Medical Research Receives Boost from CAF

August 2, 2012 – The Cooley’s Anemia Foundation is allocating $317,500 in medical research funding this year to a range of important and exciting projects. This funding represents one new and two renewed CAF Medical Fellowships; two new and two delayed clinical research grants; and a new gene therapy research grant.

Funding medical research has long been one of CAF’s primary objectives, and the Foundation is proud to have made and to continue to make significant contributions to the understanding and treatment of thalassemia through its research funding.

So what is CAF funding this year?

CAF Gene Therapy Grant

CAF’s Gene Therapy Grant is funded at a level of $60,000 per fellowship per year. Gene Therapy Grants are awarded for one with the option of renewal for a second year, pending satisfactory review of first year progress. The purpose of the Gene Therapy Grants is to speed the application of recent translational advances in gene therapy to clinical trials.

Michel Sadelain, MD, PhD of Memorial Sloan-Kettering, has received funding this year for his project, “A Phase I Clinical Trial of Lentiviral Mediated ß-globin Gene Transfer in Patients with Cooley’s Anemia.” This phase I trial will assess the safety of Dr. Sadelain’s gene therapy procedure, as well as measure the differences in the hemoglobin produced by patients before and after the procedure. This is a very important step in the creation of a potential gene therapy-based cure for thalassemia. (To read more about this trial, click here.)

CAF Clinical Research Grants

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CAF’s Clinical Research Grants are funded at a level of $40,000 per fellowship per year. Clinical Research Grants are awarded for one with the option of renewal for a second year, pending satisfactory review of first year progress. The purpose of the Clinical Research Grants is to support investigators from all disciplines and backgrounds (MD, RN, PhD, MPH, MSW or other disciplines) with their ongoing clinical projects to address one or more of the following areas impacting patients with thalassemia, including but not limited to: fertility, pregnancy and family planning; quality of life/psychosocial; burden of disease including cardiac issues and iron overload.

Projects by Ellen Fung, PhD, RD, CCD, of Children’s Hospital & Research Center at Oakland, and Janet Kwiatkowski, MD, MSCE, of Children’s Hospital of Philadelphia, received first year funding this year.

Dr. Fung’s project, entitled “Iron Overload, Zinc Status and Glucose Regulation in Patients with Thalassemia,” works from the hypothesis that in patients with thalassemia, zinc deficiency affects glucose regulation, and that regulation can therefore be improved by zinc zupplementation.

Dr. Kwiatkowski’s project, entitled “Pilot Study of Deferasirox and Deferiprone Combination Oral Chelation for Individuals with Transfusion Dependent Thalassemia and High Iron Burden,” will study the safety of the combined use of the two currently available oral chelators in patients who are heavily iron loaded.

Projects submitted by Maria Vogiatzi, MD, of Weill Medical College of Cornell University, and Sylvia Titi Singer, MD, of Children’s Hospital & Research Center at Oakland, were awarded second year funding.

Dr. Vogiatzi’s study is entitled “The effect of vitamin D supplementation on calcium excretion in thalassemia: a dose response study.” It will look at different dosing levels of vitamin D in thalassemia in order to gain more knowledge concerning the appropriate level for use in the patient population.

Dr. Singer’s study, “Determination of Reproductive State and Relation to Pituitary Iron Overload in Thalassemia Major Patients,” will examine 30 patients to determine how fertility measures may correspond to iron deposition, helping to increase our understanding of this relationship and to provide information that ultimately may lead to strategies to better preserve fertility in thalassemia.

CAF Medical Fellowships

CAF’s Medical fellowships are funded at a level of $32,500 per fellowship per year. Fellowships are awarded for one with the option of renewal for a second year, pending satisfactory review of first year progress.

This year, a first year CAF Medical Fellowship has been awarded to Laurie Steiner, MD of the University of Rochester. Dr. Steiner’s project, entitled “Detailed Genomic and Functional Analysis of the ß-Thalassemia Modifer Genes BCL11a and HSB1L-MYB,” will look at genetic elements that seem to have an effect on the severity of thalassemia in different individuals.

Second year renewals of CAF Medical Fellowships have been awarded to Sebastiaan van Zalen, PhD, of Children’s Hospital of Philadelphia, and Caiyong Chen, PhD, of Brigham and Women’s Hospital.

Dr. van Zalen’s project is entitled “Cytoplasmic factors that stabilize ß-globin mRNA.” This study will help to characterize the major cellular and molecular mechanisms that are important in controlling the expression of ß-globin mRNA.

Dr. Chen’s project will look at “Iron and heme trafficking in red cells” to develop our understanding of how iron is transported across membranes into cells and, from there, how it is transported across internal membranes of cells. This knowledge is helpful in developing future therapies for iron overload.

CAF congratulates the excellent studies submitted by all of our recipients and looks forward to the knowledge that these studies will add to our understanding of thalassemia.

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