The woman suffered from autoimmune haemolytic anaemia (AIHA), immune thrombocytopenia (ITP), and antiphospholipid syndrome (APS), conditions that had proven resistant to previous interventions. She had tried nine different treatments before CAR T-cell therapy, none of which had a lasting impact. According to Prof Fabian Müller, the patient had no treatment options left and would not have left the ward as she needed daily blood transfusions and permanent blood thinning medication.
The CAR T-cell therapy was administered at University Hospital Erlangen in Germany last year. Within weeks of the therapy, all three diseases responded, with the woman having her last blood transfusion a week after treatment and becoming strong enough for everyday activities two weeks later. According to Prof Fabian Müller, the speed and depth of the woman’s response was remarkable, noting the therapy had significantly improved her quality of life.
The therapy destroyed rogue B-cells, and when B-cells bounced back months later, they appeared healthy, suggesting the therapy reset the immune system. This scientific breakthrough indicates a potential mechanism for addressing autoimmune disorders at their root cause, though the specific processes by which the therapy resets the immune system to produce healthy B-cells remain under investigation. The woman has been in treatment-free remission for 14 months and is largely able to return to normal life.
She still has a low white blood cell count and slightly raised liver enzymes, which researchers believe are due to previous treatments rather than CAR-T therapy. The long-term durability of this remission is not yet confirmed, and it is unknown how effective the therapy will be for other autoimmune diseases beyond this case. Details of the case are published in the journal Med.
Clinical trials are needed to learn how durable the therapy is and whether it will be effective for other autoimmune diseases, according to Prof Fabian Müller. Further research must also address potential side effects or risks of using CAR T-cell therapy for autoimmune conditions in broader populations, as well as the availability and cost of this therapy for other patients with similar autoimmune diseases.