CORPORATE COUNCIL MEETING
Hyatt Carmel Highlands
Carmel, CA
September 23 & 24, 2012
Table of Contents
Page
Agenda
3
List of Participants
5
ASBMT Research Priorities
8
Anti-Trust Guidelines
12
2
ASBMT Corporate Council Agenda
Hyatt Carmel Highlands
Carmel, California
Sunday, September 23
6:00 pm
Welcome Reception
Fireside North
7:00 pm
Dinner
Yankee Point
"Bone Marrow or Peripheral Blood:
When is the Evidence Enough?”
Moderator:
Armand Keating, MD
University of Toronto
Dennis Confer, MD – (BM)
NMDP
Corey Cutler, MD – (PB)
Dan-Farber Cancer Institute
9:00 pm
Adjourn
Monday, September 24
8:00 am
Continental Breakfast
8:30 am
Welcome and Introductions
8:45 am
Scientific Session

Armand Keating, MD
University of Toronto
“Tissue Engineered Lung Technology”
9:30 am
Break
10:00 am
Critical Patient Health Issues
12:00 pm
Surf Room
David Hoganson, MD
Washington University, St. Louis

“Comorbidities and Quality of Life”
Mohamed Sorror, MD, MSc
Fred Hutchinson Cancer Center

“Transplants in Older Patients”
Keith Sullivan, MD
Duke University Medical Center

