Inside STAT: Families reeling after FDA rejects therapy for kids without a thymus gland

CHARLIE LUCKESEN, 2, SNUGGLES WITH HIS MOTHER, KATIE, AT THEIR HOME IN SAN DIEGO. (SANDY HUFFAKER FOR STAT)

Katie Luckesen of San Diego was anxiously waiting to hear whether regulators would approve a life-saving treatment for her 2-year old son, Charlie. He was born without a thymus, the gland responsible for producing T cells and ensuring a functioning immune system. Without it, any infection — whether a common cold or everyday bacteria — could be deadly. Lukesen checked her phone over and over again on Wednesday, but the good news never came. The experimental therapy involves transplanting a sliver of thymus tissue from donor babies, and though 75% of the kids treated with it since 1991 are still alive, the FDA refused to approve the treatment, citing manufacturing concerns. In a devastating turn, the decision now leaves Charlie and other kids like him in the lurch. STAT’s Eric Boodman has more here.