GoKawiilAntibodies (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.
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The depletion of her red blood cells required repeat blood transfusions — an average of one and as many as three bags a day. A loss of platelets increased the risk of uncontrolled bleeding, and a loss of fat-binding proteins made the blood more prone to clotting. 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,” which broadly dampen the body’s immune system, boosting the risks of infection.
But because steroids and more advanced immunosuppressive medication failed to control her symptoms, says Müller, the woman was driven for 3 hours in an ambulance to his clinic, which has developed a reputation for manufacturing engineered immune cells called chimeric antigen receptor (CAR) T cells for one-off treatments. “It was her last chance,” says Müller, “for controlling the disease.”
Medication-free
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