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December 11, 2014 – Exciting data from studies looking at new treatments for thalassemia presented at the annual meeting of the American Society of Hematology builds upon science supported by the Cooley’s Anemia Foundation and gives hope for the development of significant new treatments for the genetic blood disorder known as thalassemia. Two of the trials, known as the Northstar Study and HGB-205 respectively, focus on gene therapy as a curative approach; a third trial looked at a compound known as Luspatercept, which shows promise as a means of raising hemoglobin levels in patients with thalassemia.
People born with a severe form of thalassemia require lifelong transfusions as often as every two weeks. Finding a way to eliminate these transfusions is a major goal of the Cooley’s Anemia Foundation, the only national non-profit in the U.S. dedicated solely to thalassemia.
The two gene therapy trial studies reported on preliminary data from ongoing clinical trials utilizing bluebird bio’s LentiGlobin BB305 drug product. Data presented by Alexis A. Thompson, M.D., M.P.H., professor of pediatrics at Northwestern University Feinberg School of Medicine, director of the Comprehensive Thalassemia Program at Ann & Robert H. Lurie Children’s Hospital of Chicago and lead investigator of the Northstar Study, focused on two beta thalassemia patients who have undergone the experimental treatment. One patient has remained transfusion-free for five months and the other for three.
Data from the HGB-205 trial also reported that two beta thalassemia patients in this study have remained transfusion-free for twelve and nine months respectively. Both studies are open-label Phase1/2 studies.
The process in these trials involves inserting a fully functional human beta-globin gene into a patient’s own hematopoietic stem cellsand then transplanting those modified cells back into the patient. The science behind this process has been studied in several projects funded by the Cooley’s Anemia Foundation.
“The Cooley’s Anemia Foundation has long been a leader in funding investigational approaches to gene-based therapies as a potential curative treatment for thalassemia,” says Anthony J. Viola, National President of the Cooley’s Anemia Foundation. “Previous studies have also suggested that gene therapy or gene editing may be a viable path to a new curative approach to thalassemia; we are excited that this latest data continues to support that approach. Along with continued advances in the area of traditional bone marrow transplantation, the Foundation believes that the day of a universal cure for thalassemia is on the horizon.”
Antonio G. Piga, MD, of the Azienda Ospedaliero-Universitaria San Luigi Gonzaga in Turin, Italy, presented data from Acceleron Pharma’s ongoing clinical trial investigating Luspatercept, a protein that aims at increasing the production of mature red blood cells. The data presented from this Phase 2 trial showed an increase in hemoglobin levels and a decrease in transfusion requirements in adult beta-thalassemia patients participating in the study. The trial includes both transfusion-dependent and non-transfusion-dependent thalassemia patients; Transfusion-dependent patients experienced a greater than 60% reduction in their transfusion requirements. These patients also showed a 12-60% decrease in serum ferritin levels and reductions in liver iron concentration. Patients who were non-transfusion-dependent showed an increase in hemoglobin levels as well as reductions in liver iron concentration.
“For decades, the Cooley’s Anemia Foundation has been interested in science which focuses on increasing hemoglobin levels in our patients,” Viola states. “Clearly, reducing the need for transfusions has the potential to significantly increase quality of life for people with thalassemia while simultaneously reducing the risk of serious complications related to iron overload. The Foundation is encouraged that our support of this pathway is being developed in a meaningful way.”