A stem cell gene therapy developed at University of California – Los Angeles (UCLA) shows effective for babies with bubble baby disease (or ADA-SCID), in phase 2 clinical trials.
Adenosine deaminase-deficiency (ADA) is a rare life-threatening inherited condition that damages the immune system and causes severe combined immunodeficiency (SCID). People with SCID lack virtually all immune protection from bacteria, viruses, and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. Babies who born with this disease can die in the first year of life when untreated. Children with the disease must remain isolated in clean and germ-free environments to avoid exposure to viruses and bacteria; even a minor cold could prove fatal.
According to the results, all 9 babies who participated in the latest clinical trial were cured. However, another participant who was a teenager when he received the treatment showed no signs of recovery. So far, the stem cell gene therapy has restored the immune system of 30 out of 30 babies by using their own cells, during the different clinical trials.
The UCLA researchers used a strategy to correct the ADA-SCID mutation by genetically modifying the patient’s blood-forming stem cells, which can create all blood cell types. In the trial, blood stem cells were removed from each child’s bone marrow; and then were corrected in the lab by inserting the gene responsible for making the adenosine deaminase enzyme. Finally, each child received a transplant of their own corrected blood stem cells.
The fact that the nine babies were cured and the teenager was not indicates that the gene therapy for ADA-SCID works best in the youngest patients, before their bodies lose the ability to restore the immune system.
The next step is obtaining approval from the US Food and Drug Administration (FDA) to be able to treat children with ADA-SCID.
Source: UCLA Newsroom
