`
`CURRICULUM VITAE
`
`James Lawrence Michael Ferrara
`james.ferrara@mssm.edu
`
`
`APPOINTMENTS/EMPLOYMENT
`
`PREVIOUS EMPLOYMENT
`
`Instructor in Pediatrics, Harvard Medical School
`07/1985 – 06/1987
`Assistant Professor of Pediatrics, Harvard Medical School
`07/1987 – 06/1993
`Associate Professor of Pediatrics, Harvard Medical School
`07/1993 – 10/1998
`Professor of Pediatrics, Professor of Internal Medicine, University of Michigan Medical School
`11/1998 – 06/2014
`Professor of Pediatrics, Professor of Internal Medicine, Icahn School of Medicine at Mount Sinai
`07/2014 –
`
`
`
`CLINICAL/HOSPITAL APPOINTMENTS
`
`07/1980 – 06/1982
`07/1982 – 06/1985
`07/1982 – 06/1985
`07/1985 – 10/1998
`07/1985 – 10/1998
`07/1986 – 10/1998
`07/1998 – 03/2012
`
`Clinical Instructor in Pediatrics, Children’s Hospital
`Fellow in Hematology/Oncology, Children’s Hospital
`Fellow in Pediatric Oncology, Dana-Farber Cancer Institute
`Assistant Physician in Medicine, Hematology/Oncology, Children’s Hospital, BWH
`Assistant Physician, Dana-Farber Cancer Institute
`Assistant Physician, Brigham and Women’s Hospital
`Director, Combined Adult and Pediatric Bone Marrow Transplant Program, University of Michigan
`Cancer Center
`Co-Director, BMT Leukemia/Lymphoma Program, University of Michigan
`Cancer Center
`Ward-Coleman professor of Cancer Medicine, Professor and Director of BMT Translational
`Research Center, Hematologic Malignancies Translational Research Center, Icahn School of
`Medicine at Mount Sinai
`
`07/1999 – 03/2012
`
`
`07/2014 –
`
`
`EDUCATION
`
`06/1970 H.A.B.
`06/1974 M.A.
`05/1976 M.D.
`04/2009 D.Sc.
`
`POSTDOCTORAL TRAINING
`
`07/1980 – 06/1982
`07/1981 – 06/1982
`07/1982 – 06/1985
`
`Xavier University, Honors A. B., Summa Cum Laude
`Oxford University, Literae Humaniores
`Georgetown University, M.D., Cum Laude
`Oxford University, Medical Sciences
`
`Residency in Pediatrics, Children’s Hospital, Boston, MA
`Junior Resident in Pediatrics, Children’s Hospital, Boston, MA
`Fellow in Pediatric Hematology/Oncology, Children’s Hospital and the Dana-Farber Cancer
`Institute, Boston, MA
`
`
`CERTIFICATION
`
`Diplomate, American Board of Pediatrics, No. 61496, October 8, 1997
`Diplomate, American Board of Pediatric Hematology/Oncology No. 1933, November 13, 2006
`
`LICENSURE
`
`Michigan License Registration No. 4301-073406
`New York State License Registration No. 278246-1
`
`
`
`SAN EX 1023, Page 01
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`

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`HONORS/AWARDS
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`1972
`1974
`1976
`1980
`1982
`1988
`1990
`1993
`1997
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`2002
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`2003
`2004
`2005
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`2006
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`2008
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`2009
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`2010
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`2011
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`2012
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`2014
`2014
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`Fredin Memorial Scholarship, La Sorbonne
`Valedictorian, Xavier University
`Presidential Scholar, Georgetown Medical School
`Alpha Omega Alpha
`Von L. Meyer Fellowship, Boston Children’s Hospital
`Perini Foundation Fellow, DFCI
`Claudia Adams Barr Investigator, DFCI
`Scholar Award, Leukemia Society of America
`Member, American Society for Clinical Investigation
`Alexander von Humboldt Research Prize, German Republic
`Who’s Who in Medicine and Health Care Citation
`Who’s Who in Science and Technology Citation
`America’s Top Doctors Citation
`Who’s Who in America Citation
`Member, American Pediatric Society
`Doris Duke Distinguished Clinical Scientist Award
`Outstanding Clinician Award, University of Michigan Medical School
`Member, Association of American Physicians
`Senior Fellow, Michigan Society of Fellows
`Who’s Who in the World Citation
`America’s Top Doctors for Cancer (1st Edition) Citation
`Chairman, Steering Committee, BMT Clinical Trials Network
`America’s Top Pediatricians (Hematology/Oncology) Citation
`Member, Board of Trustees, Hampshire College, Amherst, MA
`IBC Top 100 Health Professionals Citation
`American Cancer Society Clinical Research Professor
`America’s Top Physicians Citation
`Ruth Heyn Endowed Professor of Pediatric Oncology, The University of Michigan
`Doctor of Medicine, honoris causa, University of Regensburg, Germany
`Doctor of Science, Oxford University
`Dr. Lyonel G. Israel’s Visiting Professor, CancerCare Manitoba, Manitoba, Canada
`E. Donnell Thomas Lecturer, ASBMT
`Visiting Fellow, All Soul’s College, Oxford, England
`Nobel Symposium Lecture, Karolinska Institutet, Stockholm, Sweden
`America´s Top Physicians Citation
`Ward-Coleman Chair in Cancer Medicine, Icahn School of Medicine at Mount Sinai
`
`
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`PATENTS
`
`
`2012
`
`Methods for Detecting Graft-Versus-Host Disease: patent pending US Patent Application Serial
`No. 13/573,766; Issued: Dec. 18, 2012
`
`
`OTHER PROFESSIONAL ROLES
`
`EDITORIAL POSITIONS, BOARDS AND PEER-REVIEW SERVICE
`2009
`
`Biology of Blood and Marrow Transplantation, 2009 Education Supplement, Editor, Volume 15,
`
`
`Supplement 1, January 2009, Pages 1-168, Elsevier.
`2011 – 2014 American Society of Hematology (ASH), Publications Committee
`2013 – 2014 BMT CTN, State of the Science Symposium Steering Committee
`2016
`
`Development of a biomarker scoring system for use in graft-versus-host disease, 2016 Biomarkers in
`
`
`Medicine, 2016 Aug 14;10(8):793-5. Epub 2016 Jul 14. PMC Journal - In Process.
`
`SCIENTIFIC ACTIVITIES
`
`
`
`Editorial Boards
`1985 - 1995
`Hematology Reviews and Communications Transplantation
`
`SAN EX 1023, Page 02
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`

