul><ul><li>“ There is considerable optimism that the targeted molecular approach being applied to the treatment of GISTs will serve as a model for the development of similar approaches to other more common tumors.” MS Saund, George Demetri; Curr Opin Gastroenterol, 2005 </li></ul></ul>
    6. 6. GIST Approved Drugs <ul><li> FDA 2008 </li></ul><ul><li> Approved Sales </li></ul><ul><li>Gleevec 2002 $3.7 B </li></ul><ul><li>Sutent 2006 $ .8 B </li></ul>The GIST community has led the way in the development of molecularly targeted drugs in solid tumors …
    7. 7. Potential Therapies Genotype Current Initial Therapy Potential Initial Therapies Imatinib-Resistance KIT exon 11 Imatinib Masitinib Nilotinib Sorafenib Imatinib + PI3K inhibitor Sunitinib (poor) Sorafenib HSP90 inhibitors KIT exon 9 Standard dose imatinib High dose imatinib Sunitinib Sunitinib HSP90 inhibitors Wild-type Imatinib (poor response) Sunitinib Nilotinib Masitinib IGF1R inhibitors Sunitinib Nilotinib Masitinib IGF1R inhibitors PDGFRA D842V (exon 18) Imatinib (very poor) Dasatinib HSP90 inhibitors Dasatinib HSP90 inhibitors
    8. 8. KIT Gleevec - Sunitinib - Dasatinib-Sorafenib-Nilotinib PKC412-AMG706-MP470-HSP-90 inhibitors (indirect) PI3K BEZ235 - GDC0941 SF1125-XL765 AKT Perifosine mTOR RAD001 - CCI779 AP-23573-Rapamycin PKC-θ RAS R115777-SCH66336 RAF-1 Sorafenib MEK MAPK BCL-2 Gentasense BCL-X L BAD Src/Fyn/Lyn Dasatinib Survive Proliferate VEGFR (antiangiogenesis) Sunitinib-AMG706-PKC412 Sorafenib-Avastin Progress in understanding GIST Targets and Treatments HSP90 IPI504 - BIIB021 AUY922-STA9090 SNX5422 Other Receptors IGF1R (wild-type) AXL HDAC LBH589-SAHA STAT3 Survive-grow
    9. 9. Gleevec Resistance: Median Time to Progression is 24 Months
    10. 10. ABOUT THE LIFE RAFT GROUP
    11. 11. Our Mission LRG Member & GIST Patient Andrea Fuller SURVIVAL
    12. 12. <ul><li>Patient Support </li></ul><ul><li>We intervene in cases where patients have needed to gain access to treatment, doctors and other life-saving options. </li></ul><ul><li>We consult with patients on individualized treatment issues. </li></ul>
    13. 13. <ul><li>Our monthly newsletter reaches thousands of patients, caregivers and medical professionals. </li></ul>
    14. 14. Webcasts <ul><li>We hold “webcasts”, online video seminars that enable us to bring up-to-date information to the public by experts in the field. </li></ul><ul><li>We then archive these webcasts so that they can be viewed later at </li></ul><ul><li>any time. </li></ul>
    15. 15. Clinical Trials Database <ul><li>We maintain an extensive on-line Clinical Trials database to help patients and physicians stay informed about up-to-the-minute treatment options </li></ul><ul><li>  </li></ul>
    16. 16. Networking Dr. David Epstein, head of Novartis Oncology worldwide Dr. James Watson, Nobel Prize Winner Ted Kennedy and Dan Vasella, CEO of Novartis ACOR Pres., Gilles Frydman and Dana-Farber Sarcoma Chief, George Demetri
    17. 17. <ul><li>Global </li></ul><ul><li>GIST Network </li></ul>Global Outreach <ul><li>The LRG has expanded and strengthened the Global GIST Network. Representatives </li></ul><ul><li>are now available in 50 countries. </li></ul><ul><li>The LRG will be launching an extensive Latin American initiative in 2010 </li></ul>
    18. 18. CHALLENGES FOR CANCER RESEARCH
    19. 19. What’s wrong with this? ASCO 2007: “Major Research Advances in Cancer Treatment” Treatment   Improvement   Avastin Doubled Progression Free Survival Sorafenib 44% longer life   Radiotherapy 53% longer life Improvement OUTCOME FOR PATIENTS 5.4 months to 10.2 months 4.8 months 7.9 months to 10.7 months 2.8 months 17 weeks to 29 weeks 3 months
    20. 20. Issues with the Clinical Trials Process <ul><li>The current system is: </li></ul><ul><ul><li>Too slow </li></ul></ul><ul><ul><li>Too costly </li></ul></ul><ul><ul><li>Too ineffective </li></ul></ul><ul><ul><li>Studies have shown that “NCI cooperative groups routinely take 2 years to bring a trial from concept to active accrual, that nearly 60% of such trials opened for 5 years had fewer than five patients enrolled at each site, and that in more than 20% of the studies, not a single subject had been accrued.” </li></ul></ul><ul><ul><li>(JNCI, 2009) </li></ul></ul>
    21. 21. The LRG Response
    22. 22. LRG’S RESEARCH STRATEGY
    23. 23. GIST Research Strategy <ul><li>Recruit a world class pre-clinical research team </li></ul><ul><li>Create a strategic plan </li></ul><ul><li>Establish agreement to collaborate and coordinate </li></ul><ul><li>Make longer grant commitments </li></ul><ul><li>Reduce overhead </li></ul><ul><li>Require in-person team meetings </li></ul>     
    24. 24. GIST Research Strategy <ul><li>Strengthen data sharing capacity of team </li></ul><ul><li>Create Patient Registry with comprehensive clinical histories across national and institutional boundaries </li></ul><ul><li>Expand researcher’s access to patient tissue through a GIST Collaborative Tissue Bank </li></ul><ul><li>Create link between lab-based research efforts and patient registry database </li></ul><ul><li>Build a GIST Clinical Trial Model </li></ul>    
