AA&MDSIF Grant Recipients

The Aplastic Anemia & MDS International Foundation offers research grants to new and established investigators in acquired aplastic anemia, myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH).  Grants awarded since 2006 include:

2011 - 2013

• AA&MDSIF Torry Yahn Research Fund to Kim-Hien T. Dao, D.O., Ph.D, Oregon Health & Science University for Beta-catenin is a molecular target of the Fanconi anemia core complex.  Dr. Dao will investigate why the blood stem cells from patients who inherit a loss-of-function mutation in one of the genes of the Fanconi anemia pathway are highly susceptible to cell death yet also highly susceptible to conversion into an acute leukemia.
• AA&MDSIF PNH Research and Support Foundation to Keith R. McCrae, MD, Cleveland Clinic for Circulating microparticles in PNH.  Dr. McCrae's study explores why patients with PNH are at increased risk for the development of thrombosis, or blood clots, that may affect arteries or veins and cause events such as pulmonary emboli or stroke. 
• AA&MDSIF Trinity Ewert Research Fund to Parinda Mehta, MD, Cincinnati Children's Hospital Medical Center for Quercetin in patients with Fanconi Anemia, a pilot study. Dr. Mehta will study the use of Quercetin, a naturally occurring flavonoid (antioxidant) in patients with Fanconi anemia, one of the most common congenital bone marrow failure syndromes.  She anticipates that the results of this pilot study will show that long term oral Quercetin therapy is feasible and well tolerated in patients with Fanconi anemia. These results will form the basis of the continuation phase of the study, which will demonstrate that Quercetin therapy in fact delays or prevents progressive marrow failure in children with Fanconi anemia.
• AA&MDSIF Harold Spielberg Research Fund to Mridul Mukherji, PhD, University of Missouri - Kansas City for Rescue of TET2 Mutations from MDS Patients. TET2 is one of the most frequently mutated genes identified in patients with myelodysplastic syndromes (MDS).  This study will help explore new avenues for the cure TET2 mutations,  and Dr. Mukherji says he and his colleagues “will use our expertise to develop strategies to restore TET2 activity for the treatment of MDS.”
  
  

2010 - 2012

• AA&MDSIF Research Grant including support from the Madden family in memory of Mary-Pat Madden Greishaber, and the MacGillivray family in memory of Erwin Umbach to Gregory A. Abel, MD, Dana-Farber Cancer Institute for Developing a Disease-Specific Measure for Quality of Life in Patients with Myelodysplastic Syndrome (MDS). Dr. Abel and his team aim to develop an MDS-specific quality of life instrument through focus groups with patients and providers and to further refine the instrument by piloting it with MDS patients.
• AA&MDSIF Emily Kass Research Grant to Christian Bellodi, PhD, University of California, San Francisco for p53 Translation Control in Hematopoietic Stem Cell Quiescence and Differentiation.  Dr. Bellodi aims to understand how impairments in the DKC1 gene alter hematopoietic stem cells' (HSC) behavior and to what extent this defect contributes to bone marrow failure. 
• AA&MDSIF Harold Spielberg Research Grant to Muneoshi Futami, MD, Northwestern University for The Mechanism for Myelodysplasia in Patients with Loss of Chromosome 7. Dr. Futami has developed two special cell lines that display abnormal proliferation and defective maturation that characterize MDS cells. He will use these two cell lines to identify the biochemical changes that make the monosomy MDS cells different from normal blood cells and will develop a mouse model to study the biochemical irregularities associated with monosomy 7. 
• AA&MDSIF Torry Yahn Research Grant to Ramon Tiu, MD, Cleveland Clinic for LFA-3/CD2 Pathway: Potential Target for Immunosuppressive Therapy in Aplastic Anemia.  Dr. Tiu will conduct a clinical trial of a new drug, Alefacept® in patients with aplastic anemia in whom the traditional treatments did not work. This trial will determine whether Alefacept® is effective and well tolerated.