“Hematopoietic Cell Transplantation in 2020:
A System Capacity Initiative”
Dennis Confer, MD
NMDP
Lunch
3
ASBMT Corporate Council Agenda (cont’d)
Monday, September 24
1:00 pm
Industry Issues Impacting Reimbursement
“Should ASBMT Develop a BMT Board Certification”
Linda Burns, MD
University of Minnesota
Jennifer Malin, MD, PhD
WellPoint
“Challenges of Outcomes Reporting and Analysis”
2:30 pm
Daniel Weisdorf, MD
University of Minnesota
Major Challenges Facing the Pharmaceutical Industry
“Getting Cellular Therapies into the Clinical Routine:
not Your Average FDA Approval.”
Kai Pinkernell, MD
Miltenyi Biotec Inc
“Competition from Generic Drugs, Regulatory Pressures
and Weak Growth in the U.S. Market.”
Panel Discussion
Moderator:
Richard Kadota, MD
Genzyme/Sanofi
3:45 pm
Other Business
Armand Keating, MD
University of Toronto
4:00 pm
Adjourn
4
Council Participants
Corporate Leaders
Amgen, Inc.
John Jarrett, Manager, Oncology Extramural Research, One Amgen Center Drive, Thousand Oaks, CA
91320.
Phone: (805) 447-1000
e-mail: jjarrett@amgen.com
Bristol-Myers Squibb Company
Stephen Van Komen, MD, Oncology MSL – West US Region, Bristol-Myers Squibb.
Phone: 916-941-2326
e-mail: Stephen.vankomen@bms.com
Cell: 916-934-8649
Celgene Corporation
Marina Rajenova, Regional Medical Liaison, 86 Morris Avenue, Summit, NJ 07901.
Phone: 310-339-5186
e-mail: mriajenova@celgene.com
Fresenius Biotech
J. Frank Glavin, Vice President, Head of North America, 920 Winter Street, Waltham, MA 02451.
Phone: (781) 699-4614
e-mail: Frank.Glavin@fresenius-biotech.com
Genzyme/Sanofi
Richard Kadota, MD, Medical Director, Oncology Transplant, Global Medical Affairs, 55 Cambridge
Parkway, Cambridge, MA 02142.
Phone: (617) 761-8509
e-mail: richard.kadota@genzyme.com
Otsuka America Pharmaceutical
Raul Perez-Olle, MD, PhD, Medical Director, 1 University Square, Suite 500, Princeton, NJ 08540.
Phone: (609) 853-2064.
e-mail: Raul.Perez-Olle@otsuka-us.com
Sharon Roell, Associate Director, Global Clinical Development, 1 University Square, Suite 500
Princeton, NJ 08540.
Phone: (612) 710-4888
e-mail: sharon.roell@otsuka.com
Millennium Pharmaceuticals, Inc.
Jim Holmes, RPh, Sr. Manager, Scientific Alliances & Research, Global Medical Affairs, 40 Landsdowne
St., Cambridge, MA 02139.
Phone: (617) 444-2196
e-mail: jim.holmes@mpi.com
Miltenyi Biotec Inc.
Tara Clark, MD, General Manager, N.A. Clinical Operations, 85 Hamilton Street, Cambridge, MA 02139.
Phone: (617) 218-0061
e-mail: tara.clark@miltenyibiotec.com
Kai Pinkernell, Dr. Med., Global Head of Clinical Business, Friedrich-Ebert-StraBe 68, 51429 Bergisch
Gladbach, Germany.
Phone: +49 2204 8306-6563
e-mail: kai.pinkernell@miltenyibiotec.de
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Guest Speakers
Dennis Confer, MD, Chief Medical Officer, National Marrow Donor Program (NMDP), 3001 Broadway Street
Northeast, Minneapolis, MN 55413.
Phone: (612) 627-5800
e-mail: dconfer@nmdp.org
David Hoganson, MD, Cardiothoracic Surgery Fellow, Washington University, 6151 McPherson Ave.
St. Louis, MO 63112.
Phone: (314) 610-6063
e-mail: hogansondm@wudosis.wustl.edu
Jennifer Malin, MD, PhD, Medical Director Oncology, WellPoint, 21555 Oxnard Street, Woodland Hills,
CA 91367-4943
Phone: (310) 486-7792
e-mail: jennifer.malin@wellpoint.com
