Download ASBMT Research Priorities - American Society for Blood and

American Society for Blood
and Marrow Transplantation
Promoting Research Priorities
Helen Heslop
ASBMT Research Priorities
• Initiative developed from
corporate council meeting
• Need for clearer picture of our
most pressing research needs
ASBMT Research Priorities
2008 Subcommittee
• Bill Murphy, William Drobyski, John
Kersey, Rob Negrin, Pavan Reddy
2009 Subcommittee
• Joachim Deeg, John DiPersio, Jim
Young, Richard Maziarz, Claude
Perreault, David Margolis, Robert Collins
ASBMT Research Priorities
• Stem Cell Biology
–Cell manipulation
–Sources of stem cells
–Inducible pluripotent stem cells
–Cancer stem cells
ASBMT Research Priorities
• Tumor Relapse
–Prevention and therapy for
post-transplant relapse
–Immunotherapy with T cells
and dendritic cells
ASBMT Research Priorities
• Graft-versus-Host Disease
– Separation of GVHD and graftversus-tumor effects
– Immune reconstitution and GVHD
– Markers predicting GVHD
– Role of regulatory T cells
ASBMT Research Priorities
• Expanded Indications for HCT
– Solid tumors
– Regenerative medicine
– Autoimmune diseases
– Response to bioterrorism
– Radiation Accidents
ASBMT Research Priorities
• Improving Current Use of HCT
– Graft sources
– Conditioning intensity
– Cost-effectiveness
ASBMT Research Priorities
• Transplants in Older Patients
–Biology of aging
–Indications
–Outcomes and quality of life
ASBMT Research Priorities
• Survivorship
–Long-term complications
–Longevity
–Quality of life
ASBMT Research Priorities
• Applying New Technology to HCT
– Genomics
– Proteomics
– Imaging
– Markers of immunologic recovery
– Pharmacogenomics
TCR V-beta analysis to track T cell
persistence in vivo
Pre-CTL
Post-CTL
CTL line
1000
10000
TCR sequences in both
CTL and peripheral blood
1000
100
100
10
10
10
100
PBMC
1000
10
100 1000 10000
PBMC
Strategies for Discussion
Working with NIH and Government
Funding Agencies
– Effects economic situation and
healthcare reform will have on
funding medical research.
Strategies for Discussion
Working with NIH and Government
Funding Agencies
– Develop strategies on how to
influence NIH to support ASBMT’s
research agenda.
– Conferences to generate RFAs
Working Group of scientific and clinical
investigators on September 22-23, 2008 in
Bethesda , Maryland , to discuss translational
research for evaluating cellular therapies for
blood diseases
http://www.nhlbi.nih.gov/meetings/workshops/e
nhance-cell-therapy-exesum.htm
Working Group Members:
Armand Keating (Working Group Chair)
Bruce Blazar, University of Minnesota
Shelly Carter The EMMES Corporation, Rockville MD
Robert J. Deans Athersys Inc, Cleveland OH
Adrian Gee, Baylor College of Medicine, Houston TX
Helen Heslop, Baylor College of Medicine, Houston TX
Mary M. Horowitz, CIBMTR
Edwin M. Horwitz, CHOP
Sally Hunsberger, PhD. Biometrics Research Branch, NCI,
Yoken Saunthararajah, MD.
David Scadden, MD. Harvard Stem Cell Institute, Boston MA
Judith A. Shizuru, Stanford CA
Daniel Weiss, University of Vermont, Burlington VT
FOA on Early-Phase Clinical Trials
for Blood Cell Therapies (R01)
Treat graft vs. host disease (GVHD) or increase
Immune reconstitution with regulatory T cells
Prevent post-transplant relapse using chimeric antigen
receptors (CARs)
Provide safe blood cell products for transfusion
applications
Treat monogenic blood diseases, using improved
genetic vectors for hematopoietic stem cells
Use mesenchymal stem cells (MSC) to treat GVHD or
as stem cell adjunct to improve engraftment
Strategies for Discussion
Do we need to update priorities?
How to influence NIH to support
ASBMT’s research agenda.
How to fund research
Other suggestions