AIDS virus used in gene therapy to treat “bubble baby” disease

Gene therapy using the unlikely helper AIDS virus provided a functioning immune system for 48 babies and toddlers born without it, doctors reported Tuesday.

The results show that all but two of the 50 children who received treatment in the study are now capable of fighting healthy bacteria.

Dr. Donald Korn, research leader at UCLA Mattel Children’s Hospital, said, “We took what would otherwise have been a fatal illness and treated most of these children with a single treatment. Healed. “

“They are basically’free range’, go to school and do the usual things,” he said.

Two other children who did not receive the help of gene therapy later succeeded in bone marrow transplantation. Doctors say it takes time to find out if any of the 50 have healed, but it seems to be working so far.

The children had severe combined immunodeficiency syndrome (SCID). This is caused by a hereditary genetic defect that prevents the bone marrow from making healthy versions of the blood cells that form the immune system. Without treatment, it often kills in the first year or two of life.

In the 1970s, a Texas boy lived in protective plastic foam for 12 years to isolate it from bacteria, and became known as “bubble boy disease.” It is now called “Bubble Baby Disease”. This is because defects in about 20 different genes can be the cause, including those that affect girls as well as boys.

Bone marrow transplants from genetically matched siblings can cure the disorder, but most children lack the proper donors and treatment is dangerous-a boy in Texas died after one.

Currently, patients are treated with antibiotics and bacteria-fighting antibodies twice a week, but that is not a permanent solution.

Doctors think gene therapy may be. They remove some of the patient’s blood cells, use the disabled AIDS virus to insert a healthy version of the gene that the child needs, and return the cells through IV.

Josselyn Kish, now living in Las Vegas at the age of 11, had it at UCLA at the age of three. As a baby, she suffered from a rash, painful shingles, and frequent diarrhea, her mother Kim Carter said. “She was always feverish, so day care called me several times a week to pick her up.”

“She got better soon,” Carter said after gene therapy. Now, “she rarely gets sick,” and she was able to recover whenever she was. That hope extends to Jocelyn’s latest infection — she has just been diagnosed with COVID-19 and has had very mild symptoms so far.

A total of 27 children were treated at a hospital in Los Angeles, three at the US National Institutes of Health near Washington and 20 at the Great Ormond Street Hospital in London. Dr. Stephen Gottschalk of St. Jude Children’s Research Hospital in Memphis said the fact that treatment seems safe in multiple hospitals providing treatment makes the study “very powerful.”

Although he played no role in the new study, he and his colleagues gave similar gene therapy to 17 other children with SCID.

“People ask us, is it a cure? Who knows the long term, but for at least three years, these kids are doing well,” Gottschalk said. “Immune function seems to be stable over time, so I think it looks very encouraging.”

The results of the UCLA-led study were published by the New England Journal of Medicine on Tuesday and presented at an online conference of the American Society for Gene and Cell Therapy. Grants from the California Institute of Regenerative Medicine, backed by US and UK government health agencies and taxation, were paid for the job. Korn is the inventor of the therapy and an advisor to London-based Orchard Therapeutics, which is currently developing it.

___

The Associated Press’s Department of Health Sciences is supported by the Department of Science Education at the Howard Hughes Medical Institute. AP is solely responsible for all content.