She was born without an immune system, but gene therapy saved her life

By Simar Bajaj

The New York Times

A common cold was enough to kill Cora Oakley.

Born in Morristown, N.J., with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare genetic condition that leaves the body without key white blood cells.

It’s better known as “bubble boy disease,” popularized by the 1976 movie, “The Boy in the Plastic Bubble.” Although nobody lives inside a plastic bubble anymore, most babies with SCID wouldn’t make it past their first birthday without treatment.

“It seems like you have this perfectly healthy child who’s happy and hitting her milestones,” said Chelsea Ferrier, Cora’s mother. “But no — she is sick. She can’t do anything. She can’t go outside.”

The standard treatment for SCID is a bone-marrow transplant, but it works best with a perfectly compatible sibling donor. Cora was Ferrier’s first child, her “miracle” after three years of trying and several miscarriages, so she had no brother or sister.

But in 2017, there was a new treatment in clinical trials — a gene therapy that could potentially fix Cora’s stem cells instead of replacing them with a donor’s. Every year in the United States, up to 100 babies are born with SCID, and this disease can be caused by more than 20 genetic defects. But Cora had ADA-SCID, one of the few types being targeted with gene therapy to restore patients’ immune system.

“I would have sold my organs to get into that trial,” Fer...