Pioneering Stem Cell Therapy Cures Infants with Bubble Baby Disease

March 29, 2017

(Medical Xpress) – In a phase 2 clinical trial led by Dr. Donald Kohn of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, all nine babies were cured. A 10th trial participant was a teenager at the time of treatment and showed no signs of immune system recovery. Kohn’s treatment method, a stem cell gene therapy that safely restores immune systems in babies with the immunodeficiency using the child’s own cells, has cured 30 out of 30 babies during the course of several clinical trials.