Plenary 1

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Plenary 1

• Empowerment Through Research
• Role of Patient Organizations

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Life Raft Group

• For patients with GIST (Gastrointestinal Stromal
• Tumor)

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Our MissionIs Survival
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Food for Thought

• Can we distinguish between acts of outrage that
  make us feel good and acts of intervention that
  actually change something ?

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Survival History of GIST

• Prior to the summer of 2000
• Most GIST patients were misdiagnosed
• The only effective treatment was surgery
• When surgery was no longer an option death was
  inevitable
• The summer of 2000 brought Gleevec, C-Kit
  testing, and Hope
• The appearance of resistance is a particularly
  cruel setback
• Our primary focus now is to find ways to overcome
  this resistance

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Evolution of the Life Raft Group

• Informal Chat Group
• 501-C-3 Non-Profit Organization
• Extraordinary Growth
• Support and Information
• Advocacy
• Media and Outreach
• Cutting Edge Research

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Our Strategy

• To think smart enough to leverage our small
  numbers and overcome the rarity of GIST
• To use the combination of our own research, and
  the ability to publish in our newsletter and on
  our website, to carve out a seat at the decision
  making table, preferably at the head of the table

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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)

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Whats wrong with the traditional research
approach?

• High indirect costs from 50 to 75
• No overall strategic plan
• Poor accountability, sometimes none at all
• Individual research projects are not coordinated.
  In fact they are competitive

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Whats wrong, continued

• There is little sense of urgency
• There are conflicts of interest between pharma.
  companies, medical institutions and individual
  researchers
• And with patients needs for survival

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Lets summarize the traditional research approach

• Too costly
• Too slow
• Too inefficient
• In other words, not finding effective cancer
  treatments fast enough to keep us alive
• Do you remember the declaration of a war against
  cancer by then President Nixon?
• It was in 1971-35 years ago

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Can you imagine any other complex undertaking
being managed this way?

• Building a house?
• Conducting a symphony orchestra?
• Going to the moon?
• Constructing the first atomic bomb? I submit that
  the world would still be nuclear free if we had
  adopted the cancer research approach

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LRG Research Approach Our strategic thoughts

• We have very limited funds
• We have a strong sense of urgency driven by our
  mission of survival
• We believe that cures can only be achieved by a
  profound change in the basic management of
  research

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LRG Research Has Two Parallel Tracks

• GIST Treatment Resistance Initiative (topic of
  the plenary session)
• Patient Driven Research (topic of the workshop)

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1. LRG GIST Treatment Resistance Initiative

• We intend to find the reasons for GIST treatment
  resistance and the means to overcome them

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Our Mantra

• Excellence over Consensus
• Cooperation over Competition
• Strategy over Chaos
• Urgency over Lethargy

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LRG GIST Treatment Resistance

• Created a research team based upon value added
  expertise and synergy, and a commitment to
  principles of excellence and cooperation
• Asked them to develop a strategic plan focused
  upon overcoming treatment resistance and
  committed two million dollars in seed money to
  implement it
• The entire plan can be found on our website
  www.liferaftgroup.org

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LRG GIST Treatment Resistance

• Selected priority research project leaders and
  awarded them an annual stipend for coordinating
  research across institutional and national
  boundaries
• Issued first round grant awards
• Created two tissue banks

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Two Year Grant Awards

• Cristina Antonescu, MSK
• Peter Besmer, MSK
• Chris Coreless, OHSU/VA Hosp
• Maria Debiec-Rychter, Catholic Univ. Belg.
• Jonathan Fletcher, BW Hosp, Dana-Farber
• Michael Heinrich, OHSU/VA Hosp
• Matt van de Rijn, Stanford
• Brian Rubin, Univ. of Wash

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Tissue Banks

• Created two tissue banks, with emphasis upon
  frozen tissues
• At Memorial Sloan Kettering for pediatric GIST
• At Stanford University for adult GIST

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Added A Supportive Grants Structure

• Capped indirect costs at 0 for lt50,000 and 10
  for over 50,000
• Streamlined the paper work
• Removed red flags that could slow down the
  process, such as intellectual property rights
• Strengthened and fast tracked the research
  process with accelerated and supplemental funding
• Awarded two year grants, with six month funding
  cycles monitored by progress reports

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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 ones 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 and provide a source of hope to
GIST patients whose disease is resistant to
existing therapies. Your truly, Dan Vasella,
M.D.
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Next Phase

• Create mechanisms for data and information
  sharing
• Create a clinical trials consortium
• Secure additional funding