Grant Recipients

For nearly 30 years, AAMDSIF has provided research grants totaling in excess of $5 million to an international group of more than 90 researchers to help advance the understanding and treatment of aplastic anemia, myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH).

The two-year grants have helped bring forth new insights into the causes and therapeutic approaches for these diseases. These grantee profiles present the grantees by year the awards were granted, and a summary of their grant-funded research projects.

Research Fund

Grant Year: 2015

Anastasios Karadimitris, MD, PhD

PNH Research and Support Foundation

Our proposed research addresses one of the main and unresolved issues in the biology of paroxysmal nocturnal hemoglobinuria (PNH) and idiopathic aplastic anemia (IAA). Both disorders are considered to have an autoimmune mechanism involving auto-reactive T cells targeting hematopoietic stem cells (HSC) leading to their profound depletion. It is widely documented that the pathogenesis of IAA and PNH overlap to a great extent, with up to 50% of patients with IAA developing a PNH clone or bonafide PNH and a subgroup of patients with PNH evolving into IAA. The proposed work has the potential to unravel novel cellular and molecular mechanisms that underpin HSC depletion in PNH and IAA, with wider implications for both the hematology and immunology scientific communities.

Katherine King, PhD

Liviya Anderson Research Fund

This AAMDSIF research grant will enable us to do basic and essential work on the effects of inflammatory signaling on bone marrow stem cells. We have known for some time that excessive inflammation can suppress bone marrow; now we are taking the next steps to find out how inflammation injures hematopoietic stem cells. We hope that our research will lead to novel ways to interrupt the development of aplastic anemia.

Shahram Kordasti, MD, PhD

Research is Hope Fund

Bone marrow failure syndromes are not only serious and debilitating diseases but they can also progress toward leukemia. Identifying the specific factors contributing into this malignant transition is extremely important. Our group focus in past few years has been the role of the immune system in bone marrow failure and we showed the importance of specific subtypes of immune cells in both pathogenesis and malignant transformation of these diseases. The AAMDSIF research grant will allow us to focus on differences in immune changes in aplastic anemia and paroxysmal nocturnal hemoglobinuria (PNH) and will enable us to utilize the state of the art technologies (such as multidimensional mass cytometry and high throughput DNA sequencing) to further understand the role of immune system in these...

Sicheng Wen, MD, PhD

Amy Gaynor Research Fund

Aplastic anemia is a potentially life-threatening bone marrow failure in which the body stops producing enough new blood cells. Marrow transplantation, antithymocyte globulin (ATG) treatment and other approaches can vary in effectiveness in approaching marrow aplasia. Extracellular vesicles are small particles released from cells that were initially thought to present cell “junk”, and later evaluated to be involved in cell-to-cell communication and affect the phenotype of target cells. In this study, we will evaluate the capacity of mesenchymal stem cell derived-extracellular vesicles (MSC-EV) to reverse aplasia in a murine model of aplastic anemia and identify the MSC-EV associated miRNA responsible for the reversal of aplastic anemia. This AA&MDSIF research grant will give us an...

Britta Will, PhD

Harold Spielberg Research Fund

Myelodysplastic syndromes (MDS) are the most common hematologic malignancies in the elderly, presenting as bone marrow failure and characterized by disorderly growth and differentiation of aberrant hematopoietic stem and progenitor cells. Bone marrow transplantation is currently the only curative option for MDS. Even though 5-azacitidine (5-aza) has resulted in clinical responses and improvements in overall survival, relapse and refractory disease continue to occur in some patients. Recent findings show that cancer-initiating cells (CIC) can exist as pools of relatively quiescent cells that do not respond well to common cell-toxic agents and contribute to treatment failure. In fact, we and others have shown that karyotypically abnormal hematopoietic stem cells (HSCs) can survive during...

Grant Year: 2014

Daria Babushok, MD, PhD

Sam Jordan Research Fund, Stephen and Joanna Janowiak Research Fund, Torry Yahn Research Fund

Aplastic anemia (AA) is a devastating blood disorder, affecting children and adults, caused by immune attack on the bone marrow. Factors that determine recovery, relapse, and transformation to myelodysplasia in aAA remain poorly defined, and there is a dearth of studies in pediatric patients. Emerging data suggest that mutations in the bone marrow and in the pathogenic immune cells contribute to disease evolution and relapse. To study this, I will employ cutting-edge genomic techniques to characterize pathogenic mutations in aAA. These studies will improve treatment of patients with pediatric AA by allowing early detection of clonal evolution and disease progression.

Luis Batista, PhD

Bradley Richard Research Fund, Research is Hope Fund

Bone marrow failure is a serious condition that occurs when the bone marrow stops making enough healthy blood cells. A risk for bone marrow failure is genetic instability, including exacerbated shortening of telomeres (repetitive DNA sequences that cap chromosome ends). Using different genetic and biochemical approaches, this proposal will use cells derived from bone marrow failure patients that have telomere attrition as a platform for the development of clinical therapies against this disease. These experiments will increase our knowledge on stem cell function and regulation in bone marrow failure syndromes.

Rosannah Cameron, PhD

Lindsay Minelli Research Fund, Research is Hope Fund

We do not yet fully understand the process of disease progression in MDS. We know certain genes are involved because mutations in those genes have been found in MDS patients. We need to understand which genes are important early in the disease and which ones act later. Our goal is to identify the genes that act early in MDS, so that ultimately, our research will lead to new treatment options that are more specific and effective.

Youmna Kfoury, PhD

Lisa Lancaster Research Fund

Myelodysplastic Syndromes (MDS) are a group of diverse and incurable pre-leukemic disorders. Even though a deregulated bone marrow microenvironment is thought to participate in the disease, its role remains elusive. Since the microenvironment is better understood in mice, we have identified a subset of cells in the human bone marrow that is equivalent to those cells in mice which, when perturbed, lead to MDS. We propose to characterize these cells in patient samples at the cellular and molecular level. This will provide a better understanding of the abnormal signaling pathways involved in MDS, and eventually identify therapeutic approaches.

Patrizia Ricci, PhD

PNH Research and Support Foundation

PNH is a rare hematological disease characterized by spontaneous destruction of red blood cells (intravascular hemolysis), poor functioning of bone marrow, and thrombosis. Recently, the availability of the complement inhibitor eculizumab has dramatically improved the treatment of PNH. Nevertheless, about half of the patients treated with eculizumab shows a persistence of clinical signs of the disease. We have recently described the reasons underlying the limited efficacy of current anti-complement treatment for PNH. With this proposal we aim to complete the pre-clinical development of a novel complement inhibitor which targets early events in complement activation, the component-3 of the complement cascade.

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Grant Recipients

Grant Year: 2015

Grant Year: 2014