Gene therapy trial offers hope for Harry

Harry and his father at Dana/Farber-Boston Children’s In their Brookline home-away-from-home, 2-year-old Duy Anh “Harry” Le plays with blocks and pop-up toys on the floor with his mother, Thao Nguyen. He is lively and happy, and his skin is clear. He looks almost nothing like the sickly baby covered in eczema who arrived in Boston from his native Vietnam in November of 2016 to participate in a gene therapy clinical trial for Wiskott-Aldrich syndrome. Children with the potentially life-threatening, inherited immune system deficiency have difficulty producing platelets, the blood component that fosters clotting, which leaves them susceptible to bleeding. Their compromised immune systems leave them susceptible to infections, eczema, autoimmunity and cancer. The disorder is so rare that Harry’s physician father, Duy Le, an adult ear, nose and throat specialist, had not heard of it. It is so rare that many doctors have trouble diagnosing it. Harry in Vietnam When Harry, at two months, exhibited bleeding spots on his legs, doctors in Vietnam suspected dengue fever. Then the baby developed eczema and pneumonia-like infections, so physicians suspected idiopathic thrombocytopenic purpura and treated him for the autoimmune disorder for four months. Finally, at the age of six months, Harry was diagnosed with WAS. He needed a stem cell transplant, but Vietnam has little experience with pediatric transplants and Harry didn’t have a matched donor. “The first few months afte...
Source: Thrive, Children's Hospital Boston - Category: Pediatrics Authors: Tags: Diseases & Conditions Our Patients’ Stories Dana-Farber/Boston Children's Cancer and Blood Disorders Center Dr. Sung-Yun Pai gene therapy Wiskott-Aldrich syndrome Source Type: news