1. 2006 CTOS Changing Role of Patient Organizations In Cancer Research

2. Announcement A cure for all cancers has just been discovered!

3. GO HOME !

4. Just kidding On the other hand, One has to wonder

5. For patients with GIST (Gastrointestinal Stromal Tumor) Life Raft Group

6. Our Mission Is Survival

7. Keeping GIST Patients Alive By Ensuring That no one dies because they cannot access treatment That no one dies because of their ignorance or that of their physician That research is coordinated to urgently find a cure

8. LRG Research Has Two Parallel Tracks. 1.We conduct our own research using medical updates provided by patients and caregivers 2.We direct a coordinated strategy by the LRG world class research team to find a cure for GIST that will serve as a model for other cancers

9. We conduct our own research using medical updates provided by patients and caregivers

10. LRG’s New Medical Database

11. Why The Life Raft Group Conducts Its Own Research Timeliness Patient Relevance Information not available in clinical trials Survival

12. History Of Life Raft Group Research Gleevec Resistance: –Introduced evaluation of actual dosage as opposed to intended (starting) dosage –Evaluated relapse amongst metastatic GIST patients –Determined that there was a relationship between dosage and relapse Growing medical data-bases for adult and pediatric GIST

13. History Of Life Raft Group Research Gleevec Response Rates: First to report Gleevec Effectiveness for GIST patients Gleevec Side Effects: –Designed Severity Rating Scale for Patient Self Evaluation –First to report that Side Effects Got Better Over Time –First to report Gender Differences in Side Effects

14. The LRG ability to collect patient driven data and publish it on our website and in our newsletters had unintended consequences Turned the flow of clinical trial information upside down: patient and patient’s doctor get the information first Increased the leverage of the LRG regarding clinical trials

15. Time does not permit a more detailed discussion of the research we conduct using medical updates provided by patients and caregivers Instead I would direct my presentation to:

16. We direct a coordinated strategy by the LRG world class research team to find a cure for GIST that will serve as a model for other cancers

17. We have not made enough progress with the traditional approach to research Too many are still dying

18. Change in the US Death Rates by Cause, 1950 & 2001 * Age-adjusted to 2000 US standard population. Sources: 1950 Mortality Data - CDC/NCHS, NVSS, Mortality Revised. 2001 Mortality Data–NVSR-Death Final Data 2001–Volume 52, No. 3. http://www.cdc.gov/nchs/data/nvsr/nvsr52/nvsr52_03.pdf Cerebrovascular Diseases Heart Diseases Pneumonia/ Influenza Cancer Rate Per 100,000

19. Traditional Research Approach Donate money to an institution in the name of research, perhaps with a specific disease designation or General call for proposals from the research community: Individual projects are selected for funding (typical government approach)

20. What’s wrong with the traditional research approach? High overhead costs: from 50 to 75% No overall strategic plan; lack of coordination Little accountability Lack of urgency

21. How Does the Life Raft Group Strategy Address the Shortcomings of The Traditional Research Approach?

22. We Reduced High Overhead Costs The LRG reduced overhead costs in our research grants from between 50 to 75% down to no more than 10%

23. We Addressed A Lack of Strategy and Coordination We assumed a leadership role We brought together the best scientists in the world We told them that if they created a coordinated strategic plan, and agreed to cooperate, that we would fund it They did We did

24. Our Own Research Team Includes World Class Scientists From Catholic University, Leuven Belgium Cleveland Clinic Dana-Farber Cancer Center/Brigham & Women’s Hospital, Boston Memorial Sloan Kettering Oregon Health Sciences University/VA Hospital Stanford University

25. Our Research Team Members Have Dual Roles To Effectively Implement Their Own Institution’s Research Component of Our Strategic Plan To Coordinate a Priority Research Area Across Institutional and National Boundaries

26. Our Research Team Created A Strategic Plan We committed two year grant awards to implement it We published the plan on our website: www.liferaftgroup.org

27. We Funded Two Tissue Banks To Support The Search For A Cure –At Memorial Sloan Kettering for pediatric GIST –At Stanford University for adult GIST

28. We Introduced Accountability to the Research Grant Process We created six month funding cycles and required a satisfactory progress report to justify further funding We are prepared to shift funds from less promising to more promising areas

29. We introduced a sense of urgency to the search for a cure for GIST We created incentives to reward and accelerate progress by building in accelerated and supplemental funding of promising breakthroughs President Nixon declared war on cancer in 1971, 35 years ago.

30. Our evaluation so far April 18, 2006 Dear Norman, In the Life Raft Group Research plan of action, one finds both true dedication and conviction that can only emerge from one’s heart as well as efficiency and professionalism… It seems to me that this combination is the best ground for successful research and I would like to express my sincere respect and admiration for your efforts and the efforts of your group. I have no doubt that you will succeed in making a significant contribution to GIST research … Yours truly, Dan Vasella, M.D., CEO, Novartis

31. GIST Is The Perfect Model For Demonstrating How To Cure Other Cancers Relatively simple and increasingly understood mechanism of cancer mutations Growing list of targeted drugs to address these mutations Innovative research strategy Unprecedented collaboration between researchers, pharmaceutical companies and patients

32. Patient Roles in Cancer Research Have Evolved Passive Participant Some Input Seat at the Decision Making Table Seat at the Head of the Decision Making Table

33. Food for Thought Can we distinguish between acts of outrage that make us feel good and acts of intervention that actually change something ?

34. Thanks For Listening The Life Raft Group