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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?