Mohamed Sorror, MD, Assistant Professor of Medicine, University of Washington, Fred Hutchinson
Cancer Research Center, 1100 Fairview Avenue North, Seattle, WA 98109.
Phone: (206) 667-2765
e-mail: msorror@fhcrc.org
ASBMT Leaders
Linda Burns, MD, ASBMT Director, Fellowship Director of the Hematology, Oncology and
Transplantation (HOT) Division, and Medical Director of the Inpatient Adult Bone Marrow Transplant Unit
at Fairview-University Medical Center, 420 Delaware Street SE, Minneapolis, MN 55455.
Phone: (612) 624-8144
e-mail: burns019@umn.edu
Corey Cutler, MD, PPH, ASBMT Director, Assistant Professor of Medicine, Harvard Medical School,
Dana-Farber Cancer Institute, 44 Binney Street, D1B13, Boston, MA 02115.
Phone: (617) 632-5946
e-mail: corey_cutler@dfci.harvard.edu
Marcos de Lima, MD, ASBMT Treasurer, Director, Bone & Marrow Transplant Program, University
Hospitals Case Medical Center, 11100 Euclid Avenue LKS5079, Cleveland, Ohio 44106.
Phone: 216-286-6869
e-mail: Marcos.deLima@UHhospitals.org
Armand Keating, MD, ASBMT Past-President, Director, Division of Hematology, University of Toronto,
Director, Cell Therapy Program, Princess Margaret Hospital, 610 University Avenue, Toronto, Ontario
M5G 2M9, Canada.
Phone: (416) 946-4595
e-mail: armand.keating@uhn.on.ca
John Kersey, MD, ASBMT Past-President, Founding Director Emeritus, Masonic Cancer Center,
Professor and Land Grant Fund Chair in Pediatric Oncology, University of Minnesota, 420 Delaware
Street, Minneapolis, MN 55455.
Phone: (416) 946-4595
e-mail: kerse001@umn.edu
Robert Korngold, PhD, Editor-in-Chief, Biology of Blood and Marrow Transplantation, Chairman,
Research Department, Hackensack University Medical Center, 40 Prospect Avenue, Hackensack,
NJ 07601
Phone: (551) 996-8664
e-mail: RKorngold@HackensackUMC.org
Ginna Laport, MD, ASBMT Secretary, Associate Professor - Med Center Line, Medicine - Blood &
Marrow Transplantation, Stanford Cancer Institute, Stanford University Medical Center, Division of BMT
300 Pasteur Drive, Room H3249, Stanford, CA 94305-5623.
Phone: (650) 723-0822
e-mail: glaport@stanford.edu
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Keith Sullivan, MD, ASBMT Past-President, James B. Wyngaarden Professor of Medicine, Duke
University Medical Center, 2400 Pratt Street, Durham, NC 27710.
Phone: (919) 668-1011
e-mail: sulli025@mc.duke.edu
Daniel Weisdorf, MD, ASBMT President, Director of the Adult Blood and Marrow Transplant Program,
University of Minnesota, 420 Delaware Street SE, Minneapolis, MN 55455.
Phone: (612) 624-0123
e-mail: weisd001@umn.edu
Staff
Thomas Joseph, MPS, CAE, Executive Director, American Society for Blood and Marrow
Transplantation, 85 West Algonquin Road, Suite 550, Arlington Heights, IL 60005.
Phone: (847) 427-0224
e-mail: thomasjoseph@asbmt.org
Robert Krawisz, MBA, Associate Executive Director, American Society for Blood and Marrow
Transplantation, 85 West Algonquin Road, Suite 550, Arlington Heights, IL 60005.
Phone: (847) 427-0224
e-mail: robertkrawisz@asbmt.org
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ASBMT POLICY STATEMENT
ASBMT Research Priorities
The American Society for Blood and Marrow Transplantation has reviewed recent advances and problems
in hematopoietic cell transplantation (HCT), and has identified priorities in several areas of basic and
clinical research:

