One woman, three autoimmune diseases: CAR-T therapy vanquishes ultra-rare disease trio

""Her disease got completely out of hand" and became "very life-threatening," says Fabian Müller, a haematologist at University Hospital Erlangen in Germany who helped to treat her."

"Without the engineered cells, the woman... would have had a "terrible" quality of life, says Carl June, an immunologist at the University of Pennsylvania."

"The flaws in the immune system that make one autoimmune disease possible can also increase the likelihood of a second, says Müller, but the woman was the first person he had seen with three such diseases."

"That combination "can kill you very rapidly," says June. "Usually, there's no cure other than treatment with long-term, high-dose steroids.""

A 47-year-old woman with three autoimmune diseases had no symptoms after a single dose of engineered immune cells. Previously, she endured nine treatments without improvement, leading to severe pain and fatigue. Her conditions included autoimmune hemolytic anemia, immune thrombocytopenia, and antiphospholipid syndrome, all linked to malfunctioning B cells. The engineered cells provided a potential cure, contrasting with the usual reliance on long-term steroids, which carry significant risks. Experts noted that her quality of life would have been severely compromised without this innovative treatment.

Read at Nature