CAF Awards $250,000 in Medical Research Fellowships for 2024-2025

We’re proud to announce five new Cooley’s Anemia Foundation Medical Research Fellowships for the 2024-2025 grant cycle. The total amount of funding for the research Fellowships is $250,000.

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.

Meet the Fellows:

Dr Jun Liu, MD., PhD

Dr. Jun Liu is a research fellow under joint mentorship by Dr Daniel Tenen and Dr Li Chai, at Harvard Medical School. She is also a clinical fellow in Transfusion Medicine at the Harvard Combined Transfusion Medicine fellowship program, with clinical and research interests in hemoglobinopathies.

Dr Liu is characterizing a novel small molecule degrader of ZBTB7A/LRF, a transcriptional repressor of gamma globin, as a means of inducing gamma globin expression in human erythroid cells. This approach can offer potential therapeutic benefit in beta-globinopathies such as sickle cell disease and beta-thalassemia.

Dr Liu is working closely with medicinal chemists to further refine the molecular structure of the compound to optimize its potency and minimize its toxicity.

Ellen B. Fung, PhD RD CCD & Vanessa Yingling, PhD

Dr. Ellen B. Fung is an Adjunct Professor in the Division of Hematology, Department of Pediatrics within the School of Medicine at the University of California, San Francisco (UCSF).

She has a long-standing curiosity on the impact of nutrition and physical activity on bone health in children and adults, particularly those with thalassemia. She is co-author of the first textbook related to the assessment of pediatric bone health, and in 2023 authored a guidebook on nutrition for patients with Thalassemia, published by the Thalassemia International Federation.

Dr. Fung is the Director of the Bone Density Clinic at UCSF Benioff Children’s Hospital Oakland and the Director of the Summer Student Research Program; a UCSF summer research internship for underrepresented students passionate about science. When she is not in the office, or mentoring students, she enjoys swimming, hiking, gardening and baking.

Dr. Vanessa Yingling is a Professor of Kinesiology at California State University, East Bay. She is trained as a biomechanist with specific training during a post-doctoral fellowship in bone mechanics and physiology. As a PI on NIH-funded grants, she has experience using animal models to investigate factors that affect bone strength development.

In 2013 she transitioned her research to focus exclusively on human subjects. She has contributed to science establishing the relationship between field tests of muscle function and bone strength in athlete and healthy populations. She has worked with American Bone Health in the revision the NIH Best Bones Forever program into a broad-based curriculum for both girls and boys that can be implemented in after school programs and camps and currently serves on the Fitness Gram Advisory Board.

Most recently, Drs. Yingling and Fung collaborated on a project to understand bone strength relative to body size in patients with thalassemia, which led to the current exercise intervention project.

A short description of the project

Over half of all adults with thalassemia (Thal) have low bone mass and are at significant risk for fracture. The reasons for low bone mass are multifactorial, though bone morbidity is due, in part, to inactivity. Bisphosphonate medications often used to treat osteoporosis have many side effects and are often contraindicated in patients with thalassemia. Therefore, non-pharmacological strategies are needed to not only improve bone health and reduce fracture risk, but may also alleviate bone pain.

In non-Thal populations, physical activity is known to increase muscle mass, improve bone density and strength, and reduce pain. Patients with Thal are less active compared to their peers, and we have shown bone deficits are associated with inactivity. Previous studies suggest that lifestyle factors can influence bone health in thalassemia. The logical question remains, can physical activity improve bone health? This study is the natural, most plausible, progression to correcting the significant burden of osteoporosis in thalassemia.

In year one of this study, we propose a 3-month home based exercise intervention. The short-term outcomes to be explored are improvements in muscle mass and function- precursors to long term bone outcomes as well as pain and quality of life.

We plan to extend the intervention with a second year of funding, and direct bone outcomes will be assessed. If effective, this simple, non-invasive, cost-effective, easy to implement exercise therapy could be expanded broadly with minimal effort yet have profound effects.

