For the first time, scientists\u00a0have\u00a0used\u00a0a cutting-edge cell therapy called CAR-T\u00a0to\u00a0treat a patient with three different life-threatening autoimmune diseases\u00a0that had resisted years of treatment.\u00a0The\u00a0patient,\u00a0who\u00a0once\u00a0required\u00a0daily blood infusions, has been in remission\u00a0without\u00a0needing\u00a0additional\u00a0treatment\u00a0for a year since\u00a0the CAR-T therapy. The case report,\u00a0publishing\u00a0April\u00a09\u00a0in the Cell Press journal\u00a0Med, suggests\u00a0that CAR-T therapies\u00a0can help treat\u00a0complex and\u00a0severe\u00a0autoimmune diseases.\u00a0<\/p>\n
“The treatment was extremely efficient in getting rid of all three autoimmune conditions\u00a0at once,” says corresponding author\u00a0Fabian M\u00fcller\u00a0of the\u00a0University Hospital\u00a0of Erlangen\u00a0in Germany.\u00a0“After being sick for more than a decade, the patient is now in treatment-free remission\u00a0and\u00a0able to return to an almost normal life. This therapy significantly improved her quality of life.”\u00a0<\/p>\n
In\u00a02025,\u00a0M\u00fcller\u00a0and his team met a 47-year-old female patient with severe\u00a0autoimmune\u00a0hemolytic anemia (AIHA),\u00a0a disease in which\u00a0the\u00a0immune system\u00a0mistakenly\u00a0attacks\u00a0and destroys\u00a0red blood cells.\u00a0<\/p>\n
In addition to AIHA,\u00a0she\u00a0had\u00a0been diagnosed with\u00a0two other\u00a0autoimmune diseases\u00a0with nearly opposing\u00a0symptoms.\u00a0She\u00a0had\u00a0immune thrombocytopenia\u00a0(ITP)-a disorder\u00a0that\u00a0caused\u00a0her dysregulated immune system\u00a0to\u00a0destroy\u00a0her platelets, increasing\u00a0the\u00a0risk of bleeding-and\u00a0antiphospholipid antibody syndrome-a\u00a0disease\u00a0that\u00a0raises\u00a0the risk of\u00a0dangerous\u00a0blood clots in her vessels.\u00a0<\/p>\n
Since the patient’s\u00a0diagnoses\u00a0more than a decade ago, she had\u00a0undergone\u00a0nine different lines of therapy, including antibody treatments,\u00a0steroids,\u00a0and\u00a0immune-suppressing medications.\u00a0None had\u00a0a\u00a0lasting impact.\u00a0<\/p>\n
When she met with\u00a0M\u00fcller’s team,\u00a0who had successfully treated patients with severe rheumatological autoimmune diseases-including lupus, a disorder that causes the body’s immune\u00a0cells\u00a0to go haywire and attack healthy tissues-the patient\u00a0was reliant\u00a0on\u00a0daily blood transfusions to manage her anemia\u00a0and permanent blood thinner\u00a0medication\u00a0to prevent clots.\u00a0<\/p>\n
Because all standard treatments had failed, the team\u00a0provided\u00a0her with\u00a0CAR-T\u00a0cell therapy, a\u00a0type\u00a0of “living drug”\u00a0that uses\u00a0a\u00a0patient’s\u00a0own immune cells to attack\u00a0harmful\u00a0cells,\u00a0which\u00a0has\u00a0been used to treat\u00a0several\u00a0cancers,\u00a0including leukemia,\u00a0or\u00a0cancer of the blood,\u00a0and\u00a0lymphoma,\u00a0or\u00a0cancer of the lymph nodes.\u00a0Dysregulated B cells\u00a0appeared to be\u00a0driving\u00a0this\u00a0patient’s\u00a0three\u00a0illnesses.\u00a0<\/p>\n
To\u00a0develop\u00a0the therapy, the team extracted the patient’s white blood cells and isolated\u00a0her\u00a0T cells<\/a>,\u00a0immune cells that\u00a0actively\u00a0scan\u00a0the body for\u00a0infected or abnormal cells and destroy them.\u00a0The researchers\u00a0then\u00a0re-engineered\u00a0the\u00a0patient’s\u00a0T cells\u00a0to recognize a protein called\u00a0CD19, which is\u00a0present on B cells-immune cells\u00a0that produce antibodies. They then infused the\u00a0CAR-T cells\u00a0back\u00a0into the patient\u00a0to look for\u00a0and\u00a0eliminate\u00a0all\u00a0her\u00a0B cells.\u00a0<\/p>\n The clinical effects were striking.\u00a0The patient\u00a0needed\u00a0her final blood transfusion\u00a0just one\u00a0week\u00a0after\u00a0the\u00a0treatment.\u00a0Two weeks later,\u00a0she\u00a0reported feeling\u00a0stronger and was\u00a0able to carry out\u00a0everyday activities.\u00a0Three\u00a0weeks after the treatment ended,\u00a0her\u00a0levels\u00a0of\u00a0hemoglobin, a protein in red blood cells, doubled and\u00a0returned to normal, suggesting her immune system was no longer destroying her red blood cells.\u00a0<\/p>\n At the same time, the therapy\u00a0improved\u00a0her other autoimmune conditions.\u00a0Her levels of antiphospholipid antibodies-associated with dangerous\u00a0blood clots-gradually fell and\u00a0remained negative.\u00a0Her platelet counts\u00a0also\u00a0stabilized.\u00a0<\/p>\n “After more than ten years of illness, the patient’s blood counts normalized within just a few weeks.\u00a0The speed and depth of the response was\u00a0remarkable,”\u00a0M\u00fcller\u00a0says.\u00a0<\/p>\n He adds that\u00a0the\u00a0reason why\u00a0the\u00a0therapy worked\u00a0so\u00a0effectively was\u00a0likely\u00a0because\u00a0the CAR-T cells\u00a0could\u00a0go into different tissues\u00a0throughout the body\u00a0and\u00a0eliminate\u00a0all\u00a0dysregulated cells, both in mature and developing stages.\u00a0When\u00a0the\u00a0patient’s\u00a0B cells eventually\u00a0returned\u00a0months later, they consisted\u00a0almost entirely\u00a0of naive cells,\u00a0suggesting the\u00a0treatment\u00a0reset her\u00a0immune\u00a0system.\u00a0<\/p>\n It has been a year since her treatment ended, and\u00a0the patient\u00a0still does not\u00a0require\u00a0transfusions\u00a0or\u00a0other treatments.\u00a0While\u00a0she\u00a0still has\u00a0lower white blood cell counts and\u00a0mild elevations in\u00a0liver enzymes associated with\u00a0potential damage to her bone marrow and liver, the team\u00a0says\u00a0these conditions\u00a0may be related to years of prior treatments rather than the CAR-T therapy itself.\u00a0<\/p>\n