Stem Cell Biology (cell manipulation, sources of stem cells, inducible pluripotent stem cells, cancer
stem cells)

Tumor Relapse (prevention and therapy for post-transplant relapse, immunotherapy with T cells and
dendritic cells)

Graft-versus-Host Disease (separation of GVHD and graft-versus-tumor effects, immune reconstitution
and GVHD, markers predicting GVHD, role of regulatory T cells)

Applying New Technology to HCT (genomics, proteomics, imaging, markers of immunologic recovery,
pharmacogenomics)

Expanded Indications for HCT (solid tumors, regenerative medicine, autoimmune diseases, response
to bioterrorism and radiation accidents)

Survivorship (long-term complications, longevity, quality of life)

Transplants in Older Patients (biology of aging, indications, outcomes and quality of life)

Improving Current Use of HCT (graft sources, conditioning intensity, cost-effectiveness)
These research areas are closely interrelated and of growing importance as a greater number and higher
risk patients are being transplanted. Each area has an interface between basic science and clinical
practice. Research in each of these areas can have direct and immediate clinical benefits.
Stem Cell Biology
Multiple sources of stem cells are now available for transplantation, including bone marrow, mobilized
peripheral blood stem cells and umbilical cord blood, as well as cells induced to provide particular
biological functions. The development of these multiple sources was initially for the purpose of expanding
the donor pool and facilitating access, but it now is clear that these cell populations have considerable
differences that can be therapeutically exploited. A better understanding of stem cell biology is needed to
optimize the therapeutic potential of these several cell sources. That understanding is essential to
successful action against cancer stem cells, with differing repopulation kinetics, repair capacities and
treatment resistance. More investigations are needed to better define the potential of inducible pluripotent
stem cells and problems that may be associated with their use.
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Tumor Relapse
Relapse remains of paramount concern after autologous HCT to treat hematologic malignancies. We
need to understand the mechanisms by which cancer cells can resist cytotoxic therapies in autologous
HSCT. Use of immune therapies, monoclonal antibodies, molecular targeted agents and
chemotherapeutics – alone or in combination – should be explored in the context of HCT. This may also
help in applying autologous HCT to a wider spectrum of malignancies.
Disease recurrence remains a major problem after allogeneic HCT in which cytotoxic therapy is combined
with immunotherapy provided by donor cells. A better understanding of the mechanisms by which cancer
cells resist cytotoxic therapy might lead to more effective immunotherapies, be they cellular, humoral or
vaccination, or a combination of these. These efforts also should include strategies for patients with
minimal residual disease after transplantation who are at a high risk of relapse but who may also be more
responsive to immunotherapy.
Studies have shown the effective use of adoptive T cell transfer after reduced-intensity conditioning in
patients with melanoma. However, adoptive therapy with allogeneic cells also is associated with the risk
of GVHD. Therefore, techniques that modify autologous cells so they can be used for tumor antigenspecific immunotherapy might improve efficacy and decrease toxicity. Studies on cell trafficking of immune
effector cells may also provide important information on the role of “homing” receptors and how those
could be used to design more effective therapy.
Graft-versus-Host Disease
GVHD is still the most frequent complication after HCT. While the use of reduced-intensity conditioning
regimens appears to lower the incidence – and possibly severity – of acute GVHD, there has been little
impact on chronic GVHD. We need a better understanding of the pathobiology of chronic GVHD and must
find new ways to target it clinically. The identification of early biomarkers and prognostic indicators could
be helpful in distinguishing patient populations who might benefit from different preemptive or therapeutic
strategies. Rather than broad immunosuppression, targeted approaches and possibly selective
immunostimulation should be explored. It is likely that this will lead to more individualized management
that considers the underlying disease, conditioning used for transplantation, prior therapy and patient age,
among other factors.
The role of regulatory T cells, shown to be of central importance for tolerance in animal models, deserves
clinical exploration. Natural killer (NK) cells are another effector arm in need of better characterization,
both as anti-tumor effectors and as immunodulators. Similarly, NK/T cells and suppressive dendritic cell
subsets require investigation of their immunomodulatory effects. Understanding pathways that can
distinguish GVHD from the graft -versus -tumor effect and the roles of the various immune cell populations
in these processes continue to be of central importance for applying HCT in cancer therapies.
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Applying New Technology to HCT
The advent of proteomic and genomics has increased the need to validate potential clinical markers.
Such markers have important implications for patient selection, prognosis and treatment. Application of
new imaging techniques will not only allow assessment of clinical efficacy and cellular therapy trafficking,
but also advance our basic understanding of disease processes. Using proteomics to determine
correlative markers for GVHD progression and to identify high-risk patients is of considerable importance.
Enzyme polymorphisms are likely relevant for drug metabolism and tissue repair, and a better
understanding of these relationships could help tailor conditioning regimens and GVHD prophylaxis.
Another critical area is development of immune monitoring parameters that reflect immune competence
and recovery, particularly with the increasing use of HCT in older patients.
Expanded Indications for HCT
HCT is known to induce tolerance and therefore may have a high impact in solid organ transplantation.
Studies to use HCT as a method to induce tolerance to a donor solid organ are on-going and have
generated significant enthusiasm with the ability to stop immune suppression in these patients. Further
studies are needed and encouraged.
HCT also may be useful in other therapies including, but not limited to, regenerative medicine (heart
