Successful off-label use of CAR-T therapy in a child with lupus
In June 2023, a patient at Universitätsklinikum Erlangen in Germany became the first child with lupus to receive CAR-T therapy.
The treatment was the last resort to slow down systemic lupus erythematosus (SLE), which is a serious autoimmune disease that was attacking the patient’s body and seriously affected her ability to lead a normal life.
The treatment team in Erlangen has now published the results of this successful treatment in the medical journal The Lancet.
“Simply the fact that we have administered CAR-T cells for an autoimmune disease is something special, since it was previously only authorised for certain advanced types of cancer,” explains Dr Tobias Krickau, Paediatric Rheumatologist at the Department of Pediatrics and Adolescent Medicine at Universitätsklinikum Erlangen.
“As Uresa’s SLE was progressing at an increasingly fast pace despite all her medication, we were confronted with the question of whether we should give these immune cells modified in a lab to a child. This off-label treatment for an autoimmune disease in a child had never been attempted anywhere in the world, as far as we knew.”
Positive results in adults with autoimmune diseases
The hospital team initiated the CAR-T cell therapy for Uresa as part of an extended access programme for severely ill patients in accordance with the German Medicinal Products Act and the Ordinance on Medicinal Products for Compassionate Use.
The experiment was based on previous success with CAR-T therapy. Since 2021, a team at the Deutsche Zentrum Immuntherapie (DZI) have successfully treated patients with various autoimmune diseases (including SLE) using CAR-T cells. In February 2024, they presented 15 cases as part of a pilot study in theNew England Journal of Medicine.
“Her kidney and lupus values improved from the third week after the treatment,” said Dr Krickau. “We had never achieved that with any medication previously. She was seriously ill and her kidneys have now fully recovered, something nobody believed could happen to this extent.”
In conjunction with the paediatric oncology department, Krickau is now planning a CAR-T study in other children and teenagers with automimmune diseases.
DZI at Universitätsklinikum Erlangen is a partnership of 18 clinical departments and institutions and serves as a central port of call for patients with chronic and acute inflammatory diseases, autoimmune diseases and cancer.
Diana Spencer, Senior Digital Content Editor, DDW