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. Last year the work of his group resulted in the first FDA approved clinical trial testing a stem cell-based treatment for β-thalassemia. β-thalassemia is caused by mutations in the β-globin gene. Dr. Sadelain’s team developed a method to isolate blood stem cells from β-thalassemia patients, insert a functional copy of the β-globin gene, and transfuse the cells back into the patients. This approach also has potential as a cure for other blood disorders. In recognition of his accomplishment, Dr. Sadelain received a 2013 Sultan Bin Khalifa International Thalassemia Award.
Dr. Sadelain received NYSTEM funding through contract C024412 to explore using induced pluripotent stem cells rather than blood stem cells for this treatment, and is part of a NYSTEM Consortium award to develop an ESC-based therapy for Parkinson's disease, contract C028503.