`1993 - 1998
`1993 - 1999
`1998 -
`
`Journal of Immunology
`Transplantation Immunology Bone Marrow Transplantation
`Biology of Blood and Marrow Transplantation
`
`OTHER PROFESSIONAL POSITIONS AND MAJOR VISITING APPOINTMENTS
`
`Junior Research Fellow, Kennedy Institute of Ethics, Georgetown University
`Premedical Tutor, Adams House, Harvard University
`Chairman, Premedical Committee, Adams House, Harvard University
`Seminar Moderator, Aspen Institute for Humanistic Studies
`Member, Medical Ethics Division, Harvard Medical School
`Faculty Moderator, Harvard Program in the Practice of Scientific Investigation
`Member, NIH Study Section (Exp. Therapeutics-2)
`Member, Scientific Review Panel, NCI (Exp. Immunology)
`Reviewer, Leukemia and Lymphoma Society, Translational Research Grants
`Scientific Director, American Society for Blood and Marrow Transplantation
`Member, Scientific Committee on Lymphocyte Biology, American Society of Hematology
`Member, National Cancer Institute Study Section – Clinical Studies
`The Bone Marrow Foundation Medical Advisory Board
`Member, Board of Trustees, The Leukemia & Lymphoma Society (Michigan Chapter)
`Chairman, Steering Committee, BMT Clinical Trials Network
`Chairman, American Society for Blood and Marrow Transplantation, Annual Meeting
`Program Committee
`Vice Chair, American Society for Hematology, 2009 Transplantation Biology Committee
`Chair, American Society for Hematology, 2010 Transplantation Biology Committee
`Member, American Society for Hematology Journals Committee
`
`
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`1976 - 1979
`1980 - 1998
`1982 - 1985
`1990 - 1992
`1990 - 1998
`1991 - 1998
`1996 - 2000
`1997 - 1998
`2001 - 2007
`2002 - 2004
`2003 - 2006
`2004 - 2008
`2006 -
`2006 -
`2006 - 2007
`2008
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`2008
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`2009
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`2011 - 2014
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`MEMBERSHIPS IN PROFESSIONAL SOCIETIES
`
`1976
`1982
`1988
`1991
`1999
`1995
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`1997
`2002
`2003
`2007
`2009
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`CCOONNSSUULLTTIINNGG PPOOSSIITTIIOONNSS
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`1992
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`1993
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`1995
`1996 - 2000
`1996
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`1997, 2007
`1997
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`1998
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`1998, 2006
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`Society for Health and Human Values Massachusetts Medical Society
`Boston Blood Club
`Transplantation Society American Association of Immunologists
`American Society of Hematology
`American Association for the Advancement of Science
`Society for Pediatric Research
`American Society of Blood and Marrow Transplantation
`Molecular Medicine Society
`American Society for Clinical Investigation
`American Pediatric Society
`Association of American Physicians
`American Society of Clinical Oncology
`American Association for Cancer Research
`
`Member, Behringwerke Scientific Advisory Board, Marburg, Germany
`Chairman, NIAID Special Review Committee for Bone Marrow Transplant
`External Reviewer, Medical Research Council, Canada
`Member, NIH Study Section (Exp. Therapeutics-2)
`Member, NCI Task Force Report on Transplantation
`Member, Scientific Advisory Board, BMT Program, Duke University
`Member, Scientific Review Panel, NCI (Exp. Immunology)
`Member, Children’s Cancer Foundation Grant Review Panel
`Member, Scientific Advisory Council, Cincinnati Children’s Research Foundation
`
`1999
`2000
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`2001
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`Member, Swiss National Science Foundation Review Panel
`Member, Amgen Oncology Research Council
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`Member, External Advisory Board, Comprehensive Cancer Center of Case Western
`Reserve University
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`SAN EX 1023, Page 03
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`

