Executive Director Report September 16, 2006

1. Executive Director ReportSeptember 16, 2006 The State of the Life Raft Group and our battle to ensure the survival of patients with GIST

2. Our Mission Remains The Same Survival

3. Continuing To Find New DrugsAnd Better Ways of Using Them Targeted Research Life Raft Group Patient Data-bases Timely, International Treatment Surveillance Routine Mutational Testing and Record Keeping Initiative Comprehensive Clinical Site Planning Next Step: Clinical Trials Consortium

4. Making Sure That No One Dies Because They Cannot Access Life-Saving Treatment Behind the scenes intervention Focused Advocacy Medicare: We successfully fought to have Gleevec included as a covered prescription drug and are fighting to eliminate the Medicare prescription donut hole Placebos: We continue to oppose the use of placebos in clinical trials when a terminally ill patient has no other treatment options but the trial drug

5. Intervention Case Study A member of the Life Raft Group reached out to us in desperation. He was resistant to Gleevec and needed to start Sutent. The problem was that he had been waiting for several months for his medical center to begin a clinical trial that would permit him to have access to this drug. We were asked to write a letter to the head of the hospital. This is what happened.

6. 1. Our Letter to the Hospital Director Dear Dr. ____ “…(Patient’s) medical condition is deteriorating rapidly as you go through your internal procedures….In other words, he is dying as you, and your staff, are working on the paperwork for this trial…..The progression of life threatening illnesses like GIST may not…wait for the normal deliberative process….and may require that a responsible person intervene…I take personal responsibility for holding you accountable to that end…We shall be covering this story in our Newsletter…”

7. 2. The Hospital Director Responds “Dear Mr. Scherzer, I received with astonishment your letter…it seems that what you are really after ….is circumventing legal procedures and trying to influence legal judgment…Furthermore, I consulted with our legal advisors and we are all in the view that your letter should it get published constitutes…slander…Our institution shall take all necessary legal measures in that event…”

8. 3.Within 24 hours the patient concerned wrote to me “ Dear Norman, The good news reached us just now!!!!! Your letter…has done it…although Dr. ___ is upset…This news was given to us today by an official of the ….Cancer Association, calling from home…If you manage to move Dr. ___, I am sure you could move the Rocky mountains to Egypt. ..May god bless you….”

9. Making Sure that No One Dies Because of Ignorance Life Raft Group Monthly Newsletter: Content is aimed at both patient and medical professional Pamphlets and Educational Materials GIST Pediatric GIST Connecting the Dots (In Progress) Accessing Treatment (In Progress) Websites Life Raft Group Global GIST Network LRG Medical Professional Meetings

10. Making Sure that No One Dies Because of Ignorance: Pediatric GIST Virtual Pediatric GIST Center of Excellence Bringing together the best specialists in Oncology, Surgery and Pathology Creating an International Pediatric GIST Review Board Pediatric GIST Medical Data-base and Tissue Bank Next Step: A Physical Pediatric GIST Center of Excellence and Support for Patient Referrals

11. Ensuring that No One Has to Face GIST Alone Life Raft Group Online Communities Main Life Raft Group Listserv Pediatric GIST Listserv Other Specialized Listservs Global GIST Network, including foreign language listservs Life Raft Group Newsletters and Websites International GIST Specialist Directory

12. Ensuring that No One Has to Face GIST Alone-2 Local Life Raft and Sister Groups Around the World Patient Meetings One to One Consults Strategic Networks, In addition to the Global GIST Network With medical and research institutions around the world With other patient organizations

13. Life Raft GroupInternal Research: Based Upon Patient Input Treatment Efficacy, including actual drug dosage levels Side Effects New Priorities: Preventive Treatment Survivor Profiles Pediatric GIST, including drug dosage tolerance levels

14. LRG’s New Medical Database

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

17. Change in the US Death Rates by Cause, 1950 & 2001 Compared to the rate in 1950, the cancer death rate was 0.2% higher in 2001, while rates for other major chronic diseases decreased during this period. Compared to the rate in 1950, the cancer death rate was 0.2% higher in 2001, while rates for other major chronic diseases decreased during this period.

18. 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)

19. 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

20. How Does Life Raft Group Directed Research Differ From The Traditional Approach?

21. We Reduced High Overhead Costs

22. We Replaced 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.

23. 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

24. We Committed Two Year Grant Awards Cristina Antonescu, Memorial Sloan Kettering Peter Besmer, Memorial Sloan Kettering Chris Coreless, OHSU/VA Hosp Maria Debiec-Rychter, Catholic Univ. Belgium Jonathan Fletcher, B&W Hosp, Dana-Farber Michael Heinrich, OHSU/VA Hosp Matt van de Rijn, Stanford University Brian Rubin, Univ. of Wash (moving to Cleveland Clinic)

25. We Funded Two Tissue BanksTo Support The Search For A Cure At Memorial Sloan Kettering for pediatric GIST At Stanford University for adult GIST

26. 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

27. 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

28. 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

29. With Your Help We Can Find a Cure For Cancer