    25. 25. About the Patient Registry LRG maintains a sophisticated patient database <ul><li>Over 1,200 patient-provided medical histories including mutational and plasma level testing </li></ul><ul><li>Data cuts across institutional and international boundaries </li></ul><ul><li>Secure, online access for research team </li></ul><ul><li>Patient Data linked to tissue bank </li></ul>
    26. 26. <ul><li>Comprehensive information is </li></ul><ul><li>collected on every </li></ul><ul><li>new patient </li></ul><ul><li>including: </li></ul><ul><li>Diagnosis </li></ul><ul><li>Evaluation </li></ul><ul><li>Treatment </li></ul>About the Patient Registry <ul><li>Other tabs : </li></ul><ul><li>Current Status </li></ul><ul><li>TimeLine </li></ul><ul><li>Outreach </li></ul>
    27. 27. About the Patient Registry <ul><li>Other features: </li></ul><ul><li>Gistory </li></ul><ul><li>Mass mailing </li></ul><ul><li>Statistics </li></ul><ul><li>Study Groups </li></ul><ul><li>Clinical Trial </li></ul><ul><li>Tissue Bank </li></ul>
    28. 28. Outcomes <ul><li>Unique information not being addressed by clinical trials </li></ul><ul><ul><li>Actual Dosage vs. Starting Dosage demonstrates that higher doses are related both to longer progression-free survival and longer overall survival </li></ul></ul><ul><ul><li>Quality of life information supplements NCI toxicity-based side-effects data </li></ul></ul><ul><li>Timely information that cannot wait for formal trial results </li></ul>
    29. 29. Outcomes <ul><li>Quality control of formal clinical trial data </li></ul><ul><ul><ul><li>Capture information which patient may not be willing to share with doctor </li></ul></ul></ul><ul><li>Complete GIST clinical histories not interrupted by change in medical care provider </li></ul><ul><li>Link patients clinical histories to their tissue samples to create unique resource for GIST researchers </li></ul>
    30. 30. What We Have Learned So Far <ul><li>Patients/caregivers can reliably report treatment efficacy </li></ul><ul><ul><li>Shrinkage </li></ul></ul><ul><ul><li>Stability </li></ul></ul><ul><ul><li>Progression </li></ul></ul><ul><li>Patients/caregivers can reliably report quality of life side-effects </li></ul><ul><li>Patients/caregivers can reliably report survival </li></ul>
    31. 31. GIST Collaborative Tissue Bank and the Patient Registry <ul><li>Partnership between Patients, GIST Researchers and the LRG Patient Registry </li></ul><ul><ul><li>Patient-driven process </li></ul></ul><ul><ul><li>GIST researchers share tissue and data </li></ul></ul><ul><ul><ul><li>Efficient use of rare tissue </li></ul></ul></ul><ul><ul><ul><li>Online access for researchers of de-identified clinical histories </li></ul></ul></ul><ul><ul><ul><li>Research-generated data shared through Stanford Tissue Microarray website (www.tma.stanford.edu) </li></ul></ul></ul><ul><ul><li>Maintains patient privacy and provides direct patient benefits including free mutational testing </li></ul></ul>
    32. 32. Our Collaborators: LRG Research Team Dr. Brian Rubin, TheCleveland Clinic Dr. Jonathan Fletcher, Brigham & Women’s Hospital Dr. Chris Corless, Oregon Health & Science University Dr. Michael Heinrich, Oregon Health & Science University Dr. Cristina Antonescu, Memorial Sloan Kettering Cancer Center Dr. Anette Duensing, University of Pittsburgh Cancer Center Dr. Matt van de Rijn, Stanford University Dr. Maria Debiec-Rychter, University of Leeuven, Belgium Dr. Sebastian Bauer, University of Essen, Germany Dr. Peter Besmer, Memorial Sloan Kettering Cancer Center
    33. 33. LRG’s Research Priorities
    34. 34. Research Budget <ul><li>Annual research expenditure of $1,000,000 for the last 3 years. </li></ul><ul><li>We want to expand pre-clinical research to $2,000,000 per year </li></ul><ul><li>Invest an additional $10-15 million dollars to create a clinical trial consortium in 2012. </li></ul>
    35. 35. What We Are Looking For <ul><li>People and companies that can think outside of the box and help us build a new model for finding the first cure for cancer </li></ul>
    36. 36. What We Are Looking For <ul><li>Venture Philanthropists who want to support a better way to find a cure for cancer </li></ul><ul><li>Partnerships with investors and companies to bring drugs to market through a more efficient and effective clinical trials process </li></ul><ul><li>Feedback </li></ul>
    37. 37. Summary <ul><li>We have a world class research team collaborating to implement a strategic plan. </li></ul><ul><li>We have made major progress in identifying mutational targets and treatments, including Gleevec. </li></ul><ul><li>We have access to a worldwide and motivated patient community. </li></ul>
    38. 38. Summary <ul><li>We have the largest GIST patient medical database in the world and a collaborative tissue bank of rare GIST tumors. </li></ul><ul><li>We have the perfect cancer research model. </li></ul>
    39. 39. For More Information Norman J. Scherzer Executive Director 973-837-9092 [email_address] www.liferaftgroup.org
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