`

`Member, Centocor Oncology Advisory Board
`Reviewer, Leukemia and Lymphoma Society, Translational Research Grants
`Member, Central Society for Clinical Research
`Member, Scientific Advisory Board, BMT Program of Case Western Reserve University
`Member, Scientific Advisory Board, Human Genome Sciences
`Member, Special Emphasis Panels, NCI, NIAID
`Scientific Director, American Society for Blood and Marrow Transplantation
`Member, NCI Initial Review Group, Subcommittee D – Clinical Studies
`Member, External Advisory Board, Therakos, Inc.
`Chairman, Steering Committee, BMT Clinical Trials Network
`Member, Special Emphasis Panel, NIH Loan Repayment Program
`
`
`
`
`2001
`2001 - 2007
`2001
`
`2001
`
`2001
`
`2001
`
`2002 - 2004
`2004 - 2008
`2005
`
`2006
`
`2007
`
`
`RREESSEEAARRCCHH PPRROOFFIILLEE
`
`My research focuses on the prevention, diagnosis and treatment of graft versus host disease (GVHD), the major
`complication of allogeneic bone marrow transplantation (BMT). My laboratory was the first to demonstrate that
`inflammatory cytokines produced by donor T cells and monocytes were critical effectors of acute GVHD in animal models,
`and coined the term “cytokine storm” to describe this process. My laboratory has also discovered and validated plasma
`biomarkers of acute GVHD that we have used to create an algorithm that accurately predicts at the time of initial
`diagnosis the eventual severity of GVHD. We are now exploring the biology of the biomarkers in animal models, which
`focuses on the interactions between the adaptive and innate immune systems, especially in the gastrointestinal tract,
`which is the GVHD target organ that is most resistant to treatment. Our goal is to translate insights from these animal
`models into innovative clinical trials.
`
`CCLLIINNIICCAALL PPRROOFFIILLEE
`
`My clinical practice focuses exclusively on patients who undergo BMT. Most recipients of allogeneic BMT are at risk for
`GVHD, the primary focus of my research. My laboratory efforts are thus essentially translational in nature, and my goal is
`to use insights gained from laboratory studies and animal models in the design and execution of innovative clinical trials
`to prevent and treat GVHD. As a clinical researcher, I have been the principal investigator or co-investigator on multiple
`trials for BMT patients. In this clinical research capacity I have served as the chairman of the steering committee of the
`BMT clinical trials network, the NIH cooperative group focused on the development of high impact clinical BMT trials that
`have the potential to change practice. To accelerate the translational aspects of my research, I have formed an
`international consortium of ten centers to provide both clinical data and samples and to conduct clinical trials in acute
`GVHD. This consortium will be the first to treat GVHD using the biomarker grading system as an inclusion criterion.
`
`IIMMPPAACCTT
`
`My long term goal is to make BMT a safer and more effective therapy for patients with life threatening disorders of the
`blood. My laboratory’s research in animal models has fundamentally changed the way we think about the
`pathophysiology of GVHD, which the schema for we proposed twenty years ago. In particular, we were the first to
`elucidate the role of inflammatory cytokines in this process. In the past ten years, my laboratory first discovered and
`then validated multiple plasma biomarkers of acute GVHD. We have now used these biomarkers to predict the maximal
`severity of GVHD and its eventual response to treatment. The impact of this new approach is likely to be global, because
`we have validated its accuracy in hundreds of patients from dozens of BMT centers across the globe. During the next
`decade these advances will give rise to a new generation of clinical trials led by researchers from Mt. Sinai that will test
`investigative agents for the prevention and treatment of acute GVHD. Our goal is to reduce the mortality from GVHD by
`50% in the next ten years.
`
`GGRRAANNTTSS,, CCOONNTTRRAACCTTSS,, FFOOUUNNDDAATTIIOONN SSUUPPPPOORRTT
`
`PPAASSTT GGRRAANNTTSS
`LLiisstt FFuunnddiinngg SSoouurrccee,, PPrroojjeecctt TTiittllee && NNuummbbeerr
`
`
`RRoollee iinn PPrroojjeecctt
`
`
`
` DDaatteess
`
`
`
`National Institutes of Health Physician
`
`
`PPII
`
`
`1985-1990
`
`
`DDiirreecctt CCoossttss
`
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`NN//AA
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`SSuupppplleemmeenntt
`aall IInnffoo
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`NN//AA
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`SAN EX 1023, Page 04
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`