disease and others), autoimmunity and restoration of hematopoiesis and immunity in the advent of
bioterrorism attack or mass radiological accident. Preclinical and clinical studies of immune reconstitution,
tolerance, efficacy and long-term effects will aid in understanding the applicability of HCT in these
conditions and situations.
Survivorship
Some former patients have now been followed for three and four decades after transplantation – cured of
their disease, but not necessarily without long-term complications. Patients are dealing with problems
such as chronic GVHD, immune deficiency, endocrinologic failure, bone loss and secondary malignancies.
Recent data suggest that life expectancy in patients after HCT is shortened by about 30 percent,
compared with age -matched controls. Thus, there is a high priority for investigations of risk factors for
late complications, development of relevant preclinical models, design of interventional strategies to
prevent complications and development of better means of assessing quality of life in long-term survivors.
Transplants in Older Patients
Older patients, who are an increasingly dominant population with respect to cancer and other diseases,
represent unique challenges in biologic understanding and extrapolation of preclinical findings and clinical
studies. Toxicities of cytoreductive therapies are heightened in the older patient. Poor immune function in
the aged leads to increased susceptibility to opportunistic infections and relapse from the cancer. The
science of aging needs to be applied to HCT. There are basic questions about the effects of age on
hematopoiesis, immune function and response to insult and stress. These need to be coupled with clinical
studies on the effects of age on recovery, outcomes and quality of life and general questions about “gain”
versus “loss” that may be associated with HCT.
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Improving Current Use of HCT
We need to define the optimal stem cell sources (cord blood, peripheral blood stem cells, bone marrow,
and ex vivo expanded stem cell populations) and conditioning intensity as they relate to the treatment of
specific diseases.
Finally, it must be acknowledged that HCT can be resource intensive and expensive. There is a need for
studies of the cost-effectiveness of HCT compared to other therapies for achieving desired outcomes.
– Amended by the ASBMT Executive Committee
October 20, 2011
___________________________________________________________________________
The ASBMT Board of Directors and Executive Committee wish to thank the members of the Task Force
on Research Priorities that helped with the development of this policy statement: H. Joachim Deeg, MD
(chair), John F. DiPersio, MD, PhD, James W. Young, MD, Richard T. Maziarz, MD, Claude Perreault,
MD, and David A. Margolis, MD, Robert H. Collins MD. The Board of Directors also wishes to
acknowledge the recommendation and encouragement of the ASBMT Corporate Council to develop these
research priorities.
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ANTITRUST GUIDELINES
FOR MEETINGS AND ACTIVITIES
Active participation in the ASBMT is an important aspect of membership in the ASBMT. Participation
not only adds to the vitality and energy of the organization, but it also furthers the ASBMT’s mission of
advancing the field of blood and bone marrow transplantation.
While the positive contributions of professional societies and associations are well recognized and
encouraged by government, association activities also are subject to close scrutiny under both federal and
state antitrust laws. The single most significant law affecting associations is the Sherman Antitrust Act,
which makes unlawful every contract, combination or conspiracy in restraint of trade. Because an
association is, by nature, a group of competitors joined together for a common business purpose, an
association satisfies what would ordinarily be a difficult element in proving an antitrust violation. As such,
any association activity that arguably could be perceived as a restraint of trade exposes ASBMT and its
members to antitrust risk.
Historically, the most significant area of antitrust concern for associations has been price fixing. Price
fixing is a very broad term which includes any concerted effort or action that has an effect on prices, terms
or conditions of trade, or on competitors. Accordingly, meeting participants should refrain from any
discussion which may provide the basis for an inference that they agreed to take any action
relating
to prices, services, production, allocation of markets or any other matter having a market effect. These
discussions should be avoided both at formal meetings and informal gatherings and activities.
In
addition, meeting participants should be sensitive to other matters that may raise particular antitrust
concern for associations: membership restrictions, codes of ethics or other forms of self-regulation,
product standardization or certification. The following are guidelines that should be followed at all ASBMT
meetings, informal gatherings and activities:

DON’T discuss your own or others’ prices or fees for service, or anything that might affect
prices or fees, such as costs, discounts, terms of sale, or profit margins.

DON’T stay at a meeting where any such price talk occurs.

DON’T make public announcements or statements about your own prices or fees, or those of
competitors, at any ASBMT meeting or activity.

DON’T talk about what other entities or their members or employees plan to do in particular
geographic or product markets or with particular customers.

DON’T speak or act on behalf of the ASBMT or any of its committees unless specifically authorized
to do so.

DO alert ASBMT staff or legal counsel about any concerns regarding proposed statements to
be made by the association on behalf of a committee.

DO consult with your own legal counsel or the ASBMT before raising any matter or making any
statement that you think may involve competitively sensitive information.

DO be alert to improper activities, and don’t participate if you think something is improper.
Adherence to these guidelines involves not only avoidance of antitrust violations, but avoidance of
behavior which might be so construed. Bear in mind that the antitrust laws are stated in general terms,
and that these guidelines only provide an overview of prohibited actions. If you have specific questions,
seek guidance from your own legal counsel or from the ASBMT’s Executive Director or legal counsel.
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