Audrey Belot, PhD, Post Doc Fellow

The objectives of my Dr. Belot’s PhD were to study iron metabolism regulation and to identify new therapeutic strategies for diseases linked to iron metabolism. Using an IRIDA mouse model, Dr. Belot showed that a hepcidin-suppressing molecule corrects the iron deficiency and anemia in IRIDA. She was also interested in the role of iron in non-alcoholic fatty liver disease (NAFLD), which can progress in some cases to a more severe stage, non-alcoholic steatohepatitis (NASH), for which no treatments are available.

Dr. Belot showed that hepcidin production is increased in this disease resulting in liver iron retention, which aggravates NAFLD. The characterization of this dysregulation has led to identifying a promising new therapeutic target, HuR, to prevent disease progression. Her third project focused on regulating hepcidin expression through an anti-matriptase-2 antibody in β-thalassemic mice to improve anemia and iron overload.

As a post-doc, Dr. Belot joined Dr. Hamza’s lab to study heme metabolism which is highly complementary to iron metabolism as the majority of iron is within heme. For her postdoctoral research, Dr. Belot specifically addressed the role of the heme transporter HRG1 in red cell maturation in both steady-state and disease conditions (Sickle cell disease, β-thalassemia).

This project combines her experience studying red cell development during her Ph.D. with heme homeostasis and transport during her Postdoctoral research. Dr. Belot’s short-term goal is to have a better understanding of the interplay of red blood cells metabolism and heme at steady-state and during diseases such as β-thalassemia to find innovative therapeutic strategies for those patients.

Her long-term goal is to establish her own laboratory and lead a research group as a PI on the implication of the unappreciated trafficking of heme between cells in different organs and its role in both steady-state and disease conditions.

Abhirup Bagchi, PhD

Dr. Bagchi currently a postdoctoral fellow in the laboratories of Dr. Gerd Blobel and Dr. Eugene Khandros, in the Department of Hematology at the Children’s Hospital of Philadelphia. Dr. Bagchi completed his doctoral studies on developing lentiviral based gene therapy vectors for hemoglobinopathies at the Centre for Stem Cell Research, Vellore, India.

Dr. Bagchi’s research focuses on understanding how individual components of the essential BAF chromatin remodeling complex are involved in regulation of red blood cell production and hemoglobin gene expression. The goal of this project is to identify new therapeutic targets to enhance fetal hemoglobin expression and red blood cell maturation in b-thalassemia.

Dr. Shobana Navaneethabalakrishnan

Dr. Navaneethabalakrishman is a biotechnologist with over a decade of expertise in reproductive biology. She obtained my master’s degree in biotechnology from Bharathiar University, India, where her research focused on the impact of titanium dioxide nanoparticles on reproductive organs of female mice.

As a Junior Research Fellow at the University of Madras, Dr. Navaneethabalakrishman investigated the effect of hexavalent chromium on the hypothalamo-hypophyseal-gonadal axis. Her doctoral studies at the University of Madras centered on understanding the molecular mechanisms underlying the effect of gestational exposure to hexavalent chromium on Sertoli cells in F1 progeny.

At Texas A&M University, as a postdoctoral researcher, she explored the intersection of hypertension and reproductive biology, specifically studying the role of gonadal macrophages in hypertension-associated reproductive dysfunction. To further deepen her knowledge of macrophage biology and its implications in disease models, Dr. Navaneethabalakrishman joined Dr. Francesca Vinchi’s lab at New York Blood Center, where she developed a keen interest in investigating the role of iron/heme-driven macrophages in the pathogenesis of beta-thalassemia.

The primary focus of the current study is to elucidate the role of heme/iron-activated macrophages in bone marrow (BM) dysfunction in beta-thalassemia, focusing on their impact on ineffective erythropoiesis and regulation of hematopoietic stem/progenitor cell (HSPC).

Furthermore, she will also evaluate therapeutic strategies aimed at modulating macrophage function and heme scavenging to alleviate BM inflammation, improve erythropoiesis, and preserve HSPCs. This research endeavors to provide insights into novel treatment approaches for β-thalassemia, potentially leading to enhanced clinical outcomes for affected individuals.

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