Miltenyi Biopharma's CD19-targeting CAR-T therapy zorpocabtagene-autoleucel (zorpo-cel) induced remission in all three autoimmune diseases—autoimmune hemolytic anemia (AIHA), antiphospholipid syndrome (APLAS), and immune thrombocytopenia (ITP)—in a 47-year-old woman refractory to nine prior therapies.1
The patient, treated at University Hospital Erlangen, Germany, has been in remission for 11 months post-treatment, leading a normal life without hospitalizations or blood transfusions, though requiring weekly blood-letting for iron overload.1
No cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome occurred, unlike common side effects in CAR-T for blood cancers.1
The case was detailed in the journal Med on April 9 by Fabian Müller, M.D., Ph.D., head of the CAR-T unit.1
Miltenyi is advancing zorpo-cel in phase 1/2 trials for lupus (NCT06347718) and has shown success in other autoimmune cases like ulcerative colitis.14
Sources:
1. https://www.fiercebiotech.com/research/first-car-t-cell-therapy-drives-3-autoimmune-diseases-remission-once
4. https://www.miltenyibiomedicine.com/cell-therapy/clinical-programs/