Gene Therapy Successful in Treating Immune Disease

Several recent studies have proven gene therapy is successful in treating immune diseases. In a recent study, two young children with Wiskott-Aldrich syndrome (WAS) showed marked clinical improvements after receiving gene therapy. The study, performed by Jordan S. Orange, MD, PhD, an immunologist at The Children’s Hospital of Philadelphia, in collaboration with European gene therapy researchers, reported that two 3-year-old boys diagnosed with WAS soon after birth were treated by first collecting some of their hematopoietic (blood cell-forming) stem cells, then transferring normal WAS genes into those cells and returning the cells to the boys’ bloodstreams. After treatment, the patients experienced fewer and less severe infections, bleeding episodes decreased after platelet counts improved, severe autoimmune anemia disappeared in one boy, and severe eczema completely resolved in the other. Three years after the gene therapy, the clinical benefits persisted.

This study is the latest example of clinical success for gene therapy. In 2009, researchers from The Children’s Hospital of Philadelphia and the University of Pennsylvania reported dramatic vision improvements in patients with Leber congenital amaurosis, a form of inherited blindness. In the same year, Parisian researchers announced success in treating adrenoleukodystrophy, the disease depicted in the movie “Lorenzo’s Oil.” And, scientists at the University of California are reporting preliminary clinical benefits of gene therapy for adenosine deaminase deficiency, an immune deficiency disorder related to “bubble boy disease.”