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`Scientist Award, “Murine Models of GVHD”
`Hood Foundation Research Grant, “Role of T
`cells in Experimental GVHD
`”
`
`American Cancer Society Research Grant,
`“GVHD to Minor H Antigen”
`
`NIAID P01, Induction of Host-Specific
`Tolerance
`
`NIAID R01, “Effector Mechanisms of GVHD”
`
`
`NCI R01, “Novel Approaches to CML”
`
`
`Scholar Award, Leukemia Society of America
`
`
`LSA Translational Research Award, “IL-4
`Protocol to Prevent GVHD”
`
`FDA, FDR-1457, “IL-1ra for the Prevention
`of Acute GVHD”
`
`FD-R-002021, FDA, Orphan Product Drug
`Grant, “A Phase I/II Trial of Keratinocyte
`Growth Factor (rHuKGF) to Prevent Acute
`GVHD in 6/6 HLA=BMT Recipients.”
`
`R01 HL55162, NIH, “Idiopathic Pneumonia
`Syndrome after BMT”
`
`FD-R-002146, FDA, “Orphan Product Drug
`Grant”
`
`FD-R-002397, FDA, “Orphan Drug Grant”
`
`Leukemia & Lymphoma Society Focused
`Group Workshop ”Blood and Marrow
`Transplant State of the Science Symposium”
`
`Distinguished Clinical Scientist Award Doris
`Duke Charitable Foundation
`Novel Strategies to Improve Allogeneic Bone
`Marrow Transplant
`
`Therakos, Inc., Basic Science Research
`Grant, “Characterization of the In Vivo
`Effects of Extracorporeal Photopheresis on
`T-cell and Antigen Presenting Cell Function
`in Murine Acute Graft-Versus-Host Disease”
`
`R13 HL088882, NIH/NHLBI, “BMT State of
`the Science Symposium”
`
`FD-R-002397, FDA/Orphan Products
`Development, Treatment of Graft-versus-
`Host Disease Using Enbrel
`
`
`PPII
`
`
`PPII
`
`
`PI (Project 1)
`
`
`PPII
`
`
`CCoo--PPII
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`Co-PI
`
`
`
`1987-1988
`
`
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`1991-1992
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`
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`1993-1995
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`
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`1992-1996
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`1993-1997
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`1993-1998
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`1997-1998
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`1997-2000
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`
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`2001-2004
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`1999-2004
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`2002-2005
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`2003-2006
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`2006-2007
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`2002-2007
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`2005-2007
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`2007-2008
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`2003-2008
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`

`

`
`U01 HL069330, NIH/NHLBI, University of
`Michigan Core Clinical Center for the BMT
`Research Network
`
`3P30 CA046592, NIH/NCI, Cancer Center
`Core Grant
`
`Hartwell Foundation Biomedical Research
`Award, Bioenergetic Strategy to Treat GVHD
`
`5T32HL007622-24, NIH/NHLBI, Training
`Program in Molecular Hematology
`
`RC1HL101102-02, NIH/NHLBI, Graft versus
`Host Disease Biomarkers: Prediction of
`Onset and Response to Therapy
`
`Leukemia and Lymphoma Society
`Translational Research Program, Mechanism
`of Action of Extracorporeal Photopheresis in
`Treating Acute GVHD
`
`U-M FASTTRACK Bridging Grant: Plasma
`Biomarkers of Acute Graft Versus Host
`Disease
`
`5R34HL105776-02 Phase II Study of a Novel
`GVHD Prevention Strategy: Etanercept and
`Photopheresis
`
`5T32HL007622-27, NIH/NHLBI, Training
`Grant in Molecular and Translational
`Hematology
`
`Hyundai Hope on Wheels Grant, Hyundai
`Motor America, Serum Biomarkers for the
`Prediction of Graft-Versus-Host Disease in
`Children
`
`Hyundai Hope on Wheels Grant, Hyundai
`Motor America, Metabolic Regulation in T
`Cells During Graft-versus-host Disease
`
`5U10HL069330-13, NIH/NHLBI, University
`of Michigan Core Clinical Center for BMT
`Research Network
`
`1R21CA173459-01, NIH/NCI, Plasma
`Biomarkers of Acute Graft Versus Host
`Disease
`CRP-13306-COUN, American Cancer Society,
`Novel Strategies to Treat Graft versus Host
`Disease
`
`National Marrow Donor Program, Ann Arbor
`aGVHD Rish Stratification Biomarker Panel
`Testing
`
`
`PI
`
`
`Program Co-
`Director
`
`PI
`
`
`PI
`
`
`Co-
`Investigator
`
`
`
`PI
`
`
`PI
`
`
`Co-
`Investigator
`
`
`PPII
`
`
`2006-2011
`
`
`
`2006-2012
`
`
`
`2009-2012
`
`
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`2007-2012
`
`
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`2009-2012
`
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`2008-2012
`
`
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`2012-2013
`
`
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`NN//AA
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`2011-2014
`
`
`NN//AA
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`
`07/01/86 -
`06/30/14
`
`
`Co-
`Investigator
`
`
`09/01/12 –
`06/30/14
`
`
`Co-
`Investigator
`
`
`09/01/13 –
`06/30/14
`
`
`SSiittee PPII
`
`
`PI
`
`PI
`
`PI
`
`
`09/30/01 –
`12/31/15
`
`
`08/01/13 –
`07/31/16
`
`07/01/13-
`06/30/18
`
`03/01/16-
`2/28/18
`
`
`NN//AA
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`N/A
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`N/A
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`N/A
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`N/A
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`SAN EX 1023, Page 06
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`

