Families are reeling after FDA rejects therapy for kids born without a thymus gland

When Charlie Luckesen turned 2 the Wednesday before Thanksgiving, his family celebrated with a golden balloon nearly twice his height, a construction-paper banner that spelled out “Oh Twodles,” and an enormous, icing-swirled cake. But the whole day was tinged with unease.

Before his birth, a flock of cells that should have swept around from his nascent spinal cord to his chest was somehow thrown off, and he never developed an organ called the thymus. Not having a thymus meant not having T cells, and not having T cells meant not having a functional immune system. This ultra-rare condition, known as pediatric congenital athymia, left Charlie deeply unprepared for life outside the womb. To him, a common cold or an everyday speck of bacteria could be deadly.

“My oldest — the one that’s 7 — he’s frequently asked what happens if Charlie dies,” said their mother, Katie Luckesen, who lives in San Diego. To her, it’s a legitimate question: Her whole family knows that without treatment, Charlie’s disorder is usually fatal by 2. “And here he is, at two years old…”

All that was supposed to change on Wednesday, when the Food and Drug Administration had to decide whether it was approving a tissue implant that can dramatically increase such children’s chances of survival. Since 1993, 101 children have gotten the experimental treatment at Duke University Hospital, and 73 of them are still alive. With numbers like that, the approval seemed like a shoo-in, almost a formality. As soon as the positive decision was announced, the Luckesens were told, they’d get a call about scheduling Charlie’s surgery.

On Wednesday, Katie Luckesen checked her phone again and again — while snuggling with her daughter, while helping the kids with schoolwork, while making stovetop, the Cambridge, Mass., company that commercialized the treatment.