Infants Without Immune System can be Cured by Gene Therapy

A Gene Therapy developed by a team of researchers from UCLA and Great Ormond Street Hospital in London has successfully treated 48 of 50 children born with a rare and deadly inherited disorder that leaves them without an immune system. This study was published in the New England Journal of Medicine,Severe combined immunodeficiency due to adenosine deaminase deficiency is caused by mutations in the adenosine deaminase gene that creates the enzyme adenosine deaminase, which is essential to a functioning immune system. It is dangerous for the children to even do normal activities like going to school or playing with friends can lead to dangerous, life-threatening infections. ADA-SCID can be fatal within the first two years of life.

The Gene Therapy method involves first collecting some of the child’s blood-forming stem cells, which can create all types of blood and immune cells. Next by using an approach developed by the research team, a new copy of the adenosine deaminase gene is delivered into the stem cells by a modified lentivirus. The rectified cells are then returned to the child’s body, where they planned to generate a continual supply of healthy immune cells capable of fighting infection.

In a co-lead authors Dr Donald Kohn of UCLA and Dr Claire Booth of Great Ormond Street Hospital, or GOSH, reports two- and three-year outcomes for children treated with the investigational lentiviral gene therapy in clinical trials at GOSH, UCLA Mattel Children’s Hospital and the National Institutes of Health between 2012 and 2017.Booth, a GOSH consultant in pediatric immunology and gene therapy said that if this treatment is approved in the future this could be the standard for ADA-SCID, and for many other genetic conditions, removing the need to find a matched donor for a bone marrow transplant and the toxic side effects often associated with that treatment.

Dr Donald Kohn of UCLA and distinguished professor of microbiology, immunology and molecular genetics and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. said that totally three clinical trials were conducted and a totally of 50 patients were treated, and the overall results were very encouraging. All the patients are alive and well, and in more than 95% of them, the therapy appears to have corrected their underlying immune system problems.