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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.

View a single grant year.

Grant Year: 2017

Sergei Vatolin, PhD

Patricia and Vincent Geczik Legacy Fund

Medicines available to the patients with severe blood diseases like aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are very limited. The idea of improvement the quality of blood cells with small molecules may give a new opportunity to cure AA and hope to reverse PNH. Our proposal is based on discovery of drugs (TCC1 and TCC2) which improve performance of blood stem cells by increasing their life span. We hypothesize that the unique biological activity of these agents can already be used in clinic to treat AA and PNH by restoration the function of damaged/exhausted blood stem cells. Such effects may help to alleviate diseases and accelerate recovery of normal blood in patients with AA or PNH. The objective of this study is preclinical testing of the identified drugs to...

Grant Year: 2016

Akihide Yoshimi, MD, PhD

Shirley and James O’Brian Research Fund

Myelodysplastic syndromes (MDS) are a heterogeneous group of disorders characterized by inefficient blood production. MDS represents the most common cause of acquired bone marrow failure in adults and there are few effective therapies for the majority of MDS patients. In 2011, it was discovered that mutations in proteins encoding RNA splicing factors (SFs) are the most common class of mutations in patients with MDS and chronic myelomonocytic leukemia (CMML). Despite these discoveries, however, we do not yet fully understand why abnormal RNA splicing results in MDS nor do we have therapies that specifically target MDS cells bearing this common class of mutations. The goals of this proposal are two-fold: (1) to determine the efficacy of clinical-grade novel spliceosome inhibitors in...

Tushar Bhagat, PhD

Carol and Peter Stewart Fund in memory of Raymond Hodor

Myelodysplastic syndromes (MDS) are a heterogeneous set of clonal disorders characterized by ineffective blood cell development. These diseases are driven by many complex genetic and non-genetic changes. Recent findings have uncovered that alterations in the bone marrow microenvironment contribute to the disease. In our preliminary data, we have found that the beta-catenin pathway is activated in the bone marrow and in the blood of patients with MDS and this predicts a poor clinical outcome. We demonstrate that changes in the stroma increase beta-catenin in the MDS cells and can be targeted by new drugs. We will utilize mouse models, cells lines and patient samples to understand how the bone marrow environment is altered in patients and utilize newly developed beta-catenin pathway...

Alina Dulau Florea, MD

Patricia and Vincent Geczik Legacy Fund

Summary Our study involves the analysis of normal and abnormal cells in the bone marrow of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). In this condition, due to a gene mutation, some bone marrow cells and their progeny in the blood lack an important cell surface component that functions as an anchor for other proteins, some of which attach to blood cells and protect them from destruction by complement, part our immune system. The result of this deficiency is a breakdown of red cells. If large quantities of red cells are destroyed, people become very tired, or have pain in the belly, head or when swallowing, or develop blood clots. The cause of the gene mutation is unknown. Some patients with other conditions, such as aplastic anemia (AA, a disease where marrow stops...

Kate MacNamara, PhD

Julia Malsin Research Fund

Bone marrow failure is the rare but devastating collapse of blood production, and if left untreated the disease is invariably fatal. Our current therapies are inadequate in the sense that they involve general immunosuppression or highly invasive treatments via bone marrow transplantation. In both genetic and acquired cases of bone marrow failure, excessive inflammation ultimately causes destruction of the stem cells required to maintain daily production of all blood cells. Inflammatory molecules such as interferon gamma (IFN-gamma) are known to contribute to pathology, yet exactly how stem cell function is compromised by these factors is not clear and remains an important question in understanding the pathogenesis of bone marrow failure. We utilized a mouse model of bone marrow failure...

Grant Year: 2015

Jing Fang, MD, PhD

Michael Fernandes Research Fund

A major complication of myelodysplastic syndromes (MDS) is worsening cytopenias due to bone marrow failure. Novel therapeutic approaches are urgently needed for MDS, as current therapies have modest responses and many patients are not suitable candidates for bone marrow transplantation. Our preliminary study revealed a role of sequestosome 1/p62 in the pathogenesis and disease evolution of MDS. For this study, we will dissect the functional domains on p62 and associated signaling pathways that are necessary for the development and maintenance of MDS. Moreover, we propose to target p62-intacting complexes using a peptide approach as an attempt to target MDS clones and prevent disease evolution. This study will improve our understanding of MDS-associated bone marrow failure and may reveal...

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...
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