PROCEEDINGS FROM
ASBMT CORPORATE COUNCIL MEETING
October 2-3, 2011 Meeting
Royal Palms Resort
Phoenix, Arizona
Corporate
Participants:
J. Frank Glavin (Fresenius Biotech), Dr. Richard Kadota (Genzyme), Dave McFadden (Amgen),
Scott Miller (Celgene), Dr. Raul Perez-Olle (Otsuka), Dr. David Peritt (Hospira) and
Sharon Roell (Otsuka)
ASBMT
Participants:
Drs. Armand Keating (Chair), Richard Champlin, Nelson Chao, Helen Heslop, John Kersey,
Robert Korngold, C. Fred LeMaistre, Elizabeth Shpall, Keith Sullivan and Daniel Weisdorf
Guests:
Linda Caples (Medical College of Wisconsin), Dr. Denis-Claude Roy (Maisonneuve-Rosemont
Hospital Research Centre) and Raymond Wood (Cell Therapies Pty Ltd)
Staff:
Thomas Joseph and Bob Krawisz
This report summarizes the information that was gathered at the ASBMT Corporate Council Meeting. Speaker
presentations are available on the ASBMT Website: www.asbmt.org.
I.
Commercializing Cell Based Therapies within or Alongside an Academic Institution
Raymond J. Wood, Cell Therapies Pty Ltd
Cell Therapies P/L is an independent CMO specializing in translational medicine where cGMP skills are
required. They are a successful commercial venture running inside/alongside an academic institution. Cell
Therapies delivers autologous cell therapies for a mix of early Phase trials and one standard of care
regenerative medicine initiative. They are a multidisciplinary resource with a track record of translating
research protocols into successful clinical trials. Clients include organizations that need to manage their risk
profile and have embraced an outsourcing policy. Raymond Wood presented his view of what is coming in
the cellular therapy space and his experience in transforming a translational research unit inside an
academic research institute into a commercially successful service business.
Learning Objectives – Participants were able to determine:
1) Why set up a commercial venture and what can an academic center offer versus a commercial CMO?
2) Why Cellular Therapeutics?
3) The basis for a commercial operation.
4) Lessons Learned: Competing objectives and pragmatic decisions structures required to resolve
internal versus external requirements (researchers, SOPs, release criteria, regulators and project
management).
5) The answer to a key question for the stakeholders: service provider or a researcher?
Ray Wood is the Managing Director of Cell Therapies P/L, a spin out venture from the Peter MacCallum
Cancer Centre in Melbourne Australia. Ray has a track record of identifying and developing technological
opportunities. Ray’s background is electronic and bio-medical engineering spanning 30+ years. His
experience ranges from the continuous flow pathology analyzers of the early 70's and the first automated
hematology counters through the development and marketing of diagnostic ultrasound machines to more
recently delivering professional consulting services and creating biotechnology spin outs. Ray has
specialized in the identification and quantification of discoveries within Universities and Research
Institutes and their transformation into commercial ventures.
II.
Selective Allodepletion of T cells
Dr. Denis-Claude Roy, Maisonneuve-Rosemont Hospital Research Centre
Graft versus host disease (GVHD) remains a frequent and severe complication of allogeneic stem cell
transplantation. One approach to reducing alloreactivity is to deplete the graft of alloreactive T cells. Global
T cell depletion results in poor immune reconstitution with high mortality from viral infections and disease
relapse. Therefore, an approach to selectively deplete alloreactive T cells without compromising other
responses would be highly beneficial.
Learning Objectives – Participants were able to determine:
1) Major risks, advantages and disadvantages of mismatached related donors: haplo-identical
transplantation.
2) The selective allodepletion concept.
3) The use of photodynamic therapy.
4) T-Cell targeting.
5) Mechanisms of PD Cytotoxicity.
6) Haplo-matched stem cell transplantation process.
7) Survival after haplo-matched transplantation and ATIR infusion.
