Erica B. Esrick, MD, Hematologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center reported on her pilot trial in which one adult patient with Sickle Cell disease (SCD) received an infusion of his own stem cells in which a genetic “switch” was flipped on to induce the cells to both start producing healthy hemoglobin and stop producing unhealthy “sickle” hemoglobin. This first-in-human pilot study provides a proof of principle for this novel approach to gene therapy for SCD.
Currently, the only established cure for SCD is a transplant of healthy stem cells from a matched sibling donor. However, many patients with SCD do not have a suitable sibling donor and sometimes stem cell transplants may fail. Gene therapy is an alternative approach that uses the patient’s own stem cells and does not rely on the availability of a compatible donor.