Doctors Find Cure for SCID Through New HIV-Based Gene Therapy

Doctors are using HIV to cure infants born without immune systems.    

One in 58,000 babies is born with SCID, or “Bubble Boy disease,” in the U.S. each year. Ja’Ceon Hawkins was born with SCID. Severe combined immunodeficiency, is a group of rare disorders that affect the development of immune cells. Without these infection-fighting immune cells, children with SCID can die from even a common cold. The cells usually develop in bone marrow within the first two years of a child’s life.  

In the past, infants depended on blood transfusions, bone marrow transplants, or gene therapy for survival. Doctors at St. Jude Children’s Hospital harvested SCID cells and inserted a healthy copy of the gene needed to restore the immune system. Doctors the infused the cells back into the patient’s bodies.

“Three to four months post the infusion not only was he able to go home and, you know, start to live a relatively normal life, but we were able to take him off all complete isolation and all prophylactic medication,” pediatrician Dr. Morton Cowan explained.

Doctors used gene therapy to treat eight infants, which took 10 days. They used a copy of the HIV virus as a delivery system for the healthy genes. The active and infectious parts of the virus were removed in this copy.