Experts have used an unusual gene therapy with the AIDS virus to provide functional immune systems to infants who have been born with bubble baby disease. Around 48 babies have been treated with this gene therapy so far, who have been born with ineffective immune systems. Health experts have confirmed that around 48 out of 50 children who have been treated with the new treatment in an experimental study have successfully developed germ-fighting abilities. The lead author of the study, Dr. Donald Kohn from UCLA Mattel Children’s Hospital has said that with a single treatment, he has been able to treat most children who have been dealing with this fatal disease. He has said that now these children can freely go to schools; play outside without the worry of any infection that can turn dangerous for them.
Two children who have not responded to gene therapy, later have been able to get successful bone marrow transplants. The authors of the study have said that it will take some time to know if any of these children are fully cured of the disease but so far, they are doing well. These children have been dealing with a life-threatening disease that is called severe combined immunodeficiency syndrome (SCID). Experts have said that the disease is led by an inherited genetic flaw that blocks bone marrow from making healthy versions of the blood cells that are responsible for the formation of the immune system. They have said that without early treatment, it can claim the lives of infants within one or two years of their birth. Earlier, it has been known as bubbly boy disease as a boy in Texas who has been diagnosed with the disease has lived in a protective plastic bubble for 12 years for his protection from germs and other infection in 1970.
Now, the disease is known as bubble baby disease as around 20 diverse gene defects that can cause the disease, are known to affect girls and boys both. Health experts have said that a bone marrow transplant from a genetically matched sibling can treat the disease but most children do not have a suitable donor and this treatment is quite risky as well. Health care providers treat these children with twice-weekly doses of antibiotics and germ-fighting antibodies. However, it is not a permanent solution to the disease. Now experts hope that gene therapy might be able to cure this disease. Scientists have removed some blood cells of patients and have used an inactive version of the AIDS virus to transfer healthy versions of the genes that children require. Later, they have reinjected the blood cells through IV.
A girl named Josselyn Kish, who is now 11 years old, has received this gene therapy at UCLA at the age of 3 years. Her family has informed scientists that earlier she has been dealing with painful shingles, rashes, and frequent diarrhea and fever. After she has been treated with gene therapy, she has become far better. The girl is now able to recover if rarely she falls sick. She has been diagnosed with COVID19 as well, but she has not shown any major symptoms so far. As per the report, around 27 kids with bubble baby disease have been given this gene therapy at the Los Angels hospital, three kids have been treated at the US National Institutes of Health and 20 children have been given the treatment at Great Ormond Street Hospital in London.
Dr. Stephen Gottschalk from St. Jude Children’s Research Hospital in Memphis has said that gene therapy appears to be safe and can be performed safely across multiple hospitals. He has not been involved in the current study, but he has performed the same kind of gene therapy on 17 kids who have been dealing with SCID. Dr. Gottschalk has said that it is uncertain how long this gene therapy will work but the kids who have been given this therapy will be safe for at least three years. The results of the study have been released in the New England Journal of Medicine. The findings have been presented at an online American Society of Gene and Cell Therapy conference.