`

`NIH/NCI, 5P30CA196521-03
`The Tisch Cancer Institute – Cancer
`Institute Grant
`
`CCoo--PPII
`
`08/01/15-
`07/31/20
`
`
`
`Total award:
`$6,701,740
`
`Current year
`direct:
`$1,353,548
`
`
`SAN EX 1023, Page 07
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`

`

`CCUURRRREENNTT GGRRAANNTTSS
`
`LLiisstt FFuunnddiinngg SSoouurrccee,, PPrroojjeecctt TTiittllee && NNuummbbeerr
`
`RRoollee iinn PPrroojjeecctt
`
`
`
` DDaatteess
`
`
`DDiirreecctt CCoossttss
`
`
`SSuupppplleemmeennttaall
`IInnffoo
`
`Doris Duke Foundation, 2017048,
`Early Plasma Biomarkers to Predict Severe
`GVHD in URM Patients
`
`PPII
`
`7/01/17 –
`12/31/18
`
`Pediatric BMT Consortium
`Multicenter Pediatric GVHD Biomarkers for
`High Risk Treatment and Pre-Emption
`
`
`CCoo--PPII
`
`02/01/16-
`01/31/19
`
`Kamada
`A Proof of Concept Pilot Trial of Alpha-1-
`Antitrysin for Pre-emption of Steroid-
`Refractory Acute GVHD
`
`Co-PI
`
`01/02/18-
`01/02/20
`
`NIH/NCI, 5P01CA039542-30, Cellular and
`Molecular Studies in Bone Marrow
`Transplant
`
`PI
`
`09/06/16-
`08/31/21
`
`1UG1HL138645-01, NIH/NHLBI
`Mount Sinai Core Clinical Consortium for
`the BMT Clinical Trials Network
`
`
`Co-PI
`
`07/28/17-
`06/30/24
`
`
`
`
`
`
`
`
`
`
`
`
`Total award:
`$64,000
`
`Current year
`direct:
`$64,000
`Total award:
`$263,915
`
`
`Current year
`direct:
`$87,972
`
`Total award:
`$540,000
`
`Current year
`direct:
`$540,000
`Total Award:
`$6,771,198
`
`Current year
`direct:
`$1,483,358
`Total award:
`$828,004
`
`Current year
`direct:
`$114,002
`
`
`
`PPEENNDDIINNGG GGRRAANNTTSS
`
`LLiisstt FFuunnddiinngg SSoouurrccee,, PPrroojjeecctt TTiittllee && NNuummbbeerr
`
`
`RRoollee iinn PPrroojjeecctt
`
`
`
`DDaatteess
`
`
`DDiirreecctt CCoossttss
`
`
`SSuupppplleemmeennttaall
`IInnffoo
`
`
`
`Role in Project
`
`PI
`
`N/A
`
`
`CCLLIINNIICCAALL TTRRIIAALLSS PPAARRTTIICCIIPPAATTIIOONN
`
`Project
`
`CTN 102: A Trial of Tandem Autologous
`Stem Cell Transplants +/- Post Second
`Autologous Transplant Maintenance
`Therapy versus Single Autologous Stem Cell
`Transplant Followed by Matched Sibling
`Non-myeloablative Allogeneic Stem Cell
`Transplant for Patients with Multiple
`Myeloma
`
`
`
`
`
`
`
`
`
`
`
`
`Dates
`
`12/2003-
`03/3013
`
`Award
`
`NN//AA
`
`Other Info
`
`NN//AA
`
`
`
`
`
`
`
`SAN EX 1023, Page 08
`
`

`

`CTN201: A Phase III Randomized
`Multicenter Trial Comparing G-CSF Mobilized
`Peripheral Blood Stem Cell with Marrow
`Transplantation from HLA Compatible
`Unrelated Donors
`
`CTN301: Fludarabine-based Conditioning for
`Allogeneic Marrow Transplantation from
`HLA-compatible Unrelated Donors in Severe
`Aplastic Anemia
`
`CTN302: Initial Systemic Treatment of
`Acute GVHD: A Phase II Randomized Trial
`Evaluating Etanercept, Mycophenolate
`Mofetil (MMF), Denileukin Diftitox (Ontak),
`and Pentostatin in Combination with
`Corticosteroids
`
`CTN401: Phase III Rituxan/BEAM vs.
`Bexxar/BEAM with Autologous
`Hematopoietic Stem Cell Transplantation
`(ASCT) for Persistent or Relapsed
`Chemotherapy Sensitive Diffuse Large B-
`Cell Non-Hodgkin’s Lymphoma
`
`CTN402: A Phase III Randomized,
`Multicenter Trial Comparing
`Sirolimus/Tacrolimus with
`Tacrolimus/Methotrexate as GVHD
`Prophylaxis After HLA-Matched, Related
`Peripheral Blood Stem Cell Transplantation
`
`CTN403: A Phase III, Randomized Double-
`Blind, Placebo Controlled Trial of Soluble
`Tumor Necrosis Factor Receptor: Enbrel
`(Etanercept) for the Treatment of Acute
`Non-Infectious Pulmonary Dysfunction
`(Idiopathic Pneumonia Syndrome) Following
`Allogeneic Stem Cell Transplantation
`
`CTN501: Multi-center, Open Label,
`Randomized Trial Comparing Single Versus
`Double Umbilical Cord Blood (UCB)
`Transplantation in Pediatric Patients with
`Leukemia and Myelodysplasia
`
`CTN601: Unrelated Donor Reduced
`Intensity Bone Marrow Transplant for
`Children with Severe Sickle Cell Disease /
`The SCURT Study
`
`CTN702: A Trial of Single Autologous
`Transplant with or without Consolidation
`Therapy versus Tandem Autologous
`Transplant with Lenalidomide Maintenance
`for Patients with Multiple Myeloma
`
`CTN801: A Phase II/III Randomized,
`Multicenter Trial Comparing Sirolimus plus
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`01/2004-
`04/2014
`
`
`01/2006-
`12/2012
`
`
`09/2005-
`06/2012
`
`
`12/2005-
`08/2013
`
`
`11/2006-
`10/2011
`
`
`08/2007-
`07/2013
`
`
`12/2006-
`05/2013
`
`
`08/2008-
`06/2014
`
`
`05/2010-
`06/2014
`
`
`04/2010-
`06/2014
`
`NN//AA
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`N/A
`
`
`NN//AA
`
`
`NN//AA
`
`SAN EX 1023, Page 09
`
`

