CAF Awards $200,000 in Medical Research Fellowships
We’re proud to announce two new Cooley’s Anemia Foundation Medical Research Fellowships and two renewal Medical Research Fellowships for the 2023-2024 grant cycle. The total amount of funding for the two research Fellowships is $200,000.
These Fellowship recipients were assessed on the basis of the quality of the scientific content, the academic accomplishments and future promise of the investigator, the quality of the mentor in the case of postdoctoral fellowships, and, of particular importance, the relevance of the project to the understanding and treatment of Thalassemia. The CAF Scientific Review Committee reviewed all applications carefully while adhering to the highest standard for scientifically un-biased reviews and made its recommendations for funding to the CAF Board of Directors, who approved those recommendations at its annual Board meeting.
We thank Dr. Janet Kwiatkowski, Chair of the CAF Scientific Review Committee and Medical Advisory Board, and the other members of the Scientific Review Committee for carefully reviewing the grant applications and making these important recommendations. We are excited to see what the investigators learn in these important and cutting-edge experiments.
First-Year Fellows
Dr. Yanan Sun is currently a postdoctoral fellow in Dr. Shuaiying Cui’s Laboratory in the Department of Medicine at Boston University School of Medicine and Boston Medical Center.
Dr. Sun’s research focuses on the reactivation of fetal hemoglobin (HbF) in adult erythroid cells, where she made quite significant breakthroughs.
Dr. Sun is playing a leading role in her ongoing β-thalassemia and SCD-related project sponsored by Cooley’s Anemia Foundation research fellowship. This project aims to study a new compound to induce fetal hemoglobin production. The focus of this project is its effect in a related hemoglobinopathy, sickle cell disease. They will also study it in normal human stem cells as project 2.
Dr. Premila P. Samuel of the University of Illinois at Urbana-Champaign will focus on uncovering hidden states in hemoglobin mis-assembly.
This project will use computational chemistry techniques to capture and characterize three-dimensional configurations and chemical interactions of states along hemoglobin mis-assembly pathways that lead to thalassemia manifestations, critical information which traditional lab-based techniques have not been able to fully access.
The overall project goal is to develop a virtual platform that will be used to model thalassemia pathways in red blood cells and subsequently screen for drugs that counter the toxic products of thalassemia in a rapid and cost-effective manner.
Second-Year Fellows
Francesca Vinchi, PhD, of New York Blood Center, is studying the role of Non-Transferrin Bound Iron (NTBI) and iron toxicity in Bone Marrow Transplant (BMT) complications, focusing on beta-thalassemia.
In the first 6 months of grant support, Dr. Vinchi showed that conditioning elicited NTBI negatively impacts BMT outcome in wild type mice. She also showed that conditioning aggravates NTBI formation in thalassemic mice and that iron restriction using hepcidin mimetics prevents this effect.
In the second year of funding she proposes to address the impact of conditioning elicited NTBI and pre-existing iron overload on BMT outcome and their impact on peri-transplant toxicities in thalassemia.
Beth Apsel Winger, MD, PhD of the University of California, San Francisco/Benioff Children’s
Hospital will use a new technique to improve dose selection of deferasirox for patients with
thalassemia.
In a study of 50 patients, Dr. Winger will develop a dosing algorithm that
takes into account multiple clinical factors such as age, body size, and liver and kidney
function as well as evidence of inadequate control of iron stores and complications of
deferasirox. She and her colleagues have already successfully applied this methodology,
known as population pharmacokinetics, to other therapies such as conditioning therapy
for bone marrow transplant.