HIV used to cure 'bubble boy' disease

Giannina Alva and son Gael

The disease is called severe combined immunodeficiency, or SCID. "I think it's incredible".

Born in 1971, David lived his whole life from birth in a plastic bubble until his death at the age of 12.

For years, stem-cell transplantation from a matched sibling was a patient's best hope for survival, Mamcarz said. The disease remains a source of great interest to researchers.

Most patients were discharged from the hospital within one month.

The cells were then frozen, tested, and infused back into the patients. Viruses are often used as vectors because they're good infecting and sticking DNA into cells.

Merlin Crossley, professor of molecular biology at the University of New South Wales, added that getting a matched donor for stem cell transplants had always been hard, and that gene therapy was a promising alternative. And they think they've succeeded. We believe that this type of virus is safer and more effective for gene therapy.

The results of eight patients were published in the New England Journal of Medicine and revealed they all now have working immune systems.

Well, brilliant doctors have figured out how to use the HIV virus to "deliver" the missing gene to babies with bubble boy syndrome. The goal is to prevent gene therapy from inadvertently causing leukemia by switching on an oncogene in the patient's blood stem cells.

Mustang Bio, which is developing the lentiviral gene therapy for commercial use as MB-107, noted in a press release that the therapy is being considered for a broader range of patients.

The study was conducted on eight infants - including now-2-year-old Gael Jesus Pino Alva - with the SCID-X1 variation of the disease, by researchers at St. Jude Children's Research Hospital in Memphis, Tennessee. This helped the new cells take up permanent residence.

Gael Jesus Pino Alva
Gael Jesus Pino Alva Peter Barta St. Jude Children’s Research Hospital

By contrast, in the current trial, not only did seven of eight infants develop normal numbers of functioning T cells, B cells and natural killer (NK) cells, within three to four months of receiving the therapies, but the vector used to correct the mutation was created to avoid the possibility of leukemia or any other illness developing.

"This study marks the first time a lentivirus vector and targeted low-dose busulfan have been used as a primary treatment for newly diagnosed infants with SCID-X1", Mamcarz said.

But that has turned around, she said. He is "mixing with people and living like a normal toddler", St. Jude's officials said. Two more infants have been treated since the paper was prepared for publication, the team says. This only partially restores the immune system and causes the children to need lifelong treatment, with frequent and complex health problems, including persistent infections. As a result, patients require monthly infusions of antibodies called immunoglobulins. He just kept getting these infections'.

Of course, she adds, they'll be watched carefully for signs of leukemia and to see if the effect of the therapy is wearing off. Earlier versions of the treatment have been less efficient and also posed a risk of triggering leukemia.

The U.S. National Institutes of Health provides more about SCID.

"In addition to the newly diagnosed infants with XSCID, we believe there are hundreds of patients with the disease who have been treated with hematopoietic stem cell transplantation, but who are experiencing decreasing T cell immunity and increasing incidence of infections". "Sometimes their families don't understand just how profoundly affected their immune system is".

The sufferers had been handled at St Jude Youngsters' Analysis Hospital in Memphis and UCSF Benioff Kids' Hospital in San Francisco.

This latest advance is not only encouraging news for these rare patients. "These exciting new results suggest that gene therapy may be an effective treatment option for infants with this extremely serious condition, particularly those who lack an optimal donor for stem cell transplant".

"There is a lot of reason to be excited and hopeful for the future", Boyle said.

Clearly it has made a comeback and has been used to treat other rare diseases, including adrenoleukodystrophy, a neurological condition better known as Lorenzo's Oil disease, after a 1992 movie that highlighted a boy with the condition and his parents' hunt for a cure.

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