National Disease Research Interchange

Report
National
Disease Research
Interchange
Advancing Medical Research Through
Organ and Tissue Donation
History & Overview
NDRI Mission Statement
To advance disease research through the
procurement, preservation and
distribution of human cells, tissues and
organs.
NDRI
•
Founded in 1980
•
Based in Philadelphia
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501(c)(3) Governed by BOD
•
Operates 7 days a week, 24 hours a day
•
Served more than 5,000 investigators with over
300,000 biospecimens
•
Funded by the NIH for thirty-plus consecutive
years
•
2,700 publications
Funded by National Institutes of
Health for 30 Years
NIH Office of the Director (ORIP, GTEx)
NIDDK NEI
NHGRI
NIAMS
NIAID
ORDR NHLBI
NIMH
NCI
Other Funding Streams
Corporate
•
•
Pharma/biotech industry
Provided tissues to > 330 for-profit
entities in last 5 years
Special Initiatives
•
•
•
•
•
Cystic Fibrosis Foundation Collaboration
Genotype-Tissue Expression Project
ATSDR/Boston VA ALS Projects
Joslin Medalist Diabetes Project
Children’s Tumor Foundation
Types of Tissue Requests
Standard Requests
• Eyes
Custom Project Requests
• Specialized sourcing
• Solid organs
• Special initiatives
• Tissue bank specimens
• Surgical specimens
o
o
o
o
Eye sections
Dorsal root ganglia
GTEx
ALS (VA, ATSDR)
NDRI Reach & Network
NDRI Procurement Network
240 Sites
56 Organ
Procurement
Organizations
46
Hospital
Donor Sites
37
Eye Banks
23
Tissue Banks
18 Satellite
Tissue
Source Sites
200
Recovery
Personnel
Biospecimen Diversity
• Normal
• Diseased – common and rare
• High quality, low-PMI organs and tissues
• Brain/CNS
• Cancer /normal adjacent
• Cord blood/placenta/umbilical cord
• Isolated normal /T1D/T2D pancreatic islet
cells
Versatility of Human Tissue in
Research
Viable, shipped at 4ºC
• Stem cell isolation
•
•
•
•
•
•
Regenerative medicine
Cell culture
Functional analysis
Single cell analysis
High throughput
screening
Toxicology/DMPK
Snap-frozen/fixed
• Expression analysis
• Genotyping
• IHC
• Proteomics
• Biomarkers
• Biomechanics
Functional Analysis
Project Examples
Genotype Tissue Expression Project
(GTEX)
• 5 year project - August 2010 to 2015
• 400 post mortem donors consented and
recovered
– 69% authorization rate
• PMI range 1:22 to 6:51 (O), 7:01 to 24:00 (T)
• 44,341 biospecimens collected
• 96% released to NIH CaHuB inventory
• 84% RNA integrity number > 6
GTEx Post-mortem Organs and Tissues
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Skeletal muscle
•
Tibial nerve
•
Tibial artery
•
Skin (leg)
•
Adipose tissue
•
Whole brain
•
Brain tissues
(cerebellum and
cortex)
•
Heart (left anterior
ventricle, right atrial
appendage, coronary
artery and aorta)
•
Greater omentum
•
Mammary tissue
(male and female)
•
Lung
•
Stomach
•
Esophagus (mucosa
and muscularis
propria)
•
Pituitary gland
•
Thyroid gland
•
Minor salivary gland
•
•
Skin (suprapubic
region)
Gastroesophageal
junction
•
Liver
• Spleen
• Pancreas
• Colon (transverse and
sigmoid)
• Lymphoid tissue of
terminal ileum
• Kidney (cortex)
• Adrenal glands
• Female (uterus, ovary
and vagina)
• Male (prostate gland
and testes)
GTEx Biospecimen Source Site
Comparisons
Heart
Line : Y = -0.18295*X + 8.31891
Pearson correlation: r = -0.639804
t-score = -7.675229
Colon
Line : Y = -0.18796*X + 7.89886
Pearson correlation: r = -0.722165
t-score = -10.543999
Cystic Fibrosis
• Explanted lungs recovered from CF patients
undergoing transplant
• NDRI provided ~200 CF lungs for Cystic Fibrosis
Foundation-funded project
• Two new drugs developed
Type 1 Diabetes
• Pancreas, kidney, eyes recovered from long-standing
T1D patients
• Nationwide recoveries using NDRI Private Donor
Program
Amyotrophic Lateral Sclerosis
• Two projects funded by
• ATSDR/McKing Corp – 30 patients targeted for
recovery
• Boston VA – 200 patients registered
• Patients identified and consented by collaborators
• Nationwide recoveries using NDRI Private Donor
Program
o
Collects brain, spinal cord, CSF, bone & skin
o
Recoveries completed < 24 hours post mortem
NDRI Research Services
Biospecimen Application Process
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Application
–
–
–
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Tissue needs/donor profile
IRB documentation
Project synopsis
CV/biosketch
•
Feasibility review – internal/external
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Customized protocol development
Standard Authorization/Consent
• Protocol approved by Univ. of Pennsylvania IRB
• Risk mitigation a priority
• Authorization for general research including
o For profit
o Therapeutics and diagnostics
o Genome sequencing
• Potential access to complete health record
• No payment to donor or family decision maker
Customized Authorization/Consent
• Project specific IRB approval
• Incorporates additional objectives (e.g.
product development)
• Right to withdraw limits
• Addresses risks associated with loss of
identity (genotyping)
• Unknown future uses
• Cell line development (unlimited future use)
Summary
• Project-driven services to scientists
• Nationwide collection centers
• Worldwide distribution capabilities
• Diseased and normal biospecimens
available from multiple donor types
• SOPs and total QMS in place for consent,
recovery, & distribution
Acknowledgements

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