One woman, three autoimmune diseases: CAR-T therapy vanquishes ultra-rare disease trio
-
-
Bluesky
-
-
-
-
-
X
Antibodies (three-lobed structures, artist’s impression) that attack a person’s own tissues can lead to autoimmune disease.Credit: Ruslanas Baranauskas/Science Photo Library
A woman with an ultra-rare combination of three autoimmune diseases has had no symptoms since receiving a single dose of engineered immune cells, doctors in Germany report today1. She had previously received nine other types of treatment without getting better, could no longer work and was sometimes bedridden for weeks with pain and fatigue. “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 and co-authored the report.
Without the engineered cells, the woman, who was 47 when she met Müller and his colleagues, would have had a “terrible” quality of life, says Carl June, an immunologist at the University of Pennsylvania in Philadelphia who pioneered the use of similar cells to treat cancer, “if she would even be alive.”
Rogue B cells
The woman’s trifecta of autoimmune diseases stemmed from problems with her B cells, a type of immune cell. Her B cells were making antibodies that mistakenly attacked her own red blood cells, causing the disease autoimmune haemolytic anaemia. They also attacked her platelets, causing immune thrombocytopenia, and some fat-binding proteins, causing antiphospholipid syndrome.
Turbocharged ‘killer’ cells show promise for autoimmune disease