The Science of Conditioning

Event Details

Meeting Resources

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Purpose

The purpose of this workshop on the science of conditioning is to bring together investigators from across basic science and clinical disciplines to share recent insights into the effects of current conditioning regimens, which can result in deleterious effects in patients with nonmalignant hematological disorders. This webinar also will highlight emerging discoveries regarding innovative conditioning regimens, as well as gaps in the science regarding how conditioning therapies affect both the bone marrow microenvironment and the engraftability of the hematopoietic stem cell.

Objectives

We expect that this meeting will provide a platform to establish productive collaborations and stimulate new ideas to enable substantial progress in understanding the role of bone marrow conditioning in outcomes of nonmalignant hematological diseases and will have promising potential to address ongoing challenges in the hematology field.

Background

Research into the treatment of nonmalignant hematological disease using hematopoietic stem cell transplant (HSCT) has seen explosive growth within the last decade. Recent outcomes of clinical trials combining HSCT and genome engineering to correct specific genetic defects have highlighted the critical need to understand the impact of bone marrow conditioning therapy on the microenvironment in these hematological disorders.

Registration Deadline

January 25, 2024

Agenda

12:00 p.m. – 12:20 p.m.

Session I: Welcome Remarks and Introductions
Griffin P. Rodgers, M.D., M.A.C.P., Director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH)
Shilpa Hattangadi, M.D., Program Director, Division of Kidney, Urologic, & Hematologic Diseases, NIDDK, NIH

12:20 p.m. – 12:40 p.m.

Session II: Setting the Stage—Selecting Conditioning Regimens for Specific Clinical Disorders
Suneet Agarwal, M.D., Ph.D., Co–Program Leader for the Stem Cell Transplant Center, Dana-Farber/Boston Children’s Cancer, and Blood Disorders Center, “Conditioning for Hematopoietic Cell Therapies in Nonmalignant Diseases—Getting the Monkey off Our Backs”

Session III: Innovations in Conditioning

12:40 p.m. – 1:35 p.m.

Preparing the Seed and Soil for Transplantation
Moderator: John Williams, Ph.D., Program Director, National Institute on Aging, NIH Anastasia Tikhonova, M.D., Ph.D., Assistant Professor, Department of Medical Biophysics, University of Toronto, Canada
Matthew Porteus, M.D., Sutardja Chuk Professor of Definitive and Curative Medicine, Stanford Medicine, Stanford University, TBD—Ex Vivo HSC Genetic Engineering
Robert Signer, Ph.D., Associate Professor, Medicine, University of California, San Diego, “Stem Cell Fitness and Longevity: The Role of Proteostasis”

Session Panel Discussion

1:35 p.m. – 2:30 p.m.

Alternative to Chemotherapy-based Conditioning: Approaches based on Antibodies
Moderator: Cindy Roy, Ph.D., Program Director, Division of Kidney, Urologic, & Hematologic Diseases, NIDDK, NIH
Agnieszka Czechowicz, M.D., Ph.D., Assistant Professor of Pediatrics (Stem Cell Transplantation), Stanford Medicine, Stanford University
John Dipersio, M.D., Ph.D., Director, Gene and Cellular Therapy, Washington University in St. Louis
Daisuke Araki, M.D., Staff Clinician, and Andre Larochelle, M.D., Ph.D., Investigator, Regenerative Therapies for Inherited Blood Disorders, National Heart, Lung, and Blood Institute (NHLBI), NIH, “Anti-cMPL Immunotoxin Effectively and Safely Depletes Human and Rhesus Hematopoietic Stem Cells: Implications for Pre-transplant Conditioning”

Session Panel Discussion

2:30 p.m. – 2:40 p.m.

Break

2:40 p.m. – 4:05 p.m.

Alternative to Chemotherapy-based Conditioning: Other Approaches
Moderator: Brian Bai, Ph.D., Program Director, NHLBI, NIH
Paula Rio, Ph.D., Head of the Bone Marrow Aplasia Unit at the Biomedical Innovation Unit, CIEMAT, Madrid, Spain, “Long-term Follow-up of Fanconi Anemia Patients Treated with LV-mediated Gene Therapy”
Susanta Hui, Ph.D., D.A.B.R., Professor, City of Hope, “Targeted Radiation-based Conditioning to Enhance Donor Mobilization and Engraftment and Preserve Organ Function”
Luigi Naldini, M.D., Director, San Raffaele Telethon Institute for Gene Therapy, Milan, Italy, “Mobilization-based Conditioning Coupled with Transient Engraftment Enhancers”
Saar Gill, M.D., Ph.D., Associate Professor of Medicine (Hematology–Oncology) at the Hospital of the University of Pennsylvania, Perelman School of Medicine, “Targeting CD45 as an Immunotherapeutic Conditioning Regimen”
Lukas Jeker, M.D., Ph.D., Assistant Professor, University of Basel, Switzerland, “Molecular Cell Shielding for Conditioning"

Session Panel Discussion

4:10 p.m. – 5:00 p.m.

Session IV: Whiteboard: Brainstorming Gaps in the Field and Needed Future Resources
Moderator: Shilpa Hattangadi, M.D., Program Director, Division of Kidney, Urologic, & Hematologic Diseases, NIDDK, NIH
All Participants

5:00 p.m.

Adjournment