2009 - 2011

• AA&MDSIF PNH Foundation Research Grant to Kazuhiko Ikeda, MD, PhD, Department of Hematology, Washington University School of Medicine, St. Louis, Missouri The Mechanism of Clonal Dominance of PNH Cells: Dr. Ikeda will be studying how PNH blood cells acquire a "growth advantage" through a gene mutation. His findings are likely to provide new insights into the role of gene expression in clonal blood disorders such as PNH and MDS.
• AA&MDSIF PNH Foundation Research Grant to Regis Peffault de Latour, MD, PhD, Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland The Role of Unfolded Protein Response in Paroxysmal Nocturnal Hemogloburnia (PNH): Dr. Peffault de Latour's study will examine the role of the Unfolded Protein Response (UPR) in the development of PNH cells. Understanding how the UPR allows for cell adaptation instead of cell self-destruction could lead to the discovery of new therapeutic strategies which target this pathway.
• AA&MDSIF Harold Spielberg Research Grant to Archibald Perkins, MD, PhD, Department of Pathology and Lab Medicine, University of Rochester, Rochester, New York Development of Targeted Therapies for 3q26-positive MDS: This study by Dr. Perkins will focus on a particular protein function believed to cause gene overexpression leading to MDS. Targeting this protein area may lead to the development of a drug, called a polyamide, which represents a new class of agent that has never been tried in the treatment of MDS.

2008 – 2010

• Torry Yahn Research Study awarded to Lisa Minter, PhD of the University of Massachusetts-Amherst, who lost a young daughter to aplastic anemia and knows all too well the importance of research in this field.  She has been awarded a grant to study Evaluating PKC-0 as a Therapeutic Target in a Mouse Model of Severe Aplastic Anemia.
• Harold Spielberg Research Grant awarded to Benjamin Braun, MD, PhD of the University of California San Francisco for Mechanisms and Therapy of Anemia Caused by Activating K-ras Mutuation. 
• Thanks to the generosity of several major donors, Jaroslaw Maciejewski, MD, PhD of the Cleveland Clinic received a grant to study Identification of Mutations in C-Cbl as Pathogenetic Factors in Patients with Myelodysplastic Syndrome.
• PNH Research and Support Foundation Research Study awarded to Antonio Maria Risitano, MD, PhD, University of Naples, Italy for Genetic Fingerprint of Complement and Complement-related Genes in PNH:  relationship with pathophysiology, clinical manifestations (including thrombosis) and response to eculizamab.

2007 – 2009

• Trinity Ewert Research Grant was awarded to Hiromi Gunshin, MD, PhD at the University of Massachusetts Amherst for Studies Toward Alternative Therapies for Iron Overload Patients with Aplastic Anemia.
• Harold Spielberg Research Grant was awarded to Lubomir Sokol, MD, PhD, H. Lee Moffitt Cancer Center, Tampa, Florida for Microarray Profiling of Micro RNA in 5q-Syndrome. 
• MacGillivray Research Grant awarded to Kay Macleod, PhD, at The Ben May Institute for Cancer Research, University of Chicago for Oxidative Stress in the Etiology of Myelodysplasia

2006 – 2008

• Marissa Marie Amuso Research Study & Jack Byrne Research Fund awarded to Lisa M. Minter, PhD, University of Massachusetts, Amherst for Gamma-secretase Inhibitors as Therapeutic Intervention in Bone Marrow Failure Syndromes.(*Also supported by the Torry Yahn Research Fund)
• Holly Cataldo Research Study awarded to Hinh Ly, PhD, Emory University, Pathology & Laboratory Medical Department for Telomere Maintenance in Patients with Aplastic Anemia (* Also supported by the Torry Yahn Research Fund.)
• Earl J. Goldberg Aplastic Anemia Foundation Research Study awarded to Seth J. Corey, MD, UT-MD Anderson Cancer Center for P13 - kinase Dysregulation in Myelodysplastic Syndromes 
• Lindsay Minelli Research Study awarded to Christine O'Keefe, Ph.D., Cleveland Clinic Tuassig Cancer Center for Genome Stability in MDS. (*Also supported by the Harold Spielberg Research Fund)
• PNH Research and Support Foundation Study awarded to Lukasz P. Gondek, MD, Cleveland Clinic Taussig Cancer Center for A Novel Approach for the Study of Genetic Predisposition in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria in Using High-density Arrays.