8) Extracorporeal Photopheresis (ECP): Treatment of GVHD.
9) T cell recovery Post-TH9402 Extracorporeal phototherapy :Increased regulatory T cells.
Dr. Denis-Claude Roy is Scientific Director of the MRH Research Centre. He is renowned for his research
on HSC transplantation, as the 165 publications in prestigious journals that he has authored or co-authored,
as well as the four patents that he holds, attest.
III.
It’s Not Over After Day 100: Studying Late Effects of Hematopoietic Cell Transplantation
Dr. Daniel Weisdorf, University of Minnesota
In patients who receive an allogeneic bone marrow transplant as treatment for acute myelogenous or
lymphoblastic leukemia, chronic myelogenous leukemia, or aplastic anemia and who are free of their
original disease two years later, the disease is probably cured. However, for many years after
transplantation, the mortality among these patients is higher than that in a normal population. Dr. Weisdorf
discussed the problem and pointed out a number of challenges in finding a solution: too many issues;
incidence and risk factors not well defined; patients are remote from HCT center; chronic morbidity and
not acute; late effects diseases are less pressing than cancer or GVHD, so they get less attention.
Learning Objectives – Participants were able to determine:
1) What and how big is the problem.
2) Recommended screening and preventive practices – organ problems and second cancers.
o Joint Recommendations of the EBMT, CIBMTR, ASBMT (Rizzo et al BBMT 2006; 12:136)
o Update being submitted for publication
3) Prospective trial challenges.
o Specific organ problems
o Guides for health screening
o BMT survivors’ study
2
IV.
Improving Accrual to Clinical Trials
Dr. Daniel Weisdorf, University of Minnesota
Prospective clinical trials for blood transplantation and BMT are made more difficult by limited patient
numbers. Dr. Weisdorf indicated that accrual is always too slow, but there are ways that investigators can
make it better. He discussed a number of solutions including the power of collaboration. To address the
challenges of prospective clinical study of BMT, the Blood and Marrow Transplant Clinical Trials Network
(BMT CTN), sponsored by the National Heart, Lung, and Blood Institute/National Cancer Institute, was
established. The network’s charge is to design and execute multicenter clinical trials requiring participation
from the network and from noncore centers for successful performance and to address rare diseases and
unique populations that could not be well studied in individual centers.
Learning Objectives – Participants were able to gain insights on:
1) Major frequent problems in clinical trials.
2) Why BMT clinical trials are difficult.
3) What makes a good protocol?
4) Cautions when writing protocols.
5) Understanding the potential patient population.
6) How not to design a protocol to fail.
7) Why biological assignments trials are sometimes necessary.
8) Accrual plan assessment.
Dr. Daniel Weisdorf is ASBMT’s President and Director of the Adult Blood and Marrow Transplant
Program at the University of Minnesota. He serves as the Scientific Director of the National Marrow Donor
Program and chairs the Acute Leukemia Committee of the International Bone Marrow Transplant
Registry/Autologous Bone Marrow Transplant Registry and is the University of Minnesota Principal
Investigator on the NIH-sponsored Bone Marrow Transplantation Clinical Trials Network
V.
The Changing CME Environment
Linda Caples, Medical College of Wisconsin
Continuing Medical Education (CME) is in a state of transition. Internal and external forces are reshaping
the CME enterprise. From the internal perspective ACCME is re-emphasizing the importance of
independence in CME activities as well as careful assessment of the impact of accredited CME programs
on physician competence, performance, and patient health status. The new focus for CME is answering this
question: "Did the CME make a difference to physician practices?" Externally, government is taking a
close look at how the CME enterprise is funded and the influence of that funding on physician behavior.
Funding agencies are requiring greater accountability for demonstrating that their investment was wisely
spent. This is a challenging time for CME professionals, who are expected to provide a new direction for
continuing medical education in the midst of an evolving paradigm and sometimes-competing expectations.
