CAF Funds Medical Research to Advance Fight Against Thalassemia

July 12, 2010 – The Cooley’s Anemia Foundation has awarded $329,000 in medical research grants and fellowships for the 2010-2011 award period.

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

The $329,000 represents one new first year fellowship; renewals of five second-year fellowships; funding for two medical research grants; and renewal of the second year of the gene therapy grant.

Applications for 2011-2012 Medical Research Fellowships and RFAs will be available soon. Contact to receive applications when they become available.

The new first year fellowships, funded at a level of $32,000, has been awarded to Jackie Eun-Ah Lim, PhD, of Duke University Medical Center and Lihong Shi, PhD, of the University of Michigan Medical School.

  • Dr. Lim’s project is entitled “Identifying genetic modifiers of iron stores in mice” and focuses on identifying genes that modulate aspects of iron absorption, utilization, recycling and storage. Identifying these genes will help to develop potential targets for managing the iron overload that accompanies thalassemia.

The renewals of five second-year fellowships, funded at a level of $32,000 each, were awarded to the following:

  • Paula Goodman, Fraenkel, MD, of Beth Israel Deaconess Medical Center, for the project “Modifiers of hepcidin expression as potential therapies for beta-thalassemia related iron overload.” Hepcidin is a hormone that helps to regulate iron absorption. Dr. Fraenkel’s research will investigate ways to modify hepcidin expression with the goal of helping to prevent and treat iron overload in thalassemia.
  • Wenjie Chen, PhD, of Duke University Medical Center, for the project “The Role of Hemojuvelin in Heart and Muscle.” Hemojuvelin is a protein that plays a role in hepcidin expression. This study will examine if hemojuvelin in the heart and muscle can play a role in regulating hepcidin, which would add to knowledge that can be used to treat iron overload.
  • Sara Gardenghi, PhD, of Weill Cornell Medical College, for the project “Increased Hepcidin expression in mice affected by beta-thalassemia reduces iron overload with no effect on anemia.” As with the previous two studies, Dr. Gardenghi’s focuses on the role of hepcidin in regulating iron absorption and seeks to discover more information that can be used to treat excess iron in people with thalassemia.
  • Rebecca J. Abergel, PhD, of Lawrence Berkeley National Laboratory, for the project “Design, Evaluation and Development of Orally Active Multidentate Iron Chelators.” Dr. Gardenghi’s project will help to identify compounds that have the potential to be developed into new treatments for removing excess iron from the blood. While there are currently two FDA-approved chelating drugs available in the U.S., more are needed in order to ensure that each individual patient is able to receive treatment that is best suited to his/her specific needs.
  • Daniel Strongin, PhD, of Fred Hutchinson Cancer Research Center, for the project “A new strategy to ensure robust gene expression from gene therapy vectors by positioning the transgene within the nucleus.” Put in the simplest terms, this study focuses on the role that a vector (the means by which a corrected gene is introduced into a patient’s cells) plays and how it can be modified to help ensure that the new, corrected gene will reproduce in an appropriate way. Gene therapy holds great promise in terms of potentially providing a cure for thalassemia in the future.


Funding was also designated for CAF’s Clinical RFA and Gene Therapy programs.

  • Ellen Fung, PhD, RD, of Children’s Hospital & Research Center at Oakland, received continuation funding for her first year Clinical RFAgrant for “Good Vibrations: A pilot study of vibration therapy to improve bone health in patients with thalassemia induced osteoporosis.” Many people with thalassemia suffer from a form of osteoporosis (weak bones), a condition that is more commonly found in people of more advanced age than the typical thalassemia patient. This study will evaluate the effect of “vibration therapy” on bone density, size and strength among a small group of people with thalassemia. This funding is in the amount of $30,000 and represents the second half of her first year funding ($60,000).
  • Sylvia Titi Singer, MD, of Children’s Hospital & Research Center at Oakland received funding for the first year of her medial research project “Determination of Reproductive State and Relation to Pituitary Iron Overload in Thalassemia Major Patients.” This study 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.
  • Derek Persons, MD, PhD, of St. Jude Children’s Research Hospital received renewal funding of $75,000 for the second year of his gene therapy project, “Translations Research in Adult Thalassemia.” This project also focuses on the role of the vector in gene therapy. Dr. Persons’ work seeks to develop a breakthrough in therapeutic vector production that will increase the probability of success in a human gene therapy clinical trial.


Ellis Neufeld, Chair of the CAF Medical Advisory Board, said of these projects, “Once again, the generous donors to CAF are helping researchers perform cutting-edge research into the biology of thalassemia, iron overload, globin gene regulation and gene therapy. The Fellowship grants support young investigators still at the training stage or junior faculty stage of their careers, while the Gene Therapy and Clinical RFA program support physicians or scientists at any stage of their careers who address problems of vital interest to CAF and our patients.”

CAF is thrilled with the level of expertise demonstrated by our fellowship and grant recipients and looks forward to the contributions that their research will make in the fight in against thalassemia.

Thalassemia Treatment Centers

A list of major thalassemia treatment centers can be found by clicking here, and a brochure about thalassemia treatment centers can be downloaded by clicking here.

Other centers are affiliated with these centers. If you need more information about thalassemia treatment centers, contact the centers on the list, or call CAF Patient Services at (212) 279-8090.

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