For Immediate Release

August 12, 2020

Media Contact

AABB Communications
Magda Yang
+1.301.215.6569
myang@aabb.org

National Blood Foundation Announces 2020 Early-Career Grant Recipients

The grants will fund innovative research in transfusion medicine and biotherapies.

Bethesda, Md. – AABB’s National Blood Foundation (NBF) announced today that it will be awarding eight early-career grants in 2020 to help fund innovative research in transfusion medicine and biotherapies. Annamaria Aprile, PhD; Agnieszka Czechowicz, MD, PhD; Areum Han, PhD; Robert H. Lee, PhD; Larry Luchsinger, PhD; Evan Orenstein, MD; David Roh, MD; and Kim Vanuytsel, PhD, will each receive a grant of up to $75,000 to further a one- or two-year research project.

“The National Blood Foundation's Scientific Research Grants Program has been such a critical part of launching the early research careers of budding scientists in transfusion medicine and biotherapies. Looking back on the NBF legacy, it is clear that the contributions of NBF recipients to the understanding and treatment of diseases that rely on transfusion medicine and biotherapies have been nothing short of remarkable,” said Jim Gorham, MD, PhD, chair of the NBF Scientific Research Grants Review Committee. “This is no more vividly illustrated than in 2020, as scientists supported by the NBF have made, and continue to make, highly impactful discoveries about COVID diagnosis and treatment, to the benefit of all throughout the world. We are thrilled to welcome this new cadre of early-career scientists to the NBF family and look forward to seeing their impact on the understanding of disease, and the delivery of life-saving transfusions to patients in need.”

The title and scope of each recipient’s research project is described below.

Annamaria Aprile, PhD
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Gene Transfer into Stem Cell Unit
Ospedale San Raffaele
Milan, Italy

Project Title: "Exploring the role of FGF23 in β-thalassemia: a novel target to ameliorate bone marrow microenvironment and anemia"

Summary of Proposed Research:
β-thalassemia (BT) is a globally widespread genetic anemia. Aprile’s team studies in BT mice highlighted an altered bone marrow (BM) microenvironment, with low bone density and increased circulating fibroblast growth factor 23(FGF23), affecting the outcome of therapeutic BM transplant. FGF23 regulates bone homeostasis and also erythropoiesis. Unraveling the mechanisms activated by FGF23 in BT and targeting its signaling might provide new therapeutic approaches to ameliorate both BM environment and anemia.


Agnieszka Czechowicz, MD, PhD
Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine
Stanford University School of Medicine
Stanford, CA

Project Title: "Determine genetic factors and mutations that regulate progression to bone marrow failure and myeloid neoplasia in Fanconi anemia"

Summary of Proposed Research:
Fanconi anemia (FA) is a disease that causes hematopoietic stem and progenitor cell (HSPC) defects, leading to bone marrow failure and leukemia. Drivers of hematological manifestations in FA are poorly understood. Czechowicz and team hypothesize that FA HSPCs must acquire unique genetic mutations and transcriptomic states before reaching full flown disease. With this research, they propose to analyze hematopoietic phenotypes, clonal hematopoiesis and HSPC transcriptomes in FA patients at different disease states.


Areum Han, PhD
Hematology/Oncology Department
Boston Children's Hospital
Biological Chemistry and Molecular Pharmacology Department
Harvard Medical School
Boston, MA

Project Title: "The role of the RNA editor-exonuclease axis in RNA turnover during erythropoiesis: from mouse embryo studies to in vitro modeling"

Summary of Proposed Research:
Han proposes to investigate the role of the RNA editor-exonuclease axis during erythropoiesis. She expects that results from this project will reveal for the first time the machinery behind global RNA decay during red blood cell differentiation and will eventually lead to advances in in vitro erythropoiesis and modeling hemoglobinopathies including for sickle cell disease from iPS cells.


