Inside STAT: Waning treatment is a warning for all ‘one-and-done’ therapies

(MOLLY FERGUSON FOR STAT)

Severe combined immunodeficiency — or SCID — is a genetic disorder that hampers the growth of essential immune cells, leaving people vulnerable to any infection. For decades, bone marrow transplants have allowed many affected babies to survive past infancy and lead relatively normal lives. But it's become increasingly clear that these patients' immunity sometimes starts to fade decades after a transplant. Now, patient advocates are trying to change the way we talk about treatments — and not just for SCID. They see this experience as a cautionary tale for anyone receiving treatments that are touted as potentially curative. STAT’s Eric Boodman has more.