`

`Prednisone, and Sirolimus/Calcineurin
`Inhibitor plus Prednisone for the Treatment
`of Chronic Graft-versus-Host Disease
`
`CTN802: A Multi-Center, Randomized,
`Double Blind, Phase III Trial Evaluating
`Corticosteroids with Mycophenolate Mofetil
`vs. Corticosteroids with Placebo as Initial
`Systemic Treatment of Acute GVHD
`
`CTN901: A Randomized, Multi-Center,
`Phase III Study of Allogeneic Stem Cell
`Transplantation Comparing Regimen
`Intensity in Patients with Myelodysplastic
`Syndrome or Acute Myeloid Leukemia
`
`CTN902: A Phase III Randomized,
`Multicenter Trial Testing Whether Exercise
`or Stress Management Improves Functional
`Status and Symptoms of Autologous and
`Allogeneic Recipients.
`
`CTN1101: A Multi-Center, Phase III,
`Randomized Trial of Reduced Intensity(RIC)
`Conditioning and Transplantation of Double
`Unrelated Umbilical Cord Blood (dUCB)
`versus HLA-Haploidentical Related Bone
`Marrow (Haplo) for Patients with
`Hematologic Malignancies
`
`CTN1202: Prospective Multi-Center Cohort
`for the Evaluation of Biomarkers Predicting
`Risk of Complications and Mortality
`Following Allogeneic HCT
`
`CTN1204: Reduced-Intensity Conditioning
`for Children and Adults with
`
`Hemophagocytic Syndromes or Selected
`Primary Immune Deficiencies.
`
`
`Phase 1 Study of Elotuzumab in
`Combination with Autologous Stem Cell
`Transplantation and Lenalidomide
`Maintenance for Multiple Myeloma
`
`
`BMT CTN 1401: Phase II Multicenter Trial of
`Single Autologous Hematopoietic Cell
`Transplant Followed by Lenalidomide
`Maintenance for Multiple Myeloma with or
`without Vaccination with Dendritic Cell
`/Myeloma Fusions
`
`
`Prospective Multi-Center Cohort for the
`Evaluation of Biomarkers Predicting Risk of
`Complications and Mortality Following
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`PI
`
`
`01/2010-
`06/2013
`
`
`05/2011-
`06/2014
`
`
`01/2011-
`06/2014
`
`
`06/2012-
`06/2014
`
`
`06/2013-
`Current
`
`
`12/2013-
`06/2014
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`Co-Inv.
`
`06/09/15-
`08/17/17
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`NN//AA
`
`
`Co-Inv.
`
`
`08/04/16-
`03/31/21
`
`
`N/A
`
`
`N/A
`
`
`Co-Inv.
`
`
`01/01/16 –
`08/01/18
`
`
`NN//AA
`
`
`NN//AA
`
`SAN EX 1023, Page 10
`
`

