good, better, best: envisioning the future€¦ · change (ccm diet)/transplants, cheaper ivf with...
TRANSCRIPT
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Good, Better, Best: Envisioning the Future
Connie Lee, President and CEO, Angioma Alliance
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Our Mission
To inform, support, and empower those affected by cavernous angioma and drive research for better treatments and a cure
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2006 2018 2024 2030
Not Good
Good BestBetter
ØDiagnosisØTracking/PredictingØLesion Treatment
OptionsØMulti-Systemic Impacts
ØRecovery/RestorationØComprehensive Clinical
CareØSupportive Community
The Full Picture
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u Inconsistent diagnosis
u Few natural history studies
u Surgery/radiosurgery were the only interventions
u What multi-systemic effects?
u Basic rehab
u What prevention?
u A few knowledgeable surgeons and fewer neurologists
u Loose patient community
2006 2018 2024 2030
Not Good
Good BestBetter
2006: Where We Were
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More questions than answers
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u Diagnosis - SWI MRI; genetic testing (Angioma Alliance)
u Course - Natural history data regarding hemorrhage, incidental lesions, genetics, proliferation.
u Treatment - Surgery, Minimally invasive surgery, Vitamin D
u Identifying multi-systemic issues and causes/consequences
2006 2018 2024 2030
Not Good
Good BestBetter
2018: Where We Are
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u Rehab - More informed PT, OT, surgery, orthotics, robotics
u Prevention - Option of IVF with PGD
u Care - 7+ US Clinical Centers recognized for providing expert multi-disciplinary care
u Community - We can find each other, offer support, participate in advocacy/research/raising funds.
2006 2018 2024 2030
Not Good
Good BestBetter
2018: Where We Are
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u Genes identified/testing
u Scientific Meeting to advance collaborations
u DNA/Tissue Bank
u Legislation and advocacy
u SAIVMS, BVMC Project and other natural history studies
u Centers of Excellence
u Community building
How Did We Get Here?
2006 2018 2024 2030
Not Good
Good BestBetter
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u Accurate diagnosis - urine biomarkers (Boston Children’s), blood biomarkers, genetic testing covered by insurance, shorter MRI
u Accurate course prediction – blood and imaging biomarkers (U of Chicago)
u Medications and non-invasive treatments to stabilize vessels, reduce hemorrhage risk, reduce proliferation, reduce post-surgical recurrence
u Reduced multi-systemic impact of hereditary disease
2006 2018 2024 2030
Not Good
Good BestBetter
2024: Real Treatment Options!
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u Restoration of lost function - stem cell therapy trials? (Stanford)
u Prevention using microbiome change via individual diet change (CCM diet)/transplants, cheaper IVF with PGD
u Many Clinical Centers providing expert care around the world
u A community that is organized and mobilized for research participation, fund and awareness raising, and outreach to clinicians/patients.
2006 2018 2024 2030
Not Good
Good BestBetter
2024: Real Treatment Options!
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Path to the Future
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u Participate and/or support clinical trials and other clinical research: join patient registry, get testing, educate peers.
u Support basic/translational research: scientific meeting, advocacy, grants?
u Form many Community Alliances for outreach to medical community, etc.
u Increase international patient participation in research.
2006 2018 2024 2030
Not Good
Good BestBetter
How Do We Get There?
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Working Together Will Speed Progress!
Oceania: 38,000,000
South America: 422,000,000
North America: 579,000,000
Europe: 741,000,000
Africa: 1,216,000,000
Asia: 4,463,000,000
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u Fast, cheap, and accurate diagnosis - urine biomarkers, $50 genetic testing, $200 10-minute MRI
u Fast, cheap, 100% accurate course prediction –blood/imaging biomarkers, something else?
u Obliterate lesions with no recurrence or side effects using a low-cost one-time medication or non-invasive treatment.
2006 2018 2024 2030
Not Good
Good BestBetter
2030: The Final Destination
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u Eliminate multi-systemic impact of hereditary disease
u Cheap restoration of lost function as well as development of functions that didn’t have a chance in those affected as young children.
u Societal microbiome changes.
u Gene therapy for prevention of generational transmission.
2006 2018 2024 2030
Not Good
Good BestBetter
2030: The Final Destination
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u Informed medical providers everywhere, even primary care.
u We will be known in all medical settings and among the general public.
u The cure, prevention, and restorative treatments will be readily available.
2006 2018 2024 2030
Not Good
Good BestBetter
2030: The Final Destination
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u Do all the same things we do for better treatments! Organize, mobilize, participate!
u Create a tandem, alternate route that supplements the pharmaceutical industry – just in case.
2006 2018 2024 2030
Not Good
Good BestBetter
How Do We Get To a Cure?
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u Partner with other disease groups internationally whose illnesses may be amenable to same cure.
u Partner with governments, industries outside of pharma, and foundations.
u Fundraise to fill gaps in the development of cures.
2006 2018 2024 2030
Not Good
Good BestBetter
How Do We Get To a Cure?
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Take-Aways
u We are in a new era for cavernous angioma.
u This era will include better prediction, non-surgical treatments, informed care, and mobilized, supported patients.
u We are moving forward because of YOU and those around you!