Ms. Caples provided the Council with valuable insights on how to navigate through the complex CME
environment and meet stakeholder objectives.
Learning Objectives – Participants were able to understand:
1) The New CME Guidelines.
2) ACCME’s impact on corporate sponsored symposia and training programs.
3) Strategies for meeting corporate and physician needs.
Linda Caples is Director of the Office of Continuing and Professional Education at the Medical College
of Wisconsin in Milwaukee, WI.
3
VI.
Shortage of BMT Physicians: What can be done to Recruit more BMT Fellows?
Dr. Richard Champlin, M.D. Anderson Cancer Center
The adult transplant workforce has very few physicians under age 40. Nearly 50% of adult transplant
physicians are over age 50 whereas only 28% of pediatric transplant physicians are over age 50. By 2020, it
is projected that the BMT community will need 1,264 new adult transplant physicians and 94 pediatric
transplant physicians. Training time for a physician is approximately 15 years. The capping of both medical
school slots and residency slots since the early ’80s is now having a very big impact on supply, but other
factors are also affecting supplies such as generational differences, lifestyle expectations, and the change of
the medical workforce from being mostly men. Workforce shortages are being reported for many
specialties. Workforce problems are also present for nurses, pharmacists and medical technologists. So
increasing use of general internists and mid-level providers may not exist as a solution. Transplant
physicians must be actively engaged in the medical education process to show young medical students and
residents who are not committed to another sub specialty career the excitement and challenges of a career
in bone marrow transplantation, so that the BMT field will have providers for the future. Dr. Champlin
provided an overview of the projected shortage of the BMT physician workforce and proposed solutions.
Learning Objectives – Participants were able to gain insights on:
1)
2)
3)
4)
5)
6)
7)
8)
The overall physician shortage – what are the root causes?
Factors driving physician demand.
BMT Physician supply and demand projections through 2020.
The demographic profile of current BMT physicians.
Training required for BMT physicians.
Profile of current BMT physician training programs.
Factors attracting Fellows to the field.
Strategies to increase the supply of BMT physicians.
Dr. Richard Champlin is a Past-President of ASBMT and Chair of the Department of Stem Cell
Transplantation and Cellular Therapy, Division of Cancer Medicine, The University of Texas MD
Anderson Cancer Center, Houston.
VII.
Promoting ASBMT Research Priorities
Dr. Helen Heslop, Baylor College of Medicine
The idea of developing research priorities came from ASBMT’s Corporate Council. Corporate members of
Council indicated that it would be helpful if they had a clearer picture of ASBMT’s most pressing research
needs to help in their decisions about allocation of research dollars in areas most beneficial to needs for
patient care. It’s not that there haven’t been discussions with industry about research needs in the past. But
ASBMT had never codified priorities. The Board agreed that it seemed like something ASBMT should do.
The first set of priorities were approved by the Executive Committee in 2009. They were published in
Biology of Blood and Marrow Transplantation and posted on the ASBMT Website. Dr. Heslop explored
development of strategies for promoting ASBMT’s research priorities to the National Institutes of Health
(NIH) and to private industry.
Learning Objectives – Participants were able to gain insights on:
1)
2)
3)
4)
The current list of research priorities – why they were selected.
The process for updating priorities.
Strategies for influencing NIH and government funding agencies.
Strategies for funding research.
Dr. Helen Heslop is a Past-President of ASBMT and Professor in the Department of Pediatrics and
Medicine Section of Hematology-Oncology, Baylor College of Medicine
4
VIII.
Costs, Charges and Reimbursements in BMT: Is there any Good News for the Future?
