St. Jude Gene Therapy Cures Babies with ‘Bubble Boy’ Disease
Infants with X-linked severe combined immunodeficiency have fully functioning immune systems following treatment with gene therapy developed and produced at St. Jude Children’s Research Hospital.
Gene therapy developed at St. Jude Children’s Research Hospital has cured infants born with X-linked severe combined immunodeficiency (SCID-X1). The children are producing functional immune cells, including T cells, B cells and natural killer (NK) cells, for the first time. The results appear in the April 18 issue of the New England Journal of Medicine.
“These patients are toddlers now, who are responding to vaccinations and have immune systems to make all immune cells they need for protection from infections as they explore the world and live normal lives. This is a first for patients with SCID-X1,” said first and corresponding author Ewelina Mamcarz, M.D., of the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy.
The patients were treated at St. Jude and UCSF Benioff Children’s Hospital San Francisco with gene therapy produced in the Children’s GMP, LLC, a Good Manufacturing Practice facility located on the St. Jude campus. The children were enrolled in an ongoing clinical trial of infants newly diagnosed with SCID-X1, the most common type of SCID, a rare, life-threatening genetic disorder that is sometimes called “bubble boy” disease. The name is a reference to measures taken to protect patients, who are born with little or no immune protection. Untreated, patients usually die early in life. Read More »