`

`Allogeneic HCT (BMT CTN 1202)
`
`
`
`
`BMT CTN 1202: Prospective Multi-Center
`Cohort for the Evaluation of Biomarkers
`Predicting Risk of Complications and
`Mortality Following Allogeneic HCT
`
`
`NMDP, 211907, Ann Arbor aGVHD Risk
`Stratification Biomarker Panel Testing
`
`
`
`Alexion Pharmaceuticals, Inc., GVHD
`biomarker analysis of samples from their
`clinical trial
`
`
`
`
`Co-Inv.
`
`
`
`
`01/01/16-
`03/17/17
`
`
`
`
`N/A
`
`
`
`
`NN//AA
`
`
`PI
`
`
`PI
`
`
`02/01/16 –
`01/31/18
`
`
`10/26/15 –
`07/01/17
`
`
`
`
`
`
`
`Total Award:
`$155,908
`
`Current year
`direct
`$45,991
`
`Total Award:
`$115,000
`
`Current Year
`direct
`$67,847
`
`
`
`TTRRAAIINNEEEESS
`
`NNaammee
`
`
`LLeevveell ooff TTrraaiinneeee
`
`
`MMDD,,PPoossttddooccttoorraall
`
`PPhhDD,,PPoossttddooccttoorraall
`
`MMDD,,PPoossttddooccttoorraall
`
`Pim van Dijken
`
`Tariq Ghayur
`
`Hirohiko
`Sakamoto
`
`Sunil Abhyankar
`
`David Franklin
`
`Giorgio Falzarano MMDD,,PPoossttddooccttoorraall
`
`MMDD,,PPoossttddooccttoorraall
`
`PPhhDD,,PPoossttddooccttoorraall
`
`Werner Krenger
`
`Anastasia
`Skandalis
`Lu Ying Pan
`
`PPhhDD,,PPoossttddooccttoorraall
`
`MMDD,,PPoossttddooccttoorraall
`
`PPhhDD,,PPoossttddooccttoorraall
`
`Vijay Reddy
`
`Ray Simmons
`
`Eric Nisbet-Brown MMDD,, PPhhDD,,
`PPoossttddooccttoorraall
`MMDD,, PPhhDD,,
`PPoossttddooccttoorraall
`DDDDSS,,PPoossttddooccttoorraall
`
`MMDD,,PPoossttddooccttoorraall
`
`Kenneth Cooke
`
` RRoollee iinn TTrraaiinniinngg
`
`
`&&
`IInncclluussiivvee DDaatteess ooff TTrraaiinniinngg
`
`FFeellllooww AAddvviissoorr
`11998877--11998899
`FFeellllooww AAddvviissoorr
`11998888--11998899
`FFeellllooww AAddvviissoorr
`11998888--11999900
`
`FFeellllooww AAddvviissoorr
`11999900--11999922
`FFeellllooww AAddvviissoorr
`11999922--11999933
`FFeellllooww AAddvviissoorr
`11999922--11999944
`FFeellllooww AAddvviissoorr
`11999933--11999966
`FFeellllooww AAddvviissoorr
`11999977--11999988
`FFeellllooww AAddvviissoorr
`11999944--11999988
`FFeellllooww AAddvviissoorr
`11999977--11999988
`FFeellllooww AAddvviissoorr
`11999977--11999988
`FFeellllooww AAddvviissoorr
`11999977--11999988
`FFeellllooww AAddvviissoorr
`
`TTrraaiinniinngg
`VVeennuuee
`
`
`
`TTrraaiinneeeess’’ CCuurrrreenntt
`SSttaattuuss//EEmmppllooyymmeenntt
`
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`Assoc. Prof. of Pediatrics,
`Univ. of Utrecht
`Staff Scientist, BASF
`
`Assoc. Prof. of Surgery,
`Univ. of Tokyo
`
`Asst. Prof. of Medicine,
`Univ. of South Carolina
`Not available
`
`Asst. Prof. Hematology,
`Univ. of Naples
`PPrrooffeessssoorr ooff IImmmmuunnoollooggyy,,
`BBaasseell UUnniivveerrssiittyy
`Not available
`
`Staff Scientist, Bio-
`transplant
`DDeecceeaasseedd
`
`Assoc. Prof. of Medicine,
`Univ. of South Florida
`NNoott AAvvaaiillaabbllee
`
`LLaabboorraattoorryy
`
`PPrrooffeessssoorr ooff PPeeddiiaattrriiccss,,
`
`SAN EX 1023, Page 11
`
`

