CAR-T therapy drives remission in patient with three autoimmune diseases, suggests study

For the first time, scientists have used a cutting-edge cell therapy called CAR-T to treat a patient with three different life-threatening autoimmune diseases that had resisted years of treatment. The patient, who once required daily blood infusions, has been in remission without needing additional treatment for a year since the CAR-T therapy. The case report, publishing April 9 in the Cell Press journal Med, suggests that CAR-T therapies can help treat complex and severe autoimmune diseases.

“The treatment was extremely efficient in getting rid of all three autoimmune conditions at once,” says corresponding author Fabian Müller of the University Hospital of Erlangen in Germany. “After being sick for more than a decade, the patient is now in treatment-free remission and able to return to an almost normal life. This therapy significantly improved her quality of life.”

In 2025, Müller and his team met a 47-year-old female patient with severe autoimmune hemolytic anemia (AIHA), a disease in which the immune system mistakenly attacks and destroys red blood cells.

In addition to AIHA, she had been diagnosed with two other autoimmune diseases with nearly opposing symptoms. She had immune thrombocytopenia (ITP)—a disorder that caused her dysregulated immune system to destroy her platelets, increasing the risk of bleeding—and antiphospholipid antibody syndrome—a disease that raises the risk of dangerous blood clots in her vessels.

Since the patient’s diagnoses more than a decade ago, she had undergone nine different lines of therapy, including antibody treatments, steroids, and immune-suppressing medications. None had a lasting impact.

When she met with Müller’s team, who had successfully treated patients with severe rheumatological autoimmune diseases—including lupus, a disorder that causes the body’s immune cells to go haywire and attack healthy tissues—the patient was reliant on daily blood transfusions to manage her anemia and permanent blood thinner medication to prevent clots.

Because all standard treatments had failed, the team provided her with CAR-T cell therapy, a type of “living drug” that uses a patient’s own immune cells to attack harmful cells, which has been used to treat several cancers, including leukemia, or cancer of the blood, and lymphoma, or cancer of the lymph nodes. Dysregulated B cells appeared to be driving this patient’s three illnesses.

To develop the therapy, the team extracted the patient’s white blood cells and isolated her T cells, immune cells that actively scan the body for infected or abnormal cells and destroy them. The researchers then re-engineered the patient’s T cells to recognize a protein called CD19, which is present on B cells—immune cells that produce antibodies. They then infused the CAR-T cells back into the patient to look for and eliminate all her B cells.

The clinical effects were striking. The patient needed her final blood transfusion just one week after the treatment. Two weeks later, she reported feeling stronger and was able to carry out everyday activities. Three weeks after the treatment ended, her levels of hemoglobin, a protein in red blood cells, doubled and returned to normal, suggesting her immune system was no longer destroying her red blood cells.

At the same time, the therapy improved her other autoimmune conditions. Her levels of antiphospholipid antibodies—associated with dangerous blood clots—gradually fell and remained negative. Her platelet counts also stabilized.

“After more than ten years of illness, the patient’s blood counts normalized within just a few weeks. The speed and depth of the response was remarkable,” Müller says.

He adds that the reason why the therapy worked so effectively was likely because the CAR-T cells could go into different tissues throughout the body and eliminate all dysregulated cells, both in mature and developing stages. When the patient’s B cells eventually returned months later, they consisted almost entirely of naive cells, suggesting the treatment reset her immune system.

It has been a year since her treatment ended, and the patient still does not require transfusions or other treatments. While she still has lower white blood cell counts and mild elevations in liver enzymes associated with potential damage to her bone marrow and liver, the team says these conditions may be related to years of prior treatments rather than the CAR-T therapy itself.

“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. “If we can intervene sooner, we may be able to stop the disease process, avoid organ damage, and give patients their lives back.”

Reference:

Korte I, Kharboutli S, Völkl S, CD19 CAR-T therapy induces remission in refractory autoimmune hemolytic anemia with ITP and antiphospholipid syndrome, Med, DOI:10.1016/j.medj.2026.101075