Robert H. Lee, PhD
Department of Biochemistry and Biophysics
University of North Carolina
Blood Research Center
Chapel Hill, North Carolina

Project Title: "Efficacy of platelet transfusion in the setting of anti-platelet therapy"

Summary of Proposed Research:
Anti-platelet therapy (APT- aspirin, plavix) is used to prevent thrombosis, but these drugs can also cause bleeding. Platelet transfusions can prevent bleeding, especially in thrombocytopenic patients (low platelet counts), but patients on APT often have normal platelet counts. Lee and team recently showed that dysfunctional platelets can interfere with the function of the healthy transfused platelets. This study will investigate the effectiveness of platelet transfusion in the setting of APT.


Larry Luchsinger, PhD
Assistant Member
Stem Cell Regenerative Medicine New York Blood Center
New York, NY

Project Title: "Caveolae Coordination of Signal Transduction Pathways in Hematopoietic Stem Cell Function"

Summary of Proposed Research:
Blood stem cells (HSCs) have the potential to produce all cell types in the blood making HSCs the ultimate resource for manufacturing transfusion and cellular products. Yet the lack of reliable strategies to expand, or maintain, functional HSCs in vitro underscores major gaps still exist in our knowledge of HSC physiology. This study proposes to discover whether plasma membrane signaling pathways underpin HSC function and to develop HSC expansion methods to generate blood products in vitro.


Evan Orenstein, MD
Assistant Professor
Pediatrics and Clinical Informatics
Emory University
Atlanta, GA

Project Title: "Improving Patient Blood Management in Pediatrics through Automated Medical Error Detection and Clinical Decision Support Design"

Summary of Proposed Research:
In this study, Orenstein and team will first create and validate automated “trigger tools” to detect medical errors related to pediatric blood transfusions. The team will then use user-centered design to develop and evaluate clinical decision support systems targeting the most burdensome errors detected. These advances will support future clinical trials to determine the best electronic health record design to promote pediatric patient blood management.


David Roh, MD
Neurology
Columbia University
New York, NY

Project Title: “Red Blood Cell Contribution to Coagulopathy and Cerebral Oxygenation after Intracerebral Hemorrhage”

Summary of Proposed Research:
Hemostasis is thought to be driven primarily by coagulation factors, platelets and fibrinogen. Intracerebral hemorrhage (ICH) treatments focus on the repletion of these factors to minimize ongoing bleeding. However, outside of their known role of oxygen delivery, red blood cells (RBCs) may play a role in hemostasis. This project will explore the contribution of RBCs on both hemostasis and cerebral oxygenation after ICH in efforts to identify novel treatment targets to improve ICH outcome.


Kim Vanuytsel, PhD
Department of Medicine: Section of Hematology and Oncology and Center for Regenerative Medicine (CReM)
Boston Medical Center
Boston, MA

Project Title: “Validation of a potential curative gene editing approach across the diverse sickle cell disease (SCD) patient population using induced pluripotent stem cells (iPSC)”

Summary of Proposed Research:
Using a sickle cell disease (SCD) patient-specific induced pluripotent stem cell (iPSC) platform as a preclinical screening tool, Vanuytsel and team will validate the efficacy of novel therapeutic gene editing strategies across a diverse SCD patient population. Upon introduction of HbF modifying edits, the researchers will investigate fetal hemoglobin induction in patient-specific iPSC-derived erythroid cells to study differences in response based on genetic background and how this can predict and inform clinical treatment.

More information about the early-career Scientific Research Grants Program is available on the NBF web page.

About the National Blood Foundation
AABB’s National Blood Foundation (NBF), established in 1983, serves the fields of transfusion medicine and cellular therapies through grant making, educational offerings and industry leadership engagement and recognition.

About AABB

AABB is an international, not-for-profit association representing individuals and institutions involved in the fields of transfusion medicine and biotherapies. The association is committed to improving health through the development and delivery of standards, accreditation and educational programs that focus on optimizing patient and donor care and safety. AABB membership includes physicians, nurses, scientists, researchers, administrators, medical technologists and other health care providers. AABB members are located in more than 80 countries and AABB accredits institutions in more than 50 countries.

http://www.aabb.org