Waning Treatment for Immune Deficiency Is a Warning for All ‘One-and-Done’ Therapies

December 19, 2019

(STAT News) – Then, when Ray was around 18, his immunity began to wane. For him, it came in the form of a norovirus he couldn’t shake. For others with the same rare disease, it appears as pneumonia or gastrointestinal trouble or an unexpected T cell decline. Over the last 10 years, the trend has become increasingly clear: The bone marrow transplants that kept certain babies with SCID alive sometimes stop working after years or decades of providing fairly reliable immune defenses. Now, to patient advocates, this has become an urgent lesson in the language people use to talk about treatment — and not just for SCID. They see their community’s experience as a cautionary tale for anyone developing or receiving a therapy that’s marketed as potentially curative.