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 graft- versus-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

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 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

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 FOA on Early-Phase Clinical Trials for Blood Cell Therapies (R01)

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