Woman with three deadly diseases has ‘remarkable’ recovery after cell therapy

A woman who lived with three life-threatening autoimmune diseases for more than a decade has returned to a near-normal life after a cell therapy reset her wayward immune system.The 47-year-old had had nine different treatments, none of which had a lasting impact, before receiving the therapy last year at University Hospital Erlangen in Germany. At the time, she required daily blood transfusions and permanent blood thinning medication to control her illness.Within weeks of having the cell therapy, doctors noticed that all three diseases had responded, marking a world first and a striking improvement in the woman’s condition. For the past 14 months she has been in treatment-free remission and largely able to return to normal life.Prof Fabian Müller, who led the team, said the speed and depth of the woman’s response was “remarkable” and the therapy had “significantly improved her quality of life”. Clinical trials were needed to learn how durable the therapy was and whether it would be effective for other autoimmune diseases, he said.The woman had a rare, life-threatening blood disorder, autoimmune haemolytic anaemia (AIHA), whereby rogue immune defences destroy red blood cells. In flare-ups, patients need immunosuppressant drugs and regular blood transfusions. In the woman’s case, standard treatments were no longer working. “The patient had no treatment options left and would not have left the ward as she needed daily transfusions,” Müller said.In addition to AIHA, the woman had two other autoimmune diseases. One, immune thrombocytopenia (ITP), is driven by immune cells destroying platelets, which raises the risk of bleeding. The other, known as antiphospholipid syndrome (APS), has an opposing effect and raises the risk of harmful blood clots. All three diseases were due to wayward B-cells which make infection-fighting antibodies.With no options left, the doctors offered a treatment known as CAR (chimeric antigen receptor) T-cell therapy, which has proved gamechanging for certain cancers. The team extracted the woman’s white blood cells and isolated her T-cells, which patrol the body and kill infected or abnormal cells. The doctors engineered the T-cells to recognise a protein called CD19 found on B-cells and re-infused them into the patient.The therapy swiftly went to work, destroying the rogue B-cells. The woman had her last blood transfusion a week after the treatment and was strong enough for everyday activities two weeks later. Her immune system appeared to have stopped attacking her red blood cells, and her other autoimmune conditions improved. When her B-cells bounced back months later, they appeared to be healthy, suggesting the therapy reset her immune system. Details are published in the journal Med.The woman still has a low white blood cell count and slightly raised liver enzymes, but the researchers believe these are due to years of previous treatments rather than the CAR-T therapy.Prof Ben Parker, a consultant rheumatologist at the Kellgren Centre for Rheumatology at Manchester University NHS foundation trust, said it was encouraging that all the patient’s conditions seemed to respond to the therapy. “The prolonged response off normal therapy suggests there has been an immune reset,” he said, though how long it would last remained unclear.Parker, who is leading CAR-T trials on lupus and related autoimmune conditions in Manchester, said: “There are many active trials currently recruiting across autoimmune conditions, including lupus, myositis, MS, systemic sclerosis, vasculitis, and some have already reported early results. Case reports don’t prove a treatment works for wider use, hence the need for trials.”