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FDA Approves Trial of Stem Cell-Based Gene Therapy for Blood Disease

Dr. Michel Sadelain, M.D., Ph.D., at Memorial Sloan-Kettering Cancer Center has been working for two decades on a gene-therapy based cure using stem cells for the blood disease β-thalassemia. The work of his team has now culminated in an FDA approved clinical trial, the first therapy of this kind for a globin disorder in the United States.  β-thalassemia affects the ability of a person’s blood to carry oxygen. It occurs primarily in people of Mediterranean, Asian and African descent as a result of mutations in the β-globin gene. There are over 10,000 patients with severe globin disorders (mostly thalassemia and sickle cell disease) in New York State who require regular blood transfusions. Dr. Sadelain’s team has developed a method to isolate the blood stem cells of patients and insert a normal copy of the β-globin gene. Once the stem cells are returned to the patients, they produce functional β-globin, restoring the ability of the blood to bind oxygen. Dr. Sadelain is working with Drs. Isabelle Rivière, Ph.D., Director of the Cell Therapy and Cell Engineering Facility, and Farid Boulad, M.D., to produce the corrected cells and treat the patients. This approach also holds potential as a cure for other blood disorders such as sickle cell anemia.

Dr. Sadelain receives NYSTEM funding through contract C024412 to explore the possibility of using induced pluripotent stem cells rather than blood stem cells for this treatment.

Information on this clinical trial can be found at