Doctors say a gene therapy that uses the AIDS virus has given the working immune system to 48 infants and children who were born without one
Doctors reported on Tuesday that a gene therapy using a helpless adjuvant, which is an AIDS virus, gave the working immune system to 48 infants and children who were born without one.
Results suggest that two out of all 50 children given for experimental therapy in one study now have healthy germ-fighting ability.
UCLA Mattel Children’s Hospital study leader Drs. Donald Cohan said, “What we are taking is a fatal disease.
“They are basically ‘free range’ – going to school, doing normal things,” without worrying that an infection could become life-threatening, he said.
The other two children, who were not helped by gene therapy, were later successful in transplanting bone marrow. Doctors say it will take longer to know if any of the 50 have been cured, but they have recovered so far.
Children had severe combined immunodeficiency syndrome or SCID, which is caused by a hereditary genetic defect that maintains the bone marrow by forming healthy versions of blood cells that build the immune system. Without treatment, it often kills the first year or two of life.
It became known as “Bubble Boy Disease” in the 1970s due to a case involving a Texas boy who lived in a protective plastic bubble for 12 years, separating him from germs. It is now called “bubble baby disease” because about 20 different gene defects, including some affecting girls as well as boys, can cause it.
Genetically matched sibling can cure a bone marrow transplant disorder, but most children lack a suitable donor and the treatment is risky – a Texas boy died one after another.
Patients are now treated with two-weekly doses of antibiotics and anti-germ antibodies, but this is not a permanent solution.
Doctors think that gene therapy can occur. They remove some of the patient’s blood cells, use an inefficient AIDS virus to insert a healthy version of the gene that children need, and return the cells via an IV.
Joslien Kish, now 11 years old and a resident of Las Vegas when she was at UCLA, was 3. As a child, she had rashes, painful herpes and frequent diarrhea, said her mother Kim Carter. “Day care was calling me a couple of times a week to get her because she was always there.”
After gene therapy, “he was better now,” Carter said. Now, “she rarely, rarely gets sick” and whenever she will be able to recover. It is hoped that Josslin’s latest infection expands – he had just been diagnosed with COVID-19 and has only very mild symptoms until now.
In total, 27 children were treated at Los Angeles Hospital, three at the US National Institutes of Health near Washington, and 20 at Great Ormond Street Hospital in London. The fact that the treatment is safe in the many hospitals it demonstrates makes this study “very powerful”, Dr. of St. Jude Children’s Research Hospital in Memphis. Stefan Gottschelk said.
He had no role in the new study but he and his colleagues have performed a similar gene therapy on 17 other children with SCID.
“People ask us, is this a cure?” Who knows, for a long time, but for at least three years, these children are doing well, ”said Gotsalak. “Immune function seems stable over time so I think it sounds very, very encouraging.”
The results of the UCLA-led study were published on Tuesday by the New England Journal of Medicine and presented at an online American Society of Gene and Cell Therapy conference. A grant from the US and British government health agencies and the tax-supported California Institute for Regenerative Medicine paid for the work. Cohan is the inventor of the treatment and is now developing a consultant from the company, London-based Orchard Therapeutics.
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