Experimental treatment: a bubble child gets better

2023-11-27 21:01:36

Born without an immune system, the health of a child from the Navajo community improved thanks to therapy to rebuild his immune system cell by cell.

Hataałii Tiisyatonii “HT” Begay, 5, has severe combined immunodeficiency syndrome (SCID), which affects the body’s natural defense mechanism against infections. He’s a bubble child.

Nearly 70 children are born with SCID each year in Canada and the United States, but only two or three have the same type as HT. Without treatment, these children usually die within the first two years of life, the Washington Post.

At just six days old, HT had to be placed in isolation and airlifted to Phoenix Children’s Hospital. Two and a half months later, the little one became the first person in the world to receive an experimental gene therapy treatment at the Benioff Children’s Hospital at the University of California, San Francisco.

The goal of the treatment is to rebuild his immune system, cell by cell, and the outcome was uncertain.

“We just prayed for him,” said his grandmother Laverna Shorty. You know, you get through things with prayers, and you have to have faith. So I had faith in Western medicine and my own beliefs.”

HT paved the way for 13 other children, all of whom after him successfully received the same experimental therapy, which could become a long-term treatment, as doctors hope.

The next step is to get it accepted by pharmaceutical companies – and that is quite a challenge, recalled the Washington Post.

SCID is caused by mutation of at least 20 different genes. The disease is so rare that most pediatricians will probably never encounter a bubble child.

Some estimates have established that among the Navajo, or Diné, population, one in 2,000 children is born with SCID, compared to one in 58,000 for the rest of the population.

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