`

`Geoffrey Hill
`
`MMDD,,PPoossttddooccttoorraall
`
`Armin Gerbitz
`
`MMDD,,PPoossttddooccttoorraall
`
`Takanori Teshima MMDD,, PPhhDD,,
`PPoossttddooccttoorraall
`MMDD,,PPoossttddooccttoorraall
`
`Eric Carson
`
`Rainer Ordemann MMDD,, PPhhDD,,
`PPoossttddooccttoorraall
`
`Tomatsu Ichiba
`
`MMDD,,PPoossttddooccttoorraall
`
`Ulrich Duffner
`
`MMDD,,PPoossttddooccttoorraall
`
`Pavan Reddy
`
`
`MMDD,,PPoossttddooccttoorraall
`
`Gerhard
`Hildebrandt
`
`Jeffrey Auletta
`
`MMDD,,PPoossttddooccttoorraall
`
`MMDD,,PPoossttddooccttoorraall
`
`Yoshinobu Maeda MMDD,,PPoossttddooccttoorraall
`
`Chang-Ki Min
`
`MMDD,,PPoossttddooccttoorraall
`
`Carrie Kitko
`
`MMDD,,PPoossttddooccttoorraall
`
`11999944--11999999
`FFeellllooww AAddvviissoorr
`11999966--22000000
`
`FFeellllooww AAddvviissoorr
`11999977--11999999
`
`FFeellllooww AAddvviissoorr
`11999977--22000022
`FFeellllooww AAddvviissoorr
`11999999--22000000
`FFeellllooww AAddvviissoorr
`11999999--22000022
`
`FFeellllooww AAddvviissoorr
`22000000--22000022
`FFeellllooww AAddvviissoorr
`22000000--22000033
`
`FFeellllooww AAddvviissoorr
`22000000--22000033
`
`FFeellllooww AAddvviissoorr
`22000011--22000033
`
`FFeellllooww AAddvviissoorr
`22000011--22000044
`FFeellllooww AAddvviissoorr
`22000011--22000044
`FFeellllooww AAddvviissoorr
`22000022--22000033
`
`FFeellllooww AAddvviissoorr
`22000044--22000066
`
`Raimon Durán-
`Struück
`
`
`DDVVMM,, PPhhDD,,
`PPoossttddooccttoorraall
`
`FFeellllooww AAddvviissoorr
`22000044--22000077
`
`Adam Hartigan
`
`GGrraadduuaattee
`SSttuuddeenntt,,
`PPrree--ddooccttoorraall
`
`Oleg I Krijanovski MMDD,, PPhhDD,,
`PPoossttddooccttoorraall
`MMDD,,PPoossttddooccttoorraall
`
`AAddvviissoorr
`22000044--22000077
`
`FFeellllooww AAddvviissoorr
`22000044--22000088
`FFeellllooww AAddvviissoorr
`22000077--22000088
`
`
`LLaabboorraattoorryy
`
`
`LLaabboorraattoorryy
`
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`LLaabboorraattoorryy
`
`JJoohhnnss HHooppkkiinnss UUnniivveerrssiittyy
`DDiirreeccttoorr ooff BBMMTT,,
`QQuueeeennssllaanndd MMeeddiiccaall
`IInnssttiittuuttee
`AAssssoocciiaattee PPrrooffeessssoorr ooff
`MMeeddiicciinnee,, UUnniivveerrssiittyy ooff
`EErrllaannggeenn
`PPrrooffeessssoorr ooff HHeemmaattoollooggyy,,
`SSaappppoorroo UUnniivveerrssiittyy
`NNoott AAvvaaiillaabbllee
`
`Associate Professor,
`University Hospital
`Dresden Department of
`Medicine, GE
`NNoott AAvvaaiillaabbllee
`
`PPeeddiiaattrriicc BBMMTT wwiitthh
`SSppeeccttrruumm HHeeaalltthh,, GGrraanndd
`RRaappiiddss,, MMII
`Professor of Translational
`Oncology and Internal
`Medicine, Associate
`Division Chief, Hem/Onc,
`Co-Director, Hematological
`Malignancies and BMT,
`UMHS
`AAssssoocciiaattee PPrrooffeessssoorr,,
`UUnniivveerrssiittyy ooff UUttaahh SScchhooooll
`ooff MMeeddiicciinnee
`AAssssoocciiaattee PPrrooffeessssoorr,, OOhhiioo
`SSttaattee UUnniivveerrssiittyy
`AAssssoocciiaattee PPrrooffeessssoorr,,
`OOkkaayyaammaa UUnniivveerrssiittyy
`PPrrooffeessssoorr,, TThhee CCaatthhoolliicc
`UUnniivveerrssiittyy ooff KKoorreeaa CCoolllleeggee
`ooff MMeeddiicciinnee
`Assistant Professor,
`Pediatric Hematology &
`Oncology, UMHS
`
`Assistant Professor in
`Surgical Sciences and
`Head of Pre-Clinical
`Transplantation
`Laboratories, Columbia
`University
`
`PPoossttddooccttoorraall ffeelllloowwsshhiipp,,
`BBiioommeeddiiccaall IInnffoorrmmaattiiccss,,
`HHoowwaarrdd UUnniivveerrssiittyy
`
`SSuutttteerr HHeeaalltthh,, BBeerrkklleeyy,, CCAA
`
`AAssssiissttaanntt PPrrooffeessssoorr ooff
`MMeeddiicciinnee,, UUnniivveerrssiittyy ooff
`WWaasshhiinnggttoonn,, SSeeaattttllee
`Staff Scientist,
`
`Christopher
`Blosser
`
`Erin Gatza
`
`MMDD,, PPhhDD,,
`
`FFeellllooww AAddvviissoorr
`
`LLaabboorraattoorryy
`
`SAN EX 1023, Page 12
`
`

`

`PPoossttddooccttoorraall
`
`Janowicz
`
`Sophie Paczesny MMDD,, PPhhDD,,
`PPoossttddooccttoorraall
`
`22000044--22000099
`
`FFeellllooww AAddvviissoorr
`22000066--22000099
`
`LLaabboorraattoorryy
`
`Daniel Wahl
`
`John Magenau
`
`GGrraadduuaattee
`SSttuuddeenntt,,
`PPrreeddooccttoorraall
`MMDD,,PPoossttddooccttoorraall
`
`AAddvviissoorr
`22000077--22001100
`
`FFeellllooww AAddvviissoorr
`22000088--22001100
`
`Mark Vander Lugt MMDD,,PPoossttddooccttoorraall
`
`Andrew Harris
`
`MMDD,,PPoossttddooc