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Establishing a Pediatric CNS Biorepository

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Presentation on theme: "Establishing a Pediatric CNS Biorepository"— Presentation transcript:

1 Establishing a Pediatric CNS Biorepository
Javad Nazarian, PhD, MS Associate Professor, Integrative Systems Biology George Washington University School of Medicine and Health Sciences Principal Investigator, Children's National Medical Center Center for Genetic Medicine Research (CGMR) Center for Cancer and Immunology Research (CCIR) May 25, 2016

2 High-quality, data-rich samples are essential for future research
High-quality, data-rich samples are essential for future research. But obtaining and storing these samples is not as straightforward as many researchers think. 7 JUNE 2012 | VOL 486 | NATURE | 141

3 The Need To Biobank at CNHS Stems From Active Research Areas
Some of the active areas of research: Medulloblastoma DIPG Immunology / Immunotherapy, Clinical Trials Low grade glioma NF, AML, etc.

4 Prerequisites and Necessities
Recognition of challenges Understanding disease biology is hindered by: Lack of biological specimen Tissue type (frozen, matched FFPE, matched blood, etc.) Clinical annotation Molecular annotation Hypothesis testing and translational research is hindered by Lack of robust preclinical models of the disease Clinical annotation

5 Rapid Translational Application of Targeted Molecules
Collaborative & Translational Vision Neurooncology Neurosurgery Neurology Research Specimen Specimen Specimens: Tumor & Non-tumor, CSF, Blood, Urine In Vivo/Vitro Models Rapid preclinical testing Molecular Testing Genome, mRNA, Proteome Rapid Translational Application of Targeted Molecules

6 Rapid Translational Application of Targeted Molecules
Collaborative & Translational Vision at CNHS Neurooncology Neurosurgery Neurology Research Specimen Specimen Specimens: Tumor & Non-tumor, CSF, Blood, Urine In Vivo/Vitro Models Rapid preclinical testing Molecular Testing Genome, mRNA, Proteome Rapid Translational Application of Targeted Molecules

7 Rapid Translational Application of Targeted Molecules
Collaborative & Translational Vision at CNHS Neurooncology Neurosurgery Neurology Research Specimen Specimen Specimens: Tumor & Non-tumor, CSF, Blood, Urine In Vivo/Vitro Models Rapid preclinical testing Molecular Testing Genome, mRNA, Proteome Rapid Translational Application of Targeted Molecules

8 Rapid Translational Application of Targeted Molecules
Collaborative & Translational Vision at CNHS Neurooncology Neurosurgery Neurology Research Specimen Specimen Specimens: Tumor & Non-tumor, CSF, Blood, Urine In Vivo/Vitro Models Preclinical testing Molecular Testing Genome, mRNA, Proteome Rapid Translational Application of Targeted Molecules

9 Collaborative & Translational Vision at CNHS
Neurooncology Neurosurgery Neurology Research Specimen Specimen Specimens: Tumor & Non-tumor, CSF, Blood, Urine In Vivo/Vitro Models Preclinical testing Molecular Testing Genome, mRNA, Proteome Rapid Translational Application of Targeted Molecules: responders vs non-responders, etc

10 Collaborative & Translational Vision at CNHS
National Collaborative Efforts Postmortem Tumor Banking (DIPG) Clinical Specimen (e.g. PNOC-003)

11 Coordinating Postmortem tissue collection
Physician Study Coordinator Hospice Nurse/Social Worker Neuro Pathologist Funeral home Availability Hours of operation Vested in detailed procedure Alternatives : Diener service Regional path Point of contact with the family Update on patient’s health status Funeral home information Paper work Timing Autopsy site

12 Non-CNMC Coordinated Autopsy Cases

13 Outcome: Biorepository
Total Number of Subjects In the Biorepository

14 Biorepository Breakdown: Age and Gender

15 Biorepository Breakdown: Tumor Type
OPG

16 Biorepository Breakdown: Tissue type
Frozen FFPE 20 2.6% 101 13.2% 113 14.4% 24 3.1% 34 4.4% 2 0.3% 474 61.7% CSF, Blood, Urine

17 Prerequisites and Necessities
Recognition of challenges STUDY THE DISEASE Lack of biological specimen Tissue type (frozen, matched FFPE, matched blood, etc.) Clinical annotation Molecular annotation √ TEST HYPOTHESIS  TRANSLATE Lack of robust preclinical models of the disease Clinical annotation

18 Characterization of DIPG primary cells:
DAPI H3K27M Merge Neurosphere DAPI GFAP Olig2 Merge Nestin Sox2 Adherent

19 Models Using Autopsied Specimen
DIPG Xenograft Models Using Autopsied Specimen Murine Pontine tumor Murine Cortical tumor a b c H&E H&E Ki67 MAB1273 Ki67 H3K27me3 H3K27M Kambhampati et. al Sridevi Yadavilli

20 Pre- clinical models of DIPG
ID Tissue Source Mutations (major) Primary Cell Xenograft CNMC 762 Biopsy (PNOC-003) H3F3A K27M/ TP53 A159P/ KDR/ KIT/ PDGFRA Yes SF 10423 H3F3A K27M / PPM1D / TP53 R282W / PIK3R1 K567E N/A CNMC 787 TP53 R110L / ATRX E2279* CNMC 586 Autopsy H3F3A K27M / TP53 / PBRM1 CNMC 739 H3F3A K27M CNMC 675 H3F3A K27M/ PPM1D E525X/ ATRX V1514D

21 Lessons Learned: DIPG Whole Brain Procurement
1 2 7 3 4 6 5

22 Lessons Learned: Detailed processing is a essential!
Kambhampati et al. 2015

23 Complexity of Biobanking

24 FreezerPro database to annotate the biobank

25 Current Challenges Logistics (e. g. pathology, shipping, etc) Funding Awareness (clinicians & families) Collaborative efforts Uniformity of consent form/process The established biobank has resulted in: Collaborations Better understanding of DIPG biology (>12 peer reviewed publications) Robust preclinical resources

26 ACKNOWLEDGEMENTS PATIENTS & THEIR FAMILIES
CHILDREN’S NATIONAL HEALTH SYSTEM Cheng-Ying Ho, Brian Rood, Lindsay Kilburn Eugene Hwang, Suresh Magge, Roger Packer Pacific Pediatric Neuro-Oncology Consortium Sabine Mueller, Michael Prados, CHOP Mariarita Santi, Adam Resnick, Angela Waanders, Michael Fisher Isabella Kerr Molina Foundation Zickler Family Foundation

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