A team of researchers from UCLA, led by Donald B. Kohn, MD, has developed a stem cell gene therapy that has restored the immune system function of all 23 ADA-deficient SCID patients involved in the experimental trial. ADA-deficient SCID (adenosine deaminase–deficient severe combined immunodeficiency) is a life-threatening condition that affects infants, preventing them from manufacturing the immune responses needed to fend off illness. Babies with this condition rarely live past their first year of life.
Before Dr. Kohn’s experimental therapy, there were only two options for treatment: (1) twice-weekly ADA injections, which are very costly and require a lifelong commitment, and (2) bone marrow transplant, which is only viable with a very close match from a sibling, but still has a high chance of rejection.
“Using a virus delivery system that he developed in his lab in the 1990s, Kohn inserted the corrected gene that produces the missing enzyme into the blood, forming stem cells in the bone marrow. The genetically corrected blood-forming stem cells then produced T cells capable of fighting infection.”