In a First, This Personalized Cell Therapy Treated Three Life-Threatening Autoimmune Diseases in One Patient
The patient was bedridden and needed daily blood transfusions, and she had tried nine different therapies that didn’t bring lasting effects. So, researchers modified some of her immune cells so they would attack her faulty cells
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A personalized therapy involving re-engineered cells has brought phenomenal results for a woman with a rare trio of life-threatening autoimmune diseases.
In a case report published April 9 in the journal Med, researchers describe the patient’s treatment and subsequent remission over the following year. After previously enduring nine different therapies—none of which brought lasting outcomes—her experience highlights the cell therapy’s potential to tackle severe autoimmune diseases.
“It was her last chance for controlling the disease,” study co-author Fabian Müller, a hematologist at the University Hospital of Erlangen in Germany, tells Nature’s Edward Chen.
When Müller and his colleagues first met the patient in 2025, she was 47 years old and suffering from three conditions that caused her immune system to mistakenly attack her body. Her red blood cells were being destroyed, and other affected components led to an increased risk of blood clots and of bleeding. She needed daily blood transfusions and was on permanent blood-thinning medication.
“She was deathly sick and bedridden at the time we met her,” Müller tells New Scientist’s Michael Le Page. But then, “we treated her, and seven days later, she got out of bed.”
All three illnesses stemmed from dysregulated B cells, immune cells that make infection-fighting antibodies. So the researchers took some of the patient’s T cells, which search for and destroy infected or abnormal cells, and altered them to identify a protein on the faulty B cells. Then, the team returned the modified T cells—called chimeric antigen receptor, or CAR T, cells—to the patient’s body so they would go after the troublemakers.
Quick fact: A CAR T first
Bill Ludwig, who had a form of leukemia, was the first person successfully treated with CAR T-cell therapy. He received it in 2010 at the Hospital of the University of Pennsylvania. Ludwig died of Covid-19 complications in 2021.
Three weeks after the treatment, blood tests indicated that the patient’s immune system had stopped destroying her red blood cells. It also relieved the other autoimmune conditions. Her number of blood platelets—cell fragments that stop a person’s bleeding—became stable, and her levels of antibodies related to dangerous blood clots decreased and stayed negative.
Over 11 months of follow-up observations, she had no major health issues from the CAR T-cell therapy. A few problems, such as lower counts of some immune cells, have lingered, but the researchers suspect they’re associated with her earlier treatments rather than the recent infusion. She stopped needing blood transfusions and medications.
“She is doing fine, back in her daily routine, with no therapy directed towards the three diseases since CAR T,” Müller tells Gizmodo’s Ed Cara.
These are encouraging results, especially since all three conditions appeared to respond to the therapy, although it’s hard to say how long the effects will last, says Ben Parker, a consultant rheumatologist at the Manchester University NHS Foundation Trust in England, who was not involved in the new research, to the Guardian’s Ian Sample.
The treatment’s success probably stems from CAR T cells’ ability to travel in various tissues of the body and destroy both mature and developing dysregulated cells. Her new B cells, which arrived months after receiving the therapy, were primed to encounter pathogens, suggesting the intervention reset her immune system.
The U.S. Food and Drug Administration has approved several CAR T-cell therapies for the treatment of certain blood cancers, like leukemia and lymphoma. And mounting research suggests that the approach might be useful for autoimmune diseases as well.
“We believe that using CAR T therapy earlier for patients with severe autoimmune disease could help prevent complications from years of ineffective treatments,” Müller says in a statement. “If we can intervene sooner, we may be able to stop the disease process, avoid organ damage and give patients their lives back.”
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