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Cancer in Our Genes International Patient Database. A patient-driven database dedicated to finding a cure for VHL and other cancers. How is CGIP “International”?. English only in Phase 1; translation to other languages will follow

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cancer in our genes international patient database

Cancer in Our GenesInternational Patient Database

A patient-driven database dedicated to finding a cure for VHL and other cancers

how is cgip international
How is CGIP “International”?
  • English only in Phase 1; translation to other languages will follow
  • US / Canada / UK / Australia / New Zealand and anyone else wishing to respond in the English language
  • Questions are designed to be of universal interest
    • Input from international group
  • The database belongs to the VHL Alliance
  • The vision is global
why multiple conditions
Why Multiple Conditions?
  • VHL, HLRCC, BHD, and SDHB all increase the odds of getting kidney cancer.
  • Each is caused by a different genetic alteration, in a different gene
  • If any one of these four genes can cause kidney cancer, what do they have in common? What is it that they all help to control?
  • What can we learn from their similarities and differences?
    • Cross-disease analysis (beyond these 4 diseases)
    • Eventual inclusion of clinical data
global rare disease initiative
Global Rare Disease Initiative
  • VHLA is working with NORD and others around the world to create a global repository for rare disease information
  • Many sources of data will feed into this common data set
  • FDA is providing feedback

on questionnaire design

who owns the cgip data
Who Owns the CGIP Data?
  • The VHL Alliance owns the data
  • The VHL Alliance is the only one who will be able to connect your identity to your health information (“identified” data)
  • All others will receive only “de-identified” data
    • No names
    • No addresses or phone numbers
    • No other identifying information
goals of the cgip database
Goals of the CGIP Database
  • Create a unified resource for both researchers and constituents
  • Include a significant number of patients to allow meaningful statistical analysis
  • Provide a natural history of VHL and other genetic disorders also leading to kidney tumors and other lesions
  • Identify best practices for diagnosis and treatment
goals of the cgip database continued
Goals of the CGIP Database(continued)
  • Give a complete “picture” of each VHL patient
  • Reveal correlations between known effects and other conditions and between diseases, learning from commonalities and differences
  • Expedite matching patients to clinical trials
advantages of a patient driven database
Advantages of a Patient-Driven Database
  • Only the patient can provide the complete “picture” of their disease and quality of life
  • Will yield insights into lifestyle and quality of life factors (ie: nutrition, exercise, mental outlook)
  • Includes usage of both prescription and non-prescription medications
  • Shows family history (blood relatives)
benefits to the participants
Benefits to the Participants
  • Expedited matching to clinical trials
    • Participants will be notified of researcher interest by the VHLA and must initiate contact if they want to be considered for a clinical trial or other research project
  • Documents on medical history and medications can be downloaded to share with physicians during office visits
  • Screening reminders sent to participants
database updated annually
Database Updated Annually
  • Participants may access the database at any time to update their information
  • Everyone will be asked to provide annual updates
    • Provides a natural history of VHL
    • Keeps database relevant for researchers
  • Questions will be revised over time as we learn more about VHL and related conditions
contribute your experiences to advance research
Contribute Your Experiences to Advance Research
  • Today researchers are limited to the data compiled by a single hospital or research team
  • This clinician sourced data provides only a limited picture of disease
  • With the global CGIP database, researchers will have access to the annually updated compiled information of thousands of patients.
  • We need as many people as possible to contribute experiences and information!
cgid support of clinical trials
CGID: Support of Clinical Trials
  • Expedite Clinical Trials
    • Identify candidate patients
      • Includes questions recommended by the FDA for clinical drug trials
    • Linked to tissue bank
  • Provide natural history data for comparison
    • Help determine promising approaches for drug development
    • Answer “Did the drug make a difference?”
  • Learn from all experimentation
    • Learn from off-label use experiences and interactions with other prescribed or over-the-counter drugs and supplements
  • Better and harmonized collection of outcomes
research questions so far
Research Questions (so far):
  • What is the natural history of these syndromes?
  • Genotype/phenotype alignment?
  • Why is there a difference in presentation between siblings?
  • Are de novo mutations more aggressive?
  • What is the role of epigenetics?
  • What is the role of environmental factors?
  • What role does lifestyle play?
    • Stress/Depression, Nutrition/Diet, Exercise
  • Do medications impact tumor growth?
research questions so far1
Research Questions (so far):
  • Is thyroid a feature of VHL?
  • Does tumor aggression correlate with …?
  • Does oral health correlate with …?
  • Does treatment with targeted therapies or SRS modify the course of the disease?
  • Does pregnancy modify the course?
    • What about other sources of hormones?
  • Does the risk of long-term CNS deficits increase with each intervention?
  • Does early pre-symptomatic screening really help?