Executive Director ReportExecutive Director ReportSeptember 16, 2006September 16, 2006

The State of the Life Raft Group and our battle to ensure the

survival of patients with GIST

Our Mission Remains The Same

SurvivalSurvival

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

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

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.

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…”

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…”

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….”

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

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

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

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

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

LRG’s New Medical Database

The Life Raft Group Has A Plan To Cure A Cancer Called GIST That Will Serve As A Model For Curing All Cancers

We have not made enough progress with the traditional

approach to research

Too many are still dying

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

CerebrovascularDiseases

21.8

193.9

586.8

48.1

180.7

245.8

194.4

57.5

0

100

200

300

400

500

600

1950

2001

HeartDiseases

Pneumonia/Influenza

Cancer

Rate Per 100,000

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)

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

How Does Life Raft Group Directed Research

Differ From The Traditional Approach?

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%

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.

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

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)

We Funded Two Tissue BanksTo Support The Search For A

Cure

–At Memorial Sloan Kettering for pediatric GIST

–At Stanford University for adult GIST

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

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.

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

With Your Help

We Can

Find a Cure For Cancer