Dr. John Kersey, University of Minnesota
ASBMT is exploring ways to monitor and update members on how to best prepare for and position the
BMT physician and transplant center for health care reform. This includes issues related to managed care,
referral networks (the medical home), electronic medical records, meaningful use, accountable health
partnerships and third-party recognition and reimbursement of diseases related to blood and marrow
transplantation and cellular therapy. ASBMT is committed to being a strong advocate for its members as
health care reform is implemented. It will recommend changes through legislation if implementation
reveals problems or unintended consequences that impede the delivery of BMT transplantation and cellular
therapy. It will recommend educational programming to help members and their patients adapt to new
delivery models. It will collaborate with payers and health systems as they make the most of opportunities
in the law to improve patient care. Dr. John Kersey led a discussion on managing BMT in the brave new
world of healthcare reform.
Learning Objectives – Participants were able to gain insights on:
1) Reimbursement problems -- the changing payer mix and clinical research issues.
2) BMT costs -- the Minnesota study on the cost of adult BMT.
3) BMT’s reliance on commercial payers to maintain margins.
4) When reimbursements fall, how to reduce costs to maintain margins.
5) Strategies for reducing costs through standardized protocols and research progress
Dr. John Kersey is a Past-President of ASBMT and the founding director of the Masonic Cancer Center,
which became a National Cancer Institute-designated comprehensive cancer center in 1998. He currently
holds the Children's Cancer Research Fund Endowed Chair in Pediatric Oncology and is a professor in the
University of Minnesota Departments of Laboratory Medicine/Pathology and Pediatrics.
IX.
Insurance for Clinical trials
Dr.Keith Sullivan, Duke University Medical Center
The National Conference of State Legislatures reports that for cancer patients, properly designed and
conducted clinical trials represent an important therapeutic option, as well as a critical means of advancing
medical knowledge. Lack of insurance coverage is a barrier to patients who might otherwise participate.
Sixty percent of patients in one survey cited fear of insurance denial as a major reason for not participating
in clinical trials. Another study found only a slight increase in treatment costs for adult clinical trial
patients compared to nonparticipants. Some large HMOs have computed costs associated with patients in
clinical trials. Kaiser Permanente discovered the cost of medical care for enrollees in clinical trials that
haven't had bone marrow transplant were no higher than for patients who were not enrolled in a trial. The
Kaiser report further states, “Kaiser has been participating in cancer clinical trials without substantial
increases in the direct costs of medical care.” Researchers at the Mayo Clinic found that patient care costs
for those enrolled in clinical trials is only slightly more than for patients who received standard therapy
protocols. The Institutes of Medicine has also found the following:

The reimbursement costs are limited to the cost of “standard care” which would be covered if the
patient were not enrolled in the trial;

Only a small percentage (approximately 20%) of cancer patients are eligible to participate in a clinical
trial and very few (approximately 3% of cancer patients and less than 0.5% of Medicare patients)
currently enroll. Even if enrollment was increased to the full 20 percent, it is unlikely that these
numbers will significantly impact overall costs to health plans;

Through clinical trials, we will be able to identify ineffective treatments, which could save health plans
money and will benefit the nation as a whole.
5
Dr. Keith Sullivan led a discussion on how insurance denials restrict clinical trials.
Learning Objectives – Participants were able to gain insights on:
1) The impact of insurer denials on clinical trials.
2) How insurance status affects outcomes.
3) State statutes for clinical trials coverage.
4) Reasons of insurer denials.
5) Strategies for improving coverage and accrual.
Dr. Keith Sullivan is a Past-President of ASBMT and serves as the James B. Wyngaarden Professor of
Medicine at Duke University Medical Center where he served as Chief, Division of Medical Oncology and
Transplantation. Dr. Sullivan also serves as the Director of Long-term Follow-up and Information Research
Program, in the Division of Cellular Therapy.He has extensively published in the area of bone marrow
transplantation with over 400 peer-reviewed publications and book chapters.
X.
Adjournment
It was agreed that a follow-up meeting should be scheduled during the 2012 BMT Tandem Meetings in
February in San Diego. The meeting adjourned